Compassionate Use Trials: Providing an investigational therapy to a patient who is not eligible to receive that therapy in a clinical trial, but who has a serious or life-threatening illness for which other treatments are not available. Compassionate use trials allow patients to receive promising but not yet fully studied or approved therapies when no other treatment option exists. Also called expanded access trial.Treatment Outcome: Evaluation undertaken to assess the results or consequences of management and procedures used in combating disease in order to determine the efficacy, effectiveness, safety, and practicability of these interventions in individual cases or series.Parenteral Nutrition: The administering of nutrients for assimilation and utilization by a patient who cannot maintain adequate nutrition by enteral feeding alone. Nutrients are administered by a route other than the alimentary canal (e.g., intravenously, subcutaneously).Parenteral Nutrition, Total: The delivery of nutrients for assimilation and utilization by a patient whose sole source of nutrients is via solutions administered intravenously, subcutaneously, or by some other non-alimentary route. The basic components of TPN solutions are protein hydrolysates or free amino acid mixtures, monosaccharides, and electrolytes. Components are selected for their ability to reverse catabolism, promote anabolism, and build structural proteins.Liver Diseases: Pathological processes of the LIVER.Parenteral Nutrition, Home: The at-home administering of nutrients for assimilation and utilization by a patient who cannot maintain adequate nutrition by enteral feeding alone. Nutrients are administered via a route other than the alimentary canal (e.g., intravenously, subcutaneously).Parenteral Nutrition Solutions: Specialized solutions for PARENTERAL NUTRITION. They may contain a variety of MICRONUTRIENTS; VITAMINS; AMINO ACIDS; CARBOHYDRATES; LIPIDS; and SALTS.Consent Forms: Documents describing a medical treatment or research project, including proposed procedures, risks, and alternatives, that are to be signed by an individual, or the individual's proxy, to indicate his/her understanding of the document and a willingness to undergo the treatment or to participate in the research.Ganciclovir: An ACYCLOVIR analog that is a potent inhibitor of the Herpesvirus family including cytomegalovirus. Ganciclovir is used to treat complications from AIDS-associated cytomegalovirus infections.Cytomegalovirus Infections: Infection with CYTOMEGALOVIRUS, characterized by enlarged cells bearing intranuclear inclusions. Infection may be in almost any organ, but the salivary glands are the most common site in children, as are the lungs in adults.Cytomegalovirus: A genus of the family HERPESVIRIDAE, subfamily BETAHERPESVIRINAE, infecting the salivary glands, liver, spleen, lungs, eyes, and other organs, in which they produce characteristically enlarged cells with intranuclear inclusions. Infection with Cytomegalovirus is also seen as an opportunistic infection in AIDS.Informed Consent: Voluntary authorization, by a patient or research subject, with full comprehension of the risks involved, for diagnostic or investigative procedures, and for medical and surgical treatment.Wasting Syndrome: A condition of involuntary weight loss of greater then 10% of baseline body weight. It is characterized by atrophy of muscles and depletion of lean body mass. Wasting is a sign of MALNUTRITION as a result of inadequate dietary intake, malabsorption, or hypermetabolism.Clostridium difficile: A common inhabitant of the colon flora in human infants and sometimes in adults. It produces a toxin that causes pseudomembranous enterocolitis (ENTEROCOLITIS, PSEUDOMEMBRANOUS) in patients receiving antibiotic therapy.Clostridium Infections: Infections with bacteria of the genus CLOSTRIDIUM.Enterocolitis, Pseudomembranous: An acute inflammation of the INTESTINAL MUCOSA that is characterized by the presence of pseudomembranes or plaques in the SMALL INTESTINE (pseudomembranous enteritis) and the LARGE INTESTINE (pseudomembranous colitis). It is commonly associated with antibiotic therapy and CLOSTRIDIUM DIFFICILE colonization.Metronidazole: A nitroimidazole used to treat AMEBIASIS; VAGINITIS; TRICHOMONAS INFECTIONS; GIARDIASIS; ANAEROBIC BACTERIA; and TREPONEMAL INFECTIONS. It has also been proposed as a radiation sensitizer for hypoxic cells. According to the Fourth Annual Report on Carcinogens (NTP 85-002, 1985, p133), this substance may reasonably be anticipated to be a carcinogen (Merck, 11th ed).Vancomycin: Antibacterial obtained from Streptomyces orientalis. It is a glycopeptide related to RISTOCETIN that inhibits bacterial cell wall assembly and is toxic to kidneys and the inner ear.Clostridium: A genus of motile or nonmotile gram-positive bacteria of the family Clostridiaceae. Many species have been identified with some being pathogenic. They occur in water, soil, and in the intestinal tract of humans and lower animals.Fat Emulsions, Intravenous: Emulsions of fats or lipids used primarily in parenteral feeding.Emulsions: Colloids formed by the combination of two immiscible liquids such as oil and water. Lipid-in-water emulsions are usually liquid, like milk or lotion. Water-in-lipid emulsions tend to be creams. The formation of emulsions may be aided by amphiphatic molecules that surround one component of the system to form MICELLES.Surgical Tape: A flat, flexible strip of material used to cover or fasten together damaged tissue.Catheterization, Peripheral: Insertion of a catheter into a peripheral artery, vein, or airway for diagnostic or therapeutic purposes.Cushing Syndrome: A condition caused by prolonged exposure to excess levels of cortisol (HYDROCORTISONE) or other GLUCOCORTICOIDS from endogenous or exogenous sources. It is characterized by upper body OBESITY; OSTEOPOROSIS; HYPERTENSION; DIABETES MELLITUS; HIRSUTISM; AMENORRHEA; and excess body fluid. Endogenous Cushing syndrome or spontaneous hypercortisolism is divided into two groups, those due to an excess of ADRENOCORTICOTROPIN and those that are ACTH-independent.Mifepristone: A progestational and glucocorticoid hormone antagonist. Its inhibition of progesterone induces bleeding during the luteal phase and in early pregnancy by releasing endogenous prostaglandins from the endometrium or decidua. As a glucocorticoid receptor antagonist, the drug has been used to treat hypercortisolism in patients with nonpituitary CUSHING SYNDROME.Serial Publications: Publications in any medium issued in successive parts bearing numerical or chronological designations and intended to be continued indefinitely. (ALA Glossary of Library and Information Science, 1983, p203)Abortifacient Agents, Steroidal: Steroidal compounds with abortifacient activity.Keratoconus: A noninflammatory, usually bilateral protrusion of the cornea, the apex being displaced downward and nasally. It occurs most commonly in females at about puberty. The cause is unknown but hereditary factors may play a role. The -conus refers to the cone shape of the corneal protrusion. (From Dorland, 27th ed)Riboflavin: Nutritional factor found in milk, eggs, malted barley, liver, kidney, heart, and leafy vegetables. The richest natural source is yeast. It occurs in the free form only in the retina of the eye, in whey, and in urine; its principal forms in tissues and cells are as FLAVIN MONONUCLEOTIDE and FLAVIN-ADENINE DINUCLEOTIDE.Corneal Pachymetry: Measurement of the thickness of the CORNEA.Corneal Stroma: The lamellated connective tissue constituting the thickest layer of the cornea between the Bowman and Descemet membranes.Photosensitizing Agents: Drugs that are pharmacologically inactive but when exposed to ultraviolet radiation or sunlight are converted to their active metabolite to produce a beneficial reaction affecting the diseased tissue. These compounds can be administered topically or systemically and have been used therapeutically to treat psoriasis and various types of neoplasms.Cross-Linking Reagents: Reagents with two reactive groups, usually at opposite ends of the molecule, that are capable of reacting with and thereby forming bridges between side chains of amino acids in proteins; the locations of naturally reactive areas within proteins can thereby be identified; may also be used for other macromolecules, like glycoproteins, nucleic acids, or other.Ultraviolet Therapy: The use of ultraviolet electromagnetic radiation in the treatment of disease, usually of the skin. This is the part of the sun's spectrum that causes sunburn and tanning. Ultraviolet A, used in PUVA, is closer to visible light and less damaging than Ultraviolet B, which is ionizing.Pharmacology, Clinical: The branch of pharmacology that deals directly with the effectiveness and safety of drugs in humans.Ethics, Pharmacy: The principles of proper professional conduct concerning the rights and duties of the pharmacist, relations with patients and fellow practitioners, as well as actions of the pharmacist in health care and interpersonal relations with patient families. (From Stedman, 25th ed)Clinical Trials as Topic: Works about pre-planned studies of the safety, efficacy, or optimum dosage schedule (if appropriate) of one or more diagnostic, therapeutic, or prophylactic drugs, devices, or techniques selected according to predetermined criteria of eligibility and observed for predefined evidence of favorable and unfavorable effects. This concept includes clinical trials conducted both in the U.S. and in other countries.Drug Discovery: The process of finding chemicals for potential therapeutic use.Drug Design: The molecular designing of drugs for specific purposes (such as DNA-binding, enzyme inhibition, anti-cancer efficacy, etc.) based on knowledge of molecular properties such as activity of functional groups, molecular geometry, and electronic structure, and also on information cataloged on analogous molecules. Drug design is generally computer-assisted molecular modeling and does not include pharmacokinetics, dosage analysis, or drug administration analysis.Research Design: A plan for collecting and utilizing data so that desired information can be obtained with sufficient precision or so that an hypothesis can be tested properly.Research: Critical and exhaustive investigation or experimentation, having for its aim the discovery of new facts and their correct interpretation, the revision of accepted conclusions, theories, or laws in the light of newly discovered facts, or the practical application of such new or revised conclusions, theories, or laws. (Webster, 3d ed)ArchivesHolidays: Days commemorating events. Holidays also include vacation periods.EuropeRivers: Large natural streams of FRESH WATER formed by converging tributaries and which empty into a body of water (lake or ocean).Evidence-Based Medicine: An approach of practicing medicine with the goal to improve and evaluate patient care. It requires the judicious integration of best research evidence with the patient's values to make decisions about medical care. This method is to help physicians make proper diagnosis, devise best testing plan, choose best treatment and methods of disease prevention, as well as develop guidelines for large groups of patients with the same disease. (from JAMA 296 (9), 2006)Europe, EasternIrelandFraud: Exploitation through misrepresentation of the facts or concealment of the purposes of the exploiter.Tricholoma: A large genus of gilled mushrooms in the family Tricholomataceae. They are most often found in mountain and northern forests.Prisons: Penal institutions, or places of confinement for war prisoners.Heteroptera: A suborder of HEMIPTERA, called true bugs, characterized by the possession of two pairs of wings. It includes the medically important families CIMICIDAE and REDUVIIDAE. (From Dorland, 28th ed)PrisonersNew YorkScientific Misconduct: Intentional falsification of scientific data by presentation of fraudulent or incomplete or uncorroborated findings as scientific fact.

Liposomal daunorubicin (Daunoxome) and polyethylated glycol conjugated asparaginase (PEG-ASPA) in children with relapsed and refractory acute lymphoblastic leukemia treated on compassionate basis. (1/35)

BACKGROUND: Daunoxome (DNX) is an encapsulated form of daunorubicin in liposomal vesicles with suggested better pharmacokinetics, pharmacodynamics and lesser cardio toxicity than the free form. Polyethyl glycol asparaginase (PEG-ASPA), a modified form of L-asparaginase, has better activity and lesser immunogenicity than its native molecule. AIM: To evaluate on a compassionate basis, the combination of Daunoxome and PEG-ASPA, as regard to efficacy and toxicity, as a salvage treatment in refractory/relapsed childhood ALL. METHODS: The combination of these 2 drugs were used in 9 multiple relapsed or refractory ALL children on the basis of: DNX weekly 100 mg/m2 D1, 8, 15. PEG-ASPA single, 2500IU/m2 dose D15. Vinca alkaloids and corticosteroids were associated. RESULTS: Median hospital stay was 4 days (0-55). COMPLICATIONS: Neutropenia grade IV n=4, grade III infection n=6, grade II cardiac toxicity n=1, grade III allergy, grade III hemostasis disorders, thrombosis, n=1 respectively. All patients achieved complete remission (CR) except one who died from disease progression. 8 patients were subjected to hematopoietic stem cell transplantatation (HSCT). Two patients of them are alive and well, 4 died from transplant related causes and 2 from disease progression. CONCLUSION: Salvage treatment containing DNX and PEG-ASPA, of small sample childhood ALL with very advanced disease, is feasible as regard toxicity and response rate allowing to HSCT and possible cure.  (+info)

Safety and efficacy results of the advanced renal cell carcinoma sorafenib expanded access program in North America. (2/35)

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Single-institution experience with ipilimumab in advanced melanoma patients in the compassionate use setting: lymphocyte count after 2 doses correlates with survival. (3/35)

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Use of intravenous zanamivir after development of oseltamivir resistance in a critically Ill immunosuppressed child infected with 2009 pandemic influenza A (H1N1) virus. (4/35)

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Temsirolimus in VEGF-refractory metastatic renal cell carcinoma. (5/35)

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Hematopoietic stem cell mobilization with the reversible CXCR4 receptor inhibitor plerixafor (AMD3100)-Polish compassionate use experience. (6/35)

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Compassionate use of interventions: results of a European Clinical Research Infrastructures Network (ECRIN) survey of ten European countries. (7/35)

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Misuse of the FDA's humanitarian device exemption in deep brain stimulation for obsessive-compulsive disorder. (8/35)

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TY - JOUR. T1 - Safety and efficacy results of the advanced renal cell carcinoma sorafenib expanded access program in North America. AU - Stadler, Walter M.. AU - Figlin, Robert A.. AU - McDermott, David F.. AU - Dutcher, Janice P.. AU - Knox, Jennifer J.. AU - Miller, Wilson H.. AU - Hainsworth, John D.. AU - Henderson, Charles A.. AU - George, Jeffrey R.. AU - Hajdenberg, Julio. AU - Kindwall-Keller, Tamila L.. AU - Ernstoff, Marc S.. AU - Drabkin, Harry A.. AU - Curti, Brendan D.. AU - Chu, Luis. AU - Ryan, Christopher W.. AU - Hotte, Sebastien J.. AU - Xia, Chenghua. AU - Cupit, Lisa. AU - Bukowski, Ronald M.. PY - 2010/3/1. Y1 - 2010/3/1. N2 - BACKGROUND: The Advanced Renal Cell Carcinoma Sorafenib (ARCCS) program made sorafenib available to patients with advanced renal cell carcinoma (RCC) before regulatory approval. METHODS: In this nonrandomized, open-label expanded access program, 2504 patients from the United States and Canada were treated with oral sorafenib 400 mg twice daily. Safety ...
Compassionate use is a treatment option that allows the use of an unauthorised medicine. Compassionate use programmes are for patients who have a disease with no satisfactory authorised therapies or cannot enter a clinical trial. They are intended to facilitate the availability to patients of new treatment options under development"- EMA site. "Global access programmes, known by various names including compassionate use, named patient, expanded access and early access, provide a mechanism by which patients with rare diseases are able to access medicines that would otherwise be unavailable.. For rare and orphan diseases, these programmes can facilitate access to:. -medicines that are in clinical development,. -drugs unlicensed in a specific market but licensed elsewhere,. -drugs under assessment in the marketing authorisation application (MAA) process.. Importantly, through a global access programme, patients can access an orphan drug that may never be commercially available in their home country ...
This is a compassionate use study. In addition to providing compassionate use access to mifepristone, objectives of the study will be to evaluate the safety and utility of mifepristone in the treatment of the signs and symptoms of endogenous Cushings syndrome when given on a compassionate use basis. The study will only enroll subjects whose physicians have determined that medical treatment is needed to control the symptoms or signs of hypercortisolemia ...
PARIS, July 11, 2016-- AB Science SA, a pharmaceutical company specialized in the research, development and marketing of protein kinase inhibitors, announces that the FDA granted a Single Patient IND for Compassionate Use of masitinib for patient with amyotrophic lateral sclerosis. FDA received the first request for single compassionate use of masitinib in...
104317: The market authorisation application for mepolizumab for the indication of hypereosinophilic syndrome (HES) was filed in 2008, but later the file was withdrawn due to outstanding questions from regulators raised from the application. On the basis of sponsors evaluation, participants with life-threatening HES who have documented failure (lack of efficacy or a contra-indication) to at least 3 standard HES therapies (compassionate use) and participants who have participated in a previous GSK sponsored study in HES (long-term access) can be consider for mepolizumab treatment where the country regulation permits. In this study, participants will receive mepolizumab in an open-labelled manner, and limited data will be collected to evaluate the long-term safety and efficacy of mepolizumab.. 201956: This is a Long-term Access Programme (LAP) which aims to support provision of mepolizumab, until it is commercially available, to eligible subjects with severe asthma who participated in a ...
Bexsero (Novartis, New York, New York), the only vaccine in production that protects against MenB, was approved for use in Europe and Australia in January 2013 (1). Its distribution in response to an outbreak marks the first time the "compassionate use" or "expanded access" mechanism for drug approval (2, 3) has been granted to distribute a nontherapeutic biological drug to a group of healthy persons. This unprecedented evolution of events at Princeton has resulted in a flurry of media coverage falling into 3 main categories: support for a cooperative response between public health institutions and the university, concern that students are being used as "guinea pigs" for an untested product, and questions of fairness when many other groups are at risk for disease from MenB ...
Hey all, This is my first time posting so if I am doing something wrong or posting in the wrong place please let me know. I have been following the work of Dr. Wise Young since I got hurt about 3 years ago. I was initially diagnosed as C4-C5 Asia C. I am able to ambulate with a regular walker for very short distances and can do about 30 minutes on a treadmill at a snails pace :) My question is, if and when the trial gets approval for compassionate use, will it be open to incomplete
In addition to being an example of unsubstantiated hype about regenerative medicine, the controversy around the Italy‐based Stamina Foundations unproven stem cell therapy represents another chapter in a continuing debate about how to balance patients requests for early access to experimental medicines with requirements for demonstrating safety and effectiveness. Compassionate use of the Stamina therapy arguably should not have been permitted under Italys laws, but public pressure was intense and judges ultimately granted access. One lesson from these events is that expert regulatory agencies may be the institutions most competent to make compassionate use decisions and that policies should include more specific criteria for authorizing compassionate use. But even where regulatory agencies make decisions based on clear rules, difficult questions will arise. ...
This webinar will cover access to innovative new therapies through expanded access programs with guest speakers Jennifer Miller, PhD, Assistant Professor at Yale University School of Medicine and Alison Bateman-House, PhD, MPH, Assistant Professor at NYU School of Medicine. The webinar will look in detail at the process involved in obtaining an investigational therapy before it gains final FDA approval. Open to anyone looking to learn more about expanded access programs. Register here: http://bit.ly/2I8UvK8
SAN DIEGO, Dec. 3, 2012 /PRNewswire/ -- Aethlon Medical Announces Initiation of Compassionate-Use Program to Treat Hepatitis C Virus (HCV).
... Glaxo Wellcome announced today (9/21) that the amprenavir expanded access program is starting for both adults and children (,4 yrs). You or your doctor can call-. 1-800-248-9757 immediately to receive information about enrollment. You or your doctor can call to give the company your doctors name and address. On Sept 28 they will send out expanded access program binders explaining the program. As soon as a person is enrolled in the program drug will be sent out. The company is submitting their NDA to the FDA for accelerated approval in October. Review and approval can take usually from 3-6 months, meaning approval will not come sooner than January 1999.. The program is uniquely offering 3 options for accessing amprenair-. 1. Individuals experiencing adverse side effects from their current PI regimen such as lipodystrophy or elevated lipids but are not failing their current PI regimen can enroll in this option. The purpose is to try and collect ...
Compassionate use is a treatment option that allows the use of an unauthorised medicine. Compassionate use programmes are for patients who have a disease with no satisfactory authorised therapies or cannot enter a clinical trial. They are intended to facilitate … Continue reading →. ...
Therapies that address the underlying cause of PAH are urgently needed. The U.S. Food and Drug Administration (FDA) approved tacrolimus in 1994 for use in lowering the risk of organ rejection in patients undergoing kidney transplant, and it is currently indicated for use in additional organ transplant settings and to treat atopic dermatitis.. Researchers completed a randomized, double-blind Phase 2a clinical trial with 23 class 1 and 2 PAH patients titrated to target blood levels. All target blood levels were well tolerated with no drug related serious adverse events, nephrotoxicity or incident diabetes.. In addition, tacrolimus was provided for compassionate use in three class 3 or 4 PAH patients. The compassionate use demonstrated dramatically reduced rates of hospitalizations and functional class improvements were observed.. We are currently focused on a proprietary formulation of tacrolimus for clinical development program and for commercial use. We are on track to file an Investigational ...
March 13, 2014 - 3:16 pm , By Jennifer Boggs , No comments yet Before its unfortunate drop in quality in its last few seasons, the medical drama House was one of my favorite shows, with its title character, a cranky, possibly drug-addicted doctor solving diagnostic riddles and saving lives with Sherlockian brilliance, and quite often breaking every rule in the book to do it. One episode in particular had House facing off with billionaire investor Vogel, who had thwarted his attempt to enroll a very sick patient in a clinical trial without mentioning to the trial investigator that she wouldnt fit the criteria, namely having just undergone a C-section. They have an impassioned exchange: Dr.: House: Hey! Youre killing her! Vogler: Really? See, I thought you were the one trying to ram her into a drug trial five minutes after surgery. Dr. House: She knew the risks! She was fully informed! Vogler: Yeah, well the guy running the study sure wasnt. Dr. House: Not his life, not his call! Vogler: His ...
The purposes of this study are to make Omegaven® available to cancer patients with liver disease and to determine if Omegaven® can improve or prevent
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We are devoted to providing patients with the highest quality of organically grown medical marijuana. 1-800 medical marijuana treats each and every patient with the courtesy, respect and understanding they so richly deserve.
DI-fusion, le Dépôt institutionnel numérique de lULB, est loutil de référencementde la production scientifique de lULB.Linterface de recherche DI-fusion permet de consulter les publications des chercheurs de lULB et les thèses qui y ont été défendues.
Expanded access, sometimes called compassionate use, is the use of an investigational medical product outside of a clinical trial (i.e., one that has not yet been approved). At Novelion Therapeutics, and our subsidiary Aegerion Pharmaceuticals, we are committed to increasing awareness of and knowledge about our expanded access programs and the procedures for obtaining access to human investigational drugs. A Message from John Orloff, M.D.
Approval for the lixelle® ß2-microglobulin apheresis column (also called the beta2-microglobulin apheresis column or ß2m). This device is indicated for the treatment of patients with clinically diagnosed dialysis-related amyloidosis (dra ...
London, United Kingdom, Friday 11 April 2014: New research announced at the International Liver CongressTM 2014 today provides new hope for the notoriously difficult-to-treat population of liver transplant patients with recurring hepatitis C (HCV).[1]. As part of a compassionate use program, 104 post-liver transplant patients with recurring HCV who had exhausted all treatment options and had poor clinical prognoses, received sofosbuvir (SOF) and ribavirin (RBV) with pegylated interferon (PEG) included at the physicians discretion for up to 48 weeks. Among patients whose clinical outcomes have been reported, 62% achieved SVR12. Additionally, 62% of patients had improvements in clinical conditions associated with hepatic decompensation (e.g., ascites and encephalopathy) and/or improvement in liver function tests. SOF+RBV±PEG was well-tolerated and led to high rates of virologic suppression.. EASLs European Policy Councillor Professor Patrizia Burra of the Multivisceral Transplant Unit, Padova ...
The treatment currently involves a relatively large number of capsules, so dogs that are difficult to orally medicate wouldnt be great candidates," said Dr Frimberger. "But we are working on reformulating the drug to make it better tasting and easier to administer to dogs before the full trials commence next year.". The MPL program involves two consultations/treatments at the Animal Referral Hospital (ARH) in Homebush, NSW. Owners would have to transport their dogs to and from Homebush for the two treatments. PharmAust will cover all compassionate use program costs, including travel expenses to and from the ARH, as well as costs for the initial conventional chemotherapy treatment upon program completion.. For more information or to enrol, please contact Dr Angela Frimberger on [email protected] What is canine lymphoma? Cancer is the number one cause of death in dogs over the age of 2, with 25 per cent of deaths attributed to cancer¹. "Approximately one in four dogs and one in ...
If approved, metreleptin would be the first medication available in the EU to treat generalized and partial lipodystrophies More than 100 patients are currently treated with metreleptin via a compassionate use program in Europe
Haas J, Linker RA, Hartung HP, Meergans M, Ortler S, Tracik F. [Fingolimod compassionate use program: case study on the concept of a therapy option for multiple sclerosis prior to marketing approval]. Nervenarzt. 2012;83(12):1575-81 ...
Ten (10) patients with uncontrolled persistent moderate to severe CD were treated with QBECO through a compassionate use program. Patients may have continued to receive conventional medical treatments and/or complementary therapies in addition to the SSI treatment. Seven of the ten patients reported full resolution of clinical symptoms with a course of QBECO treatment of three months or more. Four of these patients have had sustained clinical remission after discontinuing all medications including SSI treatment. The longest case of clinical remission reported is still ongoing, after almost 3 years. Three of the seven patients reporting full remission had follow-up colonoscopies or CT scan with confirmation of full remission. Nine of the ten patients were able to discontinue all other medications at some point while on QBECO treatment. (More information here: http://www.qucrohnstrial.com/ssi-int...cy-and-safety/ ...
Ionis Pharmaceuticals Announces Plans to Open Expanded Access Program to Provide Inotersen to Patients with Hereditary TTR Amyloidosis in the U.S. - Ionis plans to open multiple sites in the U.S.
LAVAL, QC, May 3, 2016- Prometic announces successful treatment of a plasminogen-deficient patient under an expanded access (compassionate use) protocol in the USA.
Executive Director; Medical Strategy- Boston Duties and Responsibilities: Serve as U.S. Medical Director for multiple global clinical development trials and/or U.S. Medical Affairs Trials (including phase I-IV, Expanded Access, Investigator Initiated, registries and Compassionate Use). Interface with Global and U.S. … Read More. ...
The Department of Surgery Clinical Research Program provides faculty and staff with comprehensive support to conduct clinical research studies at the University of Wisconsin.. The Department of Surgery Clinical Research Program is committed to conducting the highest quality clinical research and offering innovative new therapies to our patients, aiming to improve short and long-term patient outcomes. The dedicated clinical research staff has extensive experience in all areas of pharmaceutical and device research including Phase 1 through Phase 4 drug development, compassionate use protocols, investigator held IND studies, bioequivalence and pharmacokinetic studies, and IDE device studies.. ...
Liverpool and London, UK - November 3 2015: The INSPIRE consortium, led by Videregen Ltd, has received approval from the MHRA* to start a UK clinical trial with its tissue engineered replacement trachea. The trial, which is expected to start in the first half of 2016, will focus on severe structural airway disease (SSAD), a potentially life-threatening condition believed to affect 19,000 people in Europe and the US. Along with Videregen as commercial partner, the consortium includes the Cell Therapy Catapult, University College London (UCL), NHS Blood and Transplant in Speke and the Royal Free London NHS Foundation Trust.. The Phase I trial, which will recruit four patients with SSAD, is to assess the safety and initial efficacy of the product. While replacement trachea technologies have been tested previously in compassionate use cases, this is the first formal clinical trial of a tissue engineered autologous stem cell trachea replacement. The INSPIRE project started in 2014, and has £1.9m of ...
Thats a very valid surprise point actually. The FDA emergency approved basically for or based upon four reasons/principals in consideration, parts of the same basic principals they consider in the thought process for all emergency approvals under FDA compassionate use rules to save a life. The first principal being safety, a bio-absorbable plastic was used which has already been in use within the medical area and known to be safe, and the procedure can be accomplished safely. The second principal being commonality to existing medical process/procedure, the application/methodology/theory use of such devices/methods (not necessarily this particular device) is not that uncommon, for example, stints used for heart arteries (in the opening artery up aspect and known to be safely beneficial to health and life), and tracheal implants have already been approved (the first one done in the U.S. basically used the patients own stem cells to produce tissue over a plastic scaffold and others have been ...
America has a major cancer problem America, Please help cure this young lady Mikaela, whos developed kidney cancer! Give My Wife a Chance against Cancer: Please Grant Compassionate Use of MK-3475 for Mikaela Right Away https://www.change.org/petitions/merck-please-grant-compassionate-use-of-mk-3475-for-mikaela-right-away#shareUnfortunately, due to my own life altering experience from a medical device, I do not agree with non-clinically, or clinically tested drugs that…
I agree to provide the physician with any and all copies of my MEDICAL RECORDS, if they exist, that document my medical conditions, as requested by the physician. I agree to obtain medical FOLLOW-UP at my personal medical doctors office or obtain a personal doctor if I have none now and to return for FOLLOW-UP, as recommended by the physician. I understand this is an obligation to MY part for the continuity of care. I understand that I must be a California State resident to obtain an approval or recommendation for the use of cannabis (i.e., Medical Marijuana) under Californias Compassionate Use Act of 1996 (Health & Safety Code #11362.5). I have found or am interested in determining whether cannabis (i.e., medical marijuana) provides substantial relief and improvement of my condition. I have been assured that medical records relating to my care will be kept private and confidential and that no information will be printed, which would disclose my personal identity unless required by law. ...
I agree to provide the physician with any and all copies of my MEDICAL RECORDS, if they exist, that document my medical conditions, as requested by the physician. I agree to obtain medical FOLLOW-UP at my personal medical doctors office or obtain a personal doctor if I have none now and to return for FOLLOW-UP, as recommended by the physician. I understand this is an obligation to MY part for the continuity of care. I understand that I must be a California State resident to obtain an approval or recommendation for the use of cannabis (i.e., Medical Marijuana) under Californias Compassionate Use Act of 1996 (Health & Safety Code #11362.5). I have found or am interested in determining whether cannabis (i.e., medical marijuana) provides substantial relief and improvement of my condition. I have been assured that medical records relating to my care will be kept private and confidential and that no information will be printed, which would disclose my personal identity unless required by law. ...
There has been a lot of recent controversy surrounding the experimental treatment in clinical trials known as GM604. A lot of misinformation has been tossed around on both sides of the issue. I want to beg your attention for a little while to explain whats really going on. First, as many of you know, I was the single late-stage PALS who received GM6 in compassionate use. The intent behind this was to get a look at differences in biomarker candidate levels between earlier-stage and late-stage PALS. Any outward physical manifestation of improved condition noticed would be a bonus and, due to my advanced condition, no improvement in condition was expected. Nevertheless, Genervon and I came up with the idea to try to chart improvements in the tongue. The rationale is that since my tongue is only moderately affected, and because the hypoglossal nerve contains one of the shortest motor neurons in the body, any possible improvement would be noticed there first.. Because of my growing cooperative ...
Remdesivir (GS-5734) is an adenosine analog antiviral drug that inhibits viral RNA polymerase and has demonstrated in vitro activity against various viruses including Ebola, SARS-CoV, and Middle Eastern respiratory syndrome (MERS-CoV). More recently, remdesivir has demonstrated potent activity against SARS-CoV-2 in in vitro and animal model studies, and holds some promise for treatment of COVID-19.2,3 A recent case series describing the compassionate use of remdesivir in 61 adult hospitalized patients with COVID-19 demonstrated that 68% of patients experienced an improvement in the need for oxygen support over a median 18 day follow-up period, while 15% of patients clinically worsened. Clinical improvement was observed in 84% of patients, but was less frequent among older patients (70 or older vs less than 50), and in patients who were on invasive ventilation compared with patients on noninvasive ventilator support. Mortality occurred in 13% of patients, with older patients (70 or older) and ...
The condition Charlie suffers from, "infantile onset encephalomyopathy DNA mitochondrial depletion syndrome," is incurable and ultimately fatal. An experimental therapy is available in the US, but has not as yet been demonstrated in randomised, controlled trials to be safe and effective, and thus remains outside the canon of approved treatments; Columbia University professors had agreed to allow compassionate use for little Charlie, and a "Go Fund Me" charity was set up that would have more than paid for the expense of bringing the child and his parents to New York, but in the UK, doctors can block treatments, even if the parents wish that their child receive them, if they decide that the treatments are not in "the best interests" of the child. ...
The condition Charlie suffers from, "infantile onset encephalomyopathy DNA mitochondrial depletion syndrome," is incurable and ultimately fatal. An experimental therapy is available in the US, but has not as yet been demonstrated in randomised, controlled trials to be safe and effective, and thus remains outside the canon of approved treatments; Columbia University professors had agreed to allow compassionate use for little Charlie, and a "Go Fund Me" charity was set up that would have more than paid for the expense of bringing the child and his parents to New York, but in the UK, doctors can block treatments, even if the parents wish that their child receive them, if they decide that the treatments are not in "the best interests" of the child. ...
Existing law, the Compassionate Use Act of 1996, an initiative measure enacted by the approval of Proposition 215 at the November 6, 1996, statewide general election, authorizes the use of cannabis for medical purposes and provides certain protections to a physician and surgeon who recommends the use of medical cannabis to a patient. The Control, Regulate and Tax Adult Use of Marijuana Act of 2016 (AUMA), an initiative measure approved as Proposition 64 at the November 8, 2016, statewide general election, authorizes a person who obtains a state license under AUMA to engage in commercial adult-use cannabis activity pursuant to that license and applicable local ordinances. The Medicinal and Adult-Use Cannabis Regulation and Safety Act (MAUCRSA), among other things, provides for the licensure and regulation of commercial medicinal and adult-use cannabis activities and states that the purpose and intent of MAUCRSA is to establish a comprehensive system to control and regulate the cultivation, ...
http://www.hhs.gov/about/ So exactly when does your agency/bureaucracy actually do something helpful and effective "for protecting the health of all Americans and providing essential human services, especially for those who are least able to help themselves"? It has been two (2) years now that Ive been on this project advocating and writing to former Secretary Kathleen Sebelius and you. My original pleas were for "Compassionate Use" only to learn all along your CMS agency incorporates the doctrine of "Continuity of care" which rightfully should have insured medical coverage for this unique population of patients implanted with a medical device being denied care and/or replacement by your various contractors. The decision of December 30, 2014 reiterates this fact of "Continuity of care". What does it take to get any of you people to understand that it is rightfully your responsibility to care for these patients (read youre agencies statement at the beginning of this communication) and you folks ...
1Note.-. A. Section 1, ch. 2017-232, provides that "[i]t is the intent of the Legislature to implement s. 29, Article X of the State Constitution by creating a unified regulatory structure. If s. 29, Article X of the State Constitution is amended or a constitutional amendment related to cannabis or marijuana is adopted, this act shall expire 6 months after the effective date of such amendment." If such amendment or adoption takes place, s. 381.986, as amended by s. 1, ch. 2017-232, will read:. 381.986 Compassionate use of low-THC and medical cannabis.-. (1) DEFINITIONS.-As used in this section, the term:. (a) "Cannabis delivery device" means an object used, intended for use, or designed for use in preparing, storing, ingesting, inhaling, or otherwise introducing low-THC cannabis or medical cannabis into the human body.. (b) "Dispensing organization" means an organization approved by the department to cultivate, process, transport, and dispense low-THC cannabis or medical cannabis pursuant to ...
Texas Title 6. Food, Drugs, Alcohol, And Hazardous Substances Title 3. Health Professions - Subtitle J. Pharmacy and Pharmacists - Chapter 551. General Provisions Title 3. Health Professions - Subtitle B. Physicians - Chapter 169. Authority to Prescribe Low-thc Cannabis to Certain Patients for Compassionate Use
... Medical Marijuana Outlets On Hold Posted by CN Staff on April 14, 2005 at 09:36:07 PT By Steve Irsay, Staff Writer Source: Grunion Gazette California -- When California voters approved Proposition 215, better known as the Compassionate Use Act of 1996, they decriminalized the personal possession of marijuana for documented medical uses. What they did not do was lay out specific guidelines for getting the drug into the hands of patients. Last year s follow-up statute, SB 420, outlined identification card systems and minimum legal amounts to possess but, again, distribution instructions remained vague. The matter is further complicated by federal law, which prohibits the possession, cultivation and distribution of marijuana. The U.S. Supreme Court is expected to rule shortly on the case of two California medical marijuana users challenging the ban.In this absence of clear retail sales options, many patients have resorted to illegal street ...
In Poland, at The Hirszfeld Institute and of Immunology and Experimental Therapy, phage therapy is carried out under medical experimentation, ethical and compassionate use regulatory provisions similar to those that exist in most countries. A broad range of infections have been treated since the initial anti-staphylococcal treatment in 1925. The Institute requires that all patients treated must have previously been treated - unsuccessfully - with conventional methods such as antibiotics. Since the 1980s, their work with phages has been published in English language scientific journals. Very high success rates - 85% on average - have been obtained for infections caused by bacteria such as Escherichia, Klebsiella, Proteus, Enterobacter, Pseudomonas, and Staphylococcus aureus. Their success rates for treating Pseudomonas aeruginosa and Staphylococcus aureus, including MRSA strains, have been reported to be even higher than 85%.. ...
Does anybody know what the recommmended drug for gastro is currently , (in the US)?? , I know when I had it years ago I took propulsid but it is now on , special case only usage. , One (or more) of my kids has a mitochondrial metabolic disorder which , causes weakness and all kinds of strange side effects, in this case , the equivelence of gastro. , The doctor recommended reglan but this seems to put the poor kid to , sleep (and as soon as she wakes up she has to take the next one). There is no replacement drug. Propulsid is available from Janssen for compassionate use. Erythromycin has the same action. Reglan is not as effective and is a depessant drug. Perhaps you can get Propulcid in Mexico or Europe. I think it has been removed from the Canadian Health System Formulary. spot A. L. Bender, M. D. email @ redacted ---------------------------------------------------------- for HELP or to subscribe/unsubscribe, contact: [email protected] send a DONATION ...
Community-based organization that promotes guidelines for medical marijuana patients to be presumed in compliance with California HS 11362.5, known as the voter-approved Prop 215, the Compassionate Use Act of 1996. ...
Applications for annual licenses will be accepted through an online licensing system that will launch in December 2017. This application will require information on the business, owners and financial interest holders, and operating premises, as well as descriptions of procedures for waste disposal, inventory and quality control, transportation and security. Businesses in operation under the Compassionate Use Act prior to September 1, 2016, will receive priority application review. Applicants must be in compliance with city or county ordinances. During the application review process, CDPH will contact the city or county to verify the local authorization. If the applicant does not provide a copy of their local authorization, the local office will have 60 days to respond. If the applicant includes a copy of their local authorization with their application, the local office will have 10 days to respond. ...
New Jersey added a new herb to its state garden last Tuesday when Gov. Jon S. Corzine signed the New Jersey Compassionate Use Medical Marijuana Act on his last day in office. But though 14 states...
The Times of Trenton Op-Ed. Wednesday, November 24, 2010. SPECIAL TO THE TIMES. The proposal by the state Board of Medical Examiners (BME) to make New Jersey doctors try to wean their patients off of medical marijuana every three months is simply another example of how the crafting of medical marijuana regulations is being driven by fear, ignorance and hostility ("Christie: Tighten rules on medical marijuana," Nov. 16).. The New Jersey Compassionate Use Medical Marijuana Act is already the most restrictive of all such laws in the nation. The qualifying conditions are severely limited and include any patient with a diagnosis of less than 12 months to live. Now the BME is insisting that doctors periodically stop a medication that brings relief to a dying patient "in an effort to reduce the potential for abuse or dependence." It is absurd and cruel. Drug dependence is simply not an issue for a patient with only months to live.. Another qualifying condition is seizures, including epilepsy. After all ...
If you suffer from a debilitating illness take a look at some of the videos and links and feel free to contribute to this discussion by posting in the comments section below. http://www.cannabisinternational.org/ The U.S. Compassionate Use Act and Multiple Sclerosis are two main factors that we think of when we hear so-called experts discussing…
InVivo Therapeutics (OTC:NVIV) announced a push-back on its time-lines for enrollment in a clinical trial examining its spinal cord injury treatment, a move that brought NVIV shares down by nearly half today. InVivo announced that, under the terms of its FDA investigational device exemption, each trial patient must be followed for 3 months and the company must win agency approval before enrolling the next, meaning it will take InVivo an estimated 21 months to enroll its 5 patients.. Then, the company plans to launch a pivotal study in order to apply for FDA approval to begin selling its biopolymer scaffolding under a humanitarian device exemption. The delays could push back commercialization for a few years, according to analysts at Seeking Alpha. We remain fully committed to beginning this study as soon as possible, interim CEO Michael Astrue said in prepared remarks. While the study will take additional time, we look forward to bringing this important therapy into the clinic. The ...
Stryker has secured approval from the US Food and Drug Administration for Neuroform Atlas Stent System for marketing under a humanitarian device exemption (HDE).. The device is to be utilized in conjunction with neurovascular embolic coils for the treatment of wide neck, intracranial, saccular aneurysms and allows the company to begin U.S. commercialization efforts immediately. "The hybrid cell stent design of Neuroform Atlas is designed to improve wall apposition, ease of use, deployment accuracy, and catheter re-entry in even the most challenging cases," said Dr. Osama O. Zaidat, Director of the Neuroscience and Stroke Center at Mercy Hospital in Toledo, Ohio, and Co-Principal Investigator of the U.S. Neuroform Atlas investigational trial.. "The Atlas design may improve patient care by facilitating the treatment of wide neck aneurysms in tortuous and more complex anatomies.". "The ability to navigate distal anatomy within the brain using the lowest profile delivery on the U.S. market is a ...
Patient Care and General Interest. The British Heart Foundation reports that the number of premature deaths related to cardiovascular disease has risen in the UK for the first time in 50 years.. A federal judge in the US ruled against the Food and Drug Administrations (FDA) 2017 decision to allow e-cigarettes to remain on the commercial market prior to obtaining FDA approval, a move that was applauded by the associations that brought the suit.. Drugs and Devices. The US FDA has expanded the approved duration of support for 2 Impella heart pumps from 6 to 14 days.. Xvivo Perfusion, Inc, was granted premarket approval by the US FDA for its ex vivo lung perfusion system, a move that expands the devices previous humanitarian device exemption authorization.. The US FDA has approved dalteparin sodium for treating venous thromboembolism in children as young as 1 month old.. Research, Trials, and Funding. The antiplatelet agent ticagrelor showed antibacterial activity against Staphylococcus and ...
January 30, 2018-Stryker announced that results from WEAVE, the postmarket surveillance study of the companys Wingspan stent system, were presented at the ISC 2018: the International Stroke Conference held January 24-26 in Los Angeles, California.. According to the company, the findings provide compelling evidence that more patients with intracranial atherosclerotic disease (ICAD) may benefit from endovascular treatment with the Wingspan stent system.. Wingspan is a self-expanding stent and delivery system intended for use in the treatment of ICAD. It includes the over-the-wire Gateway percutaneous transluminal angioplasty balloon catheter that predilates the lesion before insertion and deployment of the Wingspan stent.. The WEAVE study was mandated by the US Food and Drug Administration, which approved the Wingspan stent system under a humanitarian device exemption in 2005. Wingspan also received European CE Mark approval in 2005.. The company-sponsored, multicenter, prospective WEAVE study is ...
Medimmune is the Specialty Division of AstraZeneca, a large bio-pharmaceutical company, and has products in the flu and RSV disease state. The flu product LAIV and palivizumab (Synagis) is the rsv product.. Medtronic. Medtronic deep brain stimulation (DBS) safely and effectively manages some of the most disabling symptoms of essential tremor and Parkinsons disease. Medtronic deep brain stimulation is also approved under a Humanitarian Device Exemption (HDE) for dystonia. Merces Consulting. Merces Consulting Group, Inc., formerly Management Resource Center, Inc., is an independent consulting firm that provides advice and counsel to management of all types of organizations in the development of strategies, programs, and practices that serve to effectively utilize their most important assets- their human resources. Merces has clients nationwide in virtually every industry. The firm began working with its first Health Center client in Michigan in 1993, and established a formal practice dedicated to ...
The indictment alleged that the defendants were part of a scheme to promote the combined use of a pair of separate bone-healing products, each granted a narrow, provisional "humanitarian device exemption" by the FDA. Combining the treatments and devices - the OP-1 Implant, OP-1 Putty and the bone void filler Calstrux - caused adverse effects in patients ranging from minor irritations to infections requiring follow-up surgeries. The indictment also charges that Stryker and Philip lied to the FDA about the number of patients treated each year with OP-1 Putty.. The case went before a jury last week. Heppner and Whitaker have asked OToole to declare a mistrial with prejudice, according to the documents. The judge has not yet ruled on those motions, but granted Philips move to sever the charges against him. That means Philip will be able to use communications he had with Stryker Biotech lawyers, which would otherwise fall under attorney-client privilege, in his defense.. ...
The FDA has approved an IDE for InVivos biopolymer scaffold, making it the first such treatment to dip its toe in the America patient population. The new study is expected to deliver a Humanitarian Device Exemption, giving it access to market sooner than a PMA.
Data were collected from 23 advanced melanoma patients (15 cutaneous, 6 mucosal and 2 of unknown primary). 20 patients (61%) received at least 2 lines of treatment previously, and 6 patients (26%) with stable brain metastases were enrolled. Median overall survival (OS) was 6.7 months (CI 95% 4.6-8.8). 12-month OS was 38% and 15-month OS was 30%. Among 20 evaluable patients, 4 patients achieved a partial response, and 8 patients achieved a stable disease, with an overall response rate of 20.0% (CI 95% 0-39%) and clinical benefit rate of 60% (CI 95% 36-84%). The most common drug-related adverse events of any grade were rash (35%), pruritus (30%), and fatigue (30%). Two deaths were considered treatment-related; one cytokine storm and one ventricular tachycardia. ...
This was a multicenter, open-label, single-arm phase 3B study of the combination lenalidomide plus pulse high-dose dexamethasone.This study (CC-
Urotoday an online resource providing current urology conditions information including urologic oncology | female urology | CAUTI prevention | mens health | pediatric urology
Merck is launching an expanded access program for their anti-PD1 drug lambrolizumab. Clinical trials have shown very positive results for this drug, with response rates significantly higher than any drug currently approved for melanoma. Here are the key points: · The program will initially be available at sites in the United States that have been involved in the Merck PD-1 trials. Fairly quickly it will be made available to other physicians in the US who have experience treating melanoma with immunotherapy. After that, the expansion will extend globally.
Merck is launching an expanded access program for their anti-PD1 drug lambrolizumab. Clinical trials have shown very positive results for this drug, with response rates significantly higher than any drug currently approved for melanoma. Here are the key points: · The program will initially be available at sites in the United States that have been involved in the Merck PD-1 trials. Fairly quickly it will be made available to other physicians in the US who have experience treating melanoma with immunotherapy. After that, the expansion will extend globally.
Roche announced on Tuesday that the Global Access Program is expanding beyond HIV, to include Mycobacterium tuberculosis (MTB), Hepatitis B and C (HBV and HCV), and Human Papillomavirus (HPV) for low- and middle-income country programs where the disease burden is the highest.. The expansion of the Global Access Program highlights Roches commitment to improve access to cost-effective resources, implement scale-up programs, and contribute to the elimination of diseases in the regions with the greatest need.. "With effective treatment options for these infectious agents and improved patients access to diagnostics, early detection can help save lives and ease suffering," said Michael Heuer, CEO Roche Diagnostics. "As the leader in infectious disease diagnosis testing, Roche is dedicated to supporting goals on eradicating diseases globally.. "Access to cutting-edge, best in class diagnostic test results means more patients being appropriately diagnosed and well treated, resulting in lives saved," ...
The clinical research interests of Dr. Bedimo include strategies for the optimal management of Drug-Resistant HIV Patients, analysis of metabolic Abnormalities in HIV infected Patients and effect of HCV co-infection. Presently, the latest cutting-edge antiviral therapies are available through expanded access programs, and the interested patients are offered the opportunity to join in national multicenter clinical clinical trials and novel pilot studies being performed locally. Research is an important part of HIV care, both to learn more about how to treat this infection, and to bring the latest advances to our patients. ...
In this study, we showed a high EGFR mutation rate in our NSCLC patient population, with mutation patterns very similar to those reported previously (13, 14). We documented that EGFR mutations played a crucial role in determining gefitinib response in NSCLC patients, confirming findings from two previous landmark studies of ,20 gefitinib-treated patients (13, 14). For the first time, we have also shown that mutations in EGFR could confer longer survival in gefitinib-treated NSCLC patients. Furthermore, of those patients achieving partial response, patients with mutations in EGFR had a trend of longer response durations in comparison with patients lacking EGFR mutations.. Gefitinib has shown antitumor activity in pretreated NSCLC patients with response rates of 10% to 20% (10, 11). From these two phase II clinical studies (10, 11), gefitinib seemed to be more effective in females and patients with adenocarcinoma and a good performance status. In a subsequent Expanded Access Program study, ...
Six patients followed at the HIV clinic at Veterans Affairs Connecticut Healthcare System had been on RAL + unboosted ATV+N(t)RTI for >24 weeks at the time of clinical review. All participants received RAL 400 mg BID + ATV 300 mg BID as part of their regimens, which had been started between April 2007 and May 2008. Individuals initiated on RAL before the drug became licensed and available for use in December 2008, had obtained the medication via an expanded access program. Median age was 58 (range 50 to 84) years, median time from diagnosis was 14.5 (range 9 to 21) years, and the median time on ART was 10.5 (range 9 to 21) years. Before initiation of RAL + ATV-based therapy, the mean HIV viral load was 115 752 copies/mL, and the mean CD4 count was 309 cells/mm3 (range 32 to 477). After 24 weeks of treatment, the viral load for all participants had become and remained undetectable, while the mean CD4 count was 543 cells/mm3 (range 101 to 1001), with an average gain of 234 CD4 cells/mm3 (Figures ...
This trial was undertaken based on the preclinical studies which indicated that basal-like breast cancer cells were highly sensitive to anti-DR5 (22-25), that the combination of an anti-DR5 monoclonal antibody and chemotherapy was quite effective in murine models of basal-type breast cancer in vivo (24) and that basal-type breast tumor stem cells were killed by anti-DR5 (25). Similar studies in hormone-dependent and HER2-positive breast cancer demonstrated resistance to anti-DR5 therapy (22-25).. At the time of this protocol design, it was not feasible to use platinum compounds as the chemotherapy backbone of our study in view of the expanded access program that was available then for the combination of carboplatin, gemcitabine, and iniparib for patients with newly diagnosed metastatic TNBC following a promising phase II randomized trial of chemotherapy with/without iniparib (41). In addition, there was no prior prospective experience with nab-PAC in this patient population although heavily ...
Gilead Sciences to Merge with NeXstar Pharmaceuticals - On March 1, Gilead Sciences and NeXstar Pharmaceuticals announced a definitive agreement whereby Gilead will acquire NeXstar in an all-stock, tax-free transaction accounted for as a pooling of interests, creating a biopharmaceutical company with worldwide presence focused on infectious disease and oncology. The transaction is subject to review by the Securities and Exchange Commission and approval by the stockholders of both companies, and is expected to be completed by mid-summer. PREVEON® Safety Data Profiled at 6th Conference on Retroviruses and Opportunistic Infections - Data from late stage clinical trials of adefovir dipivoxil, further defining its therapeutic role for the treatment of patients with HIV infection, were presented at the February conference in Chicago. Highlights included a presentation of safety data from more than 5,600 patients in the Expanded Access Program. In patients treated with adefovir dipivoxil, serious ...
In her comments to the FDA after the agency proposed a rule in March 2007, Karen Ignagni, president and CEO of Americas Health Insurance Plans (AHIP), pressed the agency to include a definition in the final rule that said, "A serious disease or condition is one which is persistent, substantially disabling, progressive, and likely to result in death within 6 to 12 months." She noted that schizophrenia and chronic depression are among the conditions that "cause disabling health effects and suffering for a period of time without death occurring prematurely or in a matter of months." AHIP was concerned about exposure of its insurance company members to wide claims from policy holders for reimbursement for expensive, investigational drugs obtained through expanded access programs, which might multiply because of the FDA rule liberalization ...
Obtaining a drug or biologic under an expanded access program may be an option for some patients who are not able to enroll in clinical trials. nbc acai. ...
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding timing of results from the Phase 2 clinical trial of SER-109, plans to initiate a SER-109 Phase 3 study later this year, the value for the Phase 3 study of the Expanded Access Program and the potential of SER-109 to be the first therapy for Clostridium difficile infection to treat the underlying cause of the disease. These forward-looking statements are based on managements current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements ...
For more information on the atazanavir expanded access program, check out the Access Project website at www.atdn.org/access/index.html and click on the ...
A study of the first 20,000 adults hospitalized with severe or life-threatening COVID-19 to receive convalescent plasma found the investigational therapy safe in this diverse group of patients, according to findings from the Food and Drug Administrations Expanded Access Program for COVID-19 reported in Mayo Clinic Proceedings.
A survey was conducted to ascertain the views of present and past students and staff and administrators of Cuyahoga Community Colleges three ACCESS programs: Veterans Upward Bound, Project Search, and Special Services for Disadvantaged Students. Questionnaires sent to current program participants focused on the nature and value of program activities designed as preparation for postsecondary education; satisfaction with various aspects of the programs; and student background and characteristics. Questionnaires administered to former students requested additional information on activities since program participation and assessment of the programs influence. Administrators and staff were questioned about their understanding of the programs purpose and asked to appraise program aspects including tutor and counselor activities. They were also requested to recommend changes. Study findings, based on a low response rate of 21%, with especially low responses from current (19%) and former
The purpose of this Managed Access Program is to provide access to neratinib for the treatment of early stage HER2-positive breast cancer (extended adjuvant
Dear Doctor, Roche and Trimeris are pleased to announce the Fuzeon (enfuvirtide) Accelerated Simultaneous Access Program (Fuzeon ASAP). This program is ...
Exosome Diagnostics, Inc. announced today that it has launched its Early Access Program for its point of care protein biomarker liquid biopsy instrume
During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market. ...
A New York state lawmaker says the state should make it easier for members of the public to become vaccinated against Hepatitis A.
Lt. Gov. Brian Calley has signed bills to expand the availability of drugs that block or reverse the effects of overdosing on painkillers and other prescription drugs.
Clinical Trials Market Analysis By Phase (Phase I/II/III/IV), By Study Design (Interventional, Observational, Expanded Access), By Indication (Autoimmune, Pain management, Oncology,
Review of 6 substances nominated for inclusion on the section 503A bulk drug substances list. Updates on discussions from previous meetings, including the option for expanded access.
Amifampridine (pyridine-3,4-diamine, 3,4-diaminopyridine, 3,4-DAP) is used as a drug, predominantly in the treatment of a number of rare muscle diseases. The free base form of the drug has been used to treat congenital myasthenic syndromes and Lambert-Eaton myasthenic syndrome (LEMS) through compassionate use programs since the 1990s and was recommended as a first line treatment for LEMS in 2006, using ad hoc forms of the drug, since there was no marketed form. Around 2000 doctors at Assistance Publique - Hôpitaux de Paris created a phosphate salt form, which was developed through a series of companies ending with BioMarin Pharmaceutical which obtained European approval in 2009 under the trade name Firdapse, and which licensed the US rights to Catalyst Pharmaceuticals in 2012. As of January 2017, Catalyst and another US company, Jacobus Pharmaceuticals, which had been manufacturing the free base form and giving it away for free since the 1990s, were racing to obtain FDA approval for their ...
CULVER CITY, Calif., June 27, 2014 (GLOBE NEWSWIRE) -- The Coalition for Pulmonary Fibrosis (CPF) is alerting the Pulmonary Fibrosis (PF) community throughout the US to a new Expanded Access Program (EAP) for an investigational therapy for Idiopathic Pulmonary Fibrosis (IPF). The launch of Boehringer Ingelheims EAP for Nintedanib makes 2014 a watershed year for IPF.
In previous blogs, we have discussed stem cell therapy for non-standard indications, or what we call "compassionate use" cases. These are cases where there is limited data to show that stem cell therapy is effective however what results we do have, may look promising. Examples include kidney disease, canine back pain, as well as several other diseases/conditions for which stem cells may be beneficial. One such indication is Immune Mediated Polyarthritis, or IMPA for short. While IMPA is a form of arthritis, it is not the typical osteoarthritis that stem cells are used for regularly. Rather than being caused by a malformed joint, wear and tear or trauma, IMPA is caused by the patients own immune system. It is important to note that polyarthritis can be caused by an infection in the patients body. Distinguishing between an infection and IMPA is imperative because treatment options are very different. In this blog, we will discuss stem cell therapy for the treatment of IMPA.. In patients with ...
The Ebola outbreak that has devastated parts of west Africa represents an unprecedented challenge for research and ethics. Estimates from the past three decades emphasise that the present effort to contain the epidemic in the three most affected countries (Guinea, Liberia, and Sierra Leone) has been insufficient, with more than 24,900 cases and about 10,300 deaths, as of March 25, 2015. Faced with such an exceptional event and the urgent response it demands, the use of randomised controlled trials (RCT) for Ebola-related research might be both unethical and infeasible and that potential interventions should be assessed in non-randomised studies on the basis of compassionate use ...
According to Beyond Pesticides recent report on state pesticide use on cannabis, California was one of six states silent on pesticide use on marijuana. Until now, the only discussion of the issue came from a 2012 report from the California Research Bureau, commissioned by CA Assembly member Linda Halderman, M.D. The report indicated that because no pesticide products were registered for use on cannabis by EPA, and given that applying a pesticide for an unregistered use is illegal under federal pesticide law, the state could confiscate any medical marijuana crop treated with a pesticide. However, it was noted that this conflicts with the states Compassionate Use Act, which guarantees ill Californians access to medical marijuana. Thus it was determined that growers could simply not spray pesticides in order to avoid potential confiscation. But because the state had not formulated any laws or regulations governing pesticide use on cannabis, the issue remained in a grey area.. The State Water ...
A bill that would allow terminally ill people to obtain experimental drugs appears headed to Governor Browns desk for the second year in a row. Brown vetoed a similar measure last year, but supporters believe conditions are right for him to sign it this time around. Brown said he vetoed last years "Right-to-Try" measure because he wanted time to see how changes to the FDAs "Compassionate Use" program streamlined the process of getting experimental drugs to terminal patients.. Nearly a year later, the new bills supporters say its time for California to step in with its own solution, because the modified federal initiative has not reduced the minimum 30-day wait for drugs.. "The process may have to start over if the FDA has even one question," said the bills author, Assembly Majority Leader Ian Calderon (D-Whittier). "Terminally ill patients can still end up waiting weeks and months" for an approval.. Calderons measure would allow drug manufacturers to give certain terminally ill people ...
The FDA has the authority to require sponsors to perform a post-approval study (or studies) at the time of approval of a premarket approval (PMA), humanitarian device exemption (HDE), or product development protocol (PDP) application. Post-approval studies can provide patients, health care professionals, the device industry, the FDA and other stakeholders information on the continued safety and effectiveness (or continued probable benefit, in the case of an HDE) of approved medical devices. This database allows you to search Post-Approval Study information by applicant or device information.. Learn more.... ...
SynCardia Systems, Inc. (www.syncardia.com), manufacturer of the worlds first and only FDA, Health Canada and CE (Europe) approved Total Artificial Heart, announced today record-setting results for 2012 for implants, revenue and profit.. (Photo: http://photos.prnewswire.com/prnh/20130117/LA44048) "2012 was a momentous year for the SynCardia temporary Total Artificial Heart with a record-breaking 125 implants performed at more than 50 Certified Centers worldwide," said Michael Garippa, SynCardia Chairman/CEO/President. "We expect our growth trajectory to continue in 2013 as we pursue a Humanitarian Device Exemption (HDE) from the FDA for destination therapy and plan to introduce our smaller 50cc Total Artificial Heart designed to fit patients with a body surface area of less than 1.7." SynCardias accomplishments in 2012 include: -- Completion of required patient enrollment in the FDA clinical study of the [email protected] portable driver, the worlds first wearable power supply for the SynCardia Total ...
The Food and Drug Administrations expanded access program, which is intended to provide access to unapproved drugs for patients with a life-threatening illness and no available treatment, does not require that drug companies provide these drugs.
Almond CS, Morales DL, Blackstone EH, Turrentine MW, Imamura M, Massicotte MP, Jordan LC, Devaney EJ, Ravishankar C, Kanter KR, Holman W, Kroslowitz R, Tjossem C, Thuita L, Cohen GA, Buchholz H, St Louis JD, Nguyen K, Niebler RA, Walters HL, Reemtsen B, Wearden PD, Reinhartz O, Guleserian KJ, Mitchell MB, Bleiweis MS, Canter CE, Humpl T. Berlin Heart EXCOR pediatric ventricular assist device for bridge to heart transplantation in US children. Circulation. 2013 Apr 23; 127(16):1702-11 ...
This expanded access program trial compared the efficacy of regorafenib [Bayer] in patients with patients with gastrointestinal stromal tumours after disease
in Scandinavian Journal of Gastroenterology (2002), 37(7), 818-824. Background: Two-thirds to three-fourths of patients with either refractory luminal or fistulizing Crohn disease respond to infliximab treatment. The ability or inability to respond seems to persist over ... [more ▼]. Background: Two-thirds to three-fourths of patients with either refractory luminal or fistulizing Crohn disease respond to infliximab treatment. The ability or inability to respond seems to persist over time. Biological characteristics and/or genetic background can influence the response to treatment. The aim was to assess the value of C-reactive protein and TNF-alpha serum levels before treatment as well as the TNF -308 gene polymorphism in the prediction of response to infliximab treatment in Crohn disease. Methods: Two-hundred-and-twenty-six Crohn disease patients treated in the setting of an expanded access programme to infliximab in Belgium were studied. There were 136 refractory luminal diseases and 90 ...
June 12, 2008 - The FDA designated for priority review, the New Drug Application (NDA) for GEs AdreView, a molecular imaging agent for the detection of neuroendocrine tumors in pediatric and adult patients, also encouraging the company to establish an expanded access program that is designed to grant physicians limited access to a novel agent prior to FDA approval. AdreView was submitted March 20, and accepted by the FDA May 20. A priority designation is intended for products or indications that address unmet medical needs. Under the Prescription Drug User Fee Act, the FDAs goal is to review and act on 90 percent of NDAs designated as priority review within six months of receipt.. The company began development of AdreView in 2004, and the agent was granted orphan-drug designation by FDA in December of 2006.. For more information: www.gehealthcare.com. ...
BACKGROUND. Monogenic Interferon (IFN)-mediated autoinflammatory diseases present in infancy with systemic inflammation, an IFN-response-gene-signature (IRS), inflammatory organ damage and high mortality. We used the janus kinase (JAK) inhibitor baricitinib with IFN-blocking activity in vitro, to ameliorate disease. METHODS. Between October 2011 and February 2017, 10 patients with CANDLE (chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperatures), 4 with SAVI (Stimulator of IFN genes (STING)-associated vasculopathy with onset in infancy), and 4 patients with other interferonopathies were enrolled in an Expanded Access Program. Patients underwent dose-escalation, benefit was assessed by reductions in daily disease symptoms and corticosteroid requirement. Quality-of-life, organ inflammation, changes in IFN-induced biomarkers, and safety were longitudinally assessed. RESULTS. 18 patients were treated for a mean duration of 3.0 years (1.5-4.9 years). The median daily ...
In a related real-world study presented at the same session, Graham Foster from Queen Marys University of London and colleagues looked at 467 hepatitis C patients with decompensated cirrhosis treated through the English expanded access programme.. More than 70% were men, most were white, the mean age was 56 years and 6% were HIV-positive. About half had HCV genotype 1, 41% had genotype 3 and the rest had other genotypes; 47% were treatment-experienced and 10% had received liver transplants.. These patients had MELD scores ranging from 6 to 36 (mean of about 12). Child-Pugh scores - another measure of liver disease severity - were ,7, including 66% with CP class B and 10% with class C (the most severe). More than 90% had past or present liver decompensation and many had ascites, bleeding varicose veins or encephalopathy. Others had non-hepatic manifestations deemed likely to lead to irreversible damage within a year or were judged by a review panel to have other exceptional ...
The world s first pediatric heart pump, also known as Berlin Heart, is an effective alternative for children waiting for heart transplant.
Enterra® Therapy, also known as the gastric electrical stimulator (GES), is an implantable device used to help stimulate the smooth muscles of the stomach in patients with refractory gastroparesis who have failed all drug treatment options. It may be placed either laparoscopically or through a laparotomy depending on the need of the patient.. Enterra was approved by the FDA in 2000 for use as a humanitarian device in cases of idiopathic or diabetic Gastroparesis. This means it is only available in certain hospitals which have been reviewed and approved to perform the procedure. Because it is humanitarian in nature, it is often not approved by insurance companies or the approval process can be complicated. Medtronic reps are available to assist in getting patients approved for the procedure.. While the device is not a cure for GP, it does improve nausea and vomiting symptoms in many patients. It does NOT cause the stomach to contract and will not aid digestion. The settings on the device are ...
Iryna Kuchma is the Open Access Programme Manager for EIFL. Iryna has been managing the Open Access programme since 2008. Her responsibilities include the development, delivery and evaluation of a range of open access related activities.. Under Irynas management, the Open Access Programme has many achievements, including the establishment of more than 700 open access repositories and more than 4,000 open access journals in EIFLs partner countries. The programme also supported the development of 54 open access policies in EIFL partner countries, opening up a wealth of new knowledge and research to the world. An expert on open access in developing and transition countries, Iryna is frequently asked to speak at conferences and open access events around the world. Before joining EIFL, Iryna worked as an Information Programme Manager at the International Renaissance Foundation (part of Soros Foundation network in Ukraine) and also coordinated the Arts and Culture Programme there.. Iryna is a member ...
Compassionate use guideline finalized. Applied Clinical Trials, September 2007. Hero helps fight for cancer drug. BBC News, May ... Through these programs, patients are able to access drugs in late-stage clinical trials or approved in other countries for a ... With the expansion of the EU in 2004, Regulation 726/2004 evolved compassionate use programmes in order to better meet the ... Often grouped under the labels of compassionate use, expanded access, or named patient supply, these programs are governed by ...
Compassionate Use Guideline Finalized. Applied Clinical Trials Online, September 2007.. ... Sou, Hélène (August 2010). "EU Compassionate Use Programmes (CUPs): Regulatory Framework and Points to Consider before CUP ... access for a large number of patients who would not otherwise qualify for clinical trials In the European Union, named patient ... when not available through commercial routes or the clinical trial setting. In addition, MAPs provide healthcare professionals ...
While awaiting trial, Frank-McCarron was free on home confinement on $1 million bond. Frank-McCarron went on trial on January 7 ... Liss, Jennifer (July 11, 2006). "Autism: the Art of Compassionate Living". Wiretap Magazine. Retrieved 2007-11-08. Reynolds, ... In August 2011, Frank-McCarron filed a petition for a new trial. In November 2011, her petition for a new trial was denied. She ... "Convicted child killer Karen McCarron wants new trial". Smothers, Michael. "McCarron's request for new trial denied". Reynolds ...
Sam Sheppard on Trial: The Prosecutors and the Marilyn Sheppard Murder. Kent State University Press. p. 6. ISBN 9780873387705. ... "Crusading Seltzer was Compassionate Dynamo". lakewoodhistory.org. Lakewood Sun Post. April 20, 1989. Retrieved 2016-12-22. The ... Seltzer was the subject of both glowing praise and harsh criticism, particularly for his extensive coverage of the trial of Sam ... Federal district judge Carl A. Weinmann, in his 1964 ruling that Sheppard did not receive a fair trial, partially blamed the ...
Black is often referred to as the architect of holding the non-jury Pan Am Flight 103 bombing trial at the neutral venue of ... "Lockerbie bomber Abdelbaset Al Megrahi released from jail on compassionate grounds". Daily Record. 20 August 2009. Retrieved 21 ... At the end of the nine-month trial, on 31 January 2001, Abdelbaset al-Megrahi was convicted of the murder of 259 passengers and ... Jennifer Veitch, "Architect of Lockerbie trial vows to fight for an appeal", The Scotsman, 1 November 2005. Severin Carrell, ...
... they may ask the trial sponsor to consolidate these requests, creating an intermediate-size group. "Compassionate use" is a ... FDA cannot compel a company to provide a drug for compassionate use, and a company may decline a compassionate use request for ... The FDA does not have jurisdiction over this issue but there is a bill in Congress, the Compassionate Access Act of 2010 (H.R. ... Applied Clinical Trials, September 2007. Cancer Patients Deserve Faster Access to Life-Saving Drugs. Wall Street Journal, May 2 ...
During Morse's 2014 trial, he was dubbed as the "waterboarding doctor" by the media. Trial testimony did not substantiate any ... He was a teacher of medical students, a compassionate clinician, and a loving father and husband.Then his life changed". ... "Offender Status Sheet" (PDF). "The Real Melvin L Morse MD - Arrest and Trial". melvinmorse.net. Retrieved 2016-12-02. "Trial ... Morse's trial started on January 28, 2014 at the Sussex County Superior Court in Delaware. The accusation made by the State ...
After a three-week trial, Ajax offered Fandi a three-year contract, but Fandi instead signed a one-year contract with Niac ... filial and compassionate. He married South African model Wendy Jacobs in 1996 and the couple have five children; the eldest two ... are youth footballers who impressed at trials at Arsenal, Chelsea and Milan. He is the first Singaporean sportsperson to be the ...
At her trial, Musikka's doctor testified and she was acquitted. She applied and was approved to get legal cannabis cigarettes ... Musikka is one of four surviving patients enrolled in the Compassionate Investigational New Drug Program, getting marijuana ...
He was tried in the High Command Trial, as part of the Subsequent Nuremberg Trials. Reinhardt was found guilty of war crimes ... Reinhardt was released in 1952 on compassionate grounds. From 1954. Reinhardt served as president of the Gesellschaft für ... Reinhardt was tried in the High Command Trial, as part of the Subsequent Nuremberg Trials. He was found guilty of war crimes ... Hebert, Valerie (2010). Hitler's Generals on Trial: The Last War Crimes Tribunal at Nuremberg. Lawrence, Kansas: University ...
Trial[edit]. Main article: Pan Am Flight 103 bombing trial. The Scottish High Court of Justiciary at Camp Zeist was presided ... Compassionate release[edit]. Family and health[edit]. Megrahi married Aisha in 1982.[9] They had five children: four sons and ... The parties eventually agreed on a compromise and a trial was held in the Netherlands under Scots law. The trial format was ... "Lockerbie trial verdict" (PDF). Retrieved 5 June 2010.. *^ "Fhimah was found "not guilty"". I-p-o.org. 31 January 2001. ...
On 8 December 2017, Matthews signed a train and trial contract with NRL club The Gold Coast Titans. Matthews was released by ... Widnes earlier in the year on compassionate grounds. Matthews played for the Australian Schoolboys team in 2006. ...
Pan Am Flight 103 bombing trial Hans Köchler's Lockerbie trial observer mission "Uncertain future for Camp Zeist". BBC News. ... Megrahi was released from Greenock prison on compassionate grounds by the Scottish justice minister (Kenny MacAskill) on 20 ... A school on the former base was converted into a judicial court for the trial. The court was established in a neutral country ... The base was converted into a prison and a courtroom to provide the venue for the Lockerbie trial - the largest and most ...
Tongo was expected to give evidence in the trials of Mngeni and Qwabe in 2011 and 2012. Qwabe avoided trial by pleading guilty ... "Anni Dewani hitman Xolile Mngeni set for release from prison on compassionate grounds". The Telegraph. 6 July 2014. "Anni ... "Dewani trial: what really happened and how did police get it so wrong?". the Guardian. 8 December 2014. "S v Mngeni (CC25/2011 ... In pre-trial hearings on 18 February at Wynberg Magistrates Court, counsel for Mziwamadoda Qwabe said the court was unable to ...
... on compassionate grounds, suffering from terminal cancer.[citation needed] Hans Köchler's Lockerbie trial observer mission ... In week 7 of the trial, Meister testified that Mebo had supplied Libya with 20 MST-13 timing devices, and identified one of the ... Seven years later, on 18 July 2007, Lumpert claimed he had lied at the trial. In an affidavit before a Zurich notary, Lumpert ... During week 11 of the trial, Mebo lawyer Dieter Neupert filed an official criminal complaint against the Crown over what he ...
Matthew and his wife Katie raised $700k so Matthew could participate in a clinical trial. The Ridley Sandler Memorial ... After their daughter's death, Riley's parents started the Riley's Way Foundation to help her joyful spirit and compassionate ... the most compassionate. Many journalists have pointed to crowdfunding for medical expenses as evidence for the failings of for- ...
Clinical trials of sargramostim were initiated in 1987; in that same year it was administered to six people as part of a ... compassionate-use protocol for the victims of cesium irradiation from the Goiânia accident. It was approved by the FDA in March ... This medication is being investigated in trials to treat autoimmune pulmonary alveolar proteinosis (PAP). It is also being ... Phase II/III trial 2009-2013". Clinicaltrials.gov. Retrieved 2012-06-20. "Wild-Type Reovirus in Combination With Sargramostim ...
GM-CSF has also been evaluated in clinical trials for its potential as a vaccine adjuvant in HIV-infected patients. The ... in that same year it was administered to six people as part of a compassionate-use protocol for the victims of cesium ... "Trial Watch: Immunostimulatory cytokines". Oncoimmunology. 2 (7): e24850. doi:10.4161/onci.24850. PMC 3782010 . PMID 24073369. ... Clinical trials of sargramostim were initiated in 1987; ...
Also called compassionate use trial. (NCI) Experimental In clinical trials, refers to a drug (including a new drug, dose, ... NCI) Blind A randomized trial is "Blind" if the participant is not told which arm of the trial he is on. A clinical trial is " ... NCI) A person responsible for the conduct of the clinical trial at a trial site. If a trial is conducted by a team of ... 21CFR312) Phase I/II trial A trial to study the safety, dosage levels, and response to a new treatment. (NCI) Phase II trial A ...
In a remarkably short time, she gained prominence as a competent and compassionate member of the profession. Ricker worked on ... the famous Star Route trial with Robert Ingersoll - perhaps the United States' most well-known agnostic. Ricker's legal career ...
He was awaiting trial having been charged with conspiracy to murder a man from a rival loyalist paramilitary organisation. At ... While he was out on compassionate leave in early 1999, he allegedly organised the killing of Catholic human rights lawyer ... At the time of his death, Fulton had been awaiting trial, having been charged with conspiracy to murder Rodney Jennett, a ... in 1999 while he was out of prison on compassionate leave. In 2002, he was found hanged in his cell at Maghaberry Prison, an ...
They also take compassion and attempt to protect the rights of all parties involved so that they too may have an adequate trial ... effective and compassionate justice system. They primarily look to exonerate people whose case allows DNA evidence to be ... Kathleen "Cookie" RIdolfi and Linda Starr worked as trial and appellate attorneys for the Criminal Justice System. After ...
His public corruption trial ended on October 28, 2009, with convictions on 60 counts, and resulted in his automatic removal ... Langford was rejected for a compassionate release after describing himself as "terminally-ill". The date is disputed: Thomas ... After being convicted in a trial in federal court in Tuscaloosa, Alabama, Langford is now serving a 15-year federal felony ... In 2010, Langford began serving a 15-year federal felony sentence, the result of a highly publicized public corruption trial in ...
An experimental drug in Phase III clinical trials, Synsorb Pk, was used to treat 19 children on compassionate grounds under ...
The FDA currently[when?] restricts access to those drugs to patients in clinical trials and patients who get a compassionate ... whether to take an investigational drug that the FDA has approved for clinical trials. ...
Access to Nitschke's online Peaceful Pill Handbook was blocked during trials of the government's filter. A government ... "Nitschke's insight has been to recognise that assisted suicide is no longer about compassionate medicine, but about technology ...
... if and when the trial gets approval for compassionate use, will it be open to incomplete ... Compassionate use for upcoming Dr. Wise Young trial Hey all,. This is my first time posting so if I am doing something wrong or ... My question is, if and when the trial gets approval for compassionate use, will it be open to incomplete injuries like mine? ... My question is, if and when the trial gets approval for compassionate use, will it be open to incomplete injuries like mine? ...
Interventional (Clinical Trial) Actual Enrollment :. 4 participants. Intervention Model:. Single Group Assignment. ... This is a compassionate use study. In addition to providing compassionate use access to mifepristone, objectives of the study ... Compassionate Use of CORLUX® (Mifepristone) in the Treatment of Signs and Symptoms of Endogenous Cushings Syndrome. The safety ... Compassionate Use Protocol for the Administration of CORLUX® (Mifepristone) in the Treatment of the Signs and Symptoms of ...
This is a compassionate use study of the effects of pasireotide on individuals with suboptimally treated congenital ... ClinicalTrialsLocator.com provides clinical trial listings in an easy to view format. All clinical trial information is pulled ... or has recently completed a clinical trial that has been terminated and, after considering other options (for example., trial ... This is a compassionate use study of the effects of pasireotide on individuals with suboptimally treated congenital ...
The patient was treated under an Israeli compassionate use program. ... Trial Subscription. Get a 4-week free trial subscription to BioCentury Week In Review ...
... our focus is on enrolling patients in clinical trials to obtain the data required for review and approval by regulatory ... clinical trials, expanded access programs (EAPs) or compassionate use (CU). ... To find a clinical trial, visit clinicaltrials.gov. For more information on Genentech Trials, call the Trial Information ... Find Open Clinical Trials. Search clinicaltrials.gov to find out if a clinical trial exists for a specific medicine for a ...
Compassionate Release Practicum to Help Terminally Ill Inmates Seek Relief. Available in spring 2019, BU Law students in the ... Trial Advocacy: LAW JD 894. 3 credits. This course introduces the student to the structure of the trial process and the skills ... The final exam of this course will be a mock trial of a case, one that is much more compact than the Fuller case. This trial ... The Trial Advocacy Program at Boston University introduces students to the structure of the trial process and gives them ...
... Trial Phase:. N/A. Minimum Age: ... Trial Information. Compassionate Use of Omegaven® in the Treatment of Parenteral Nutrition Induced Hepatic Injury ... This compassionate use study will include patients with advanced cancer requiring PN for. long-term nutrition support who have ...
Trial Information. Feasibility of a Compassionate Same-Day Evaluation and Delivery of Single Fraction Radiotherapy for ... Trial Phase:. N/A. Minimum Age:. 18 Years. Maximum Age:. N/A ... Feasibility of a Compassionate Same-Day Evaluation and Delivery ...
A randomised controlled trial of high-dose isoniazid adjuvant therapy for multidrug-resistant tuberculosis.International ... Towards more compassionate care for people with drug resistant TB and their caregivers. "The policy was to hospitalise people ... Three-drug regimen beats XDR-TB in first trial. 17 February 2017. ... is showing that community-based care is not only more compassionate, it reduces costs and may be more effective. Similar models ...
Compassionate Use, and Randomized Clinical Trials During Pandemics ... Treating COVID-19-Off-Label Drug Use, Compassionate Use, and Randomized Clinical Trials During Pandemics Andre C Kalil. JAMA. ... Treating COVID-19-Off-Label Drug Use, Compassionate Use, and Randomized Clinical Trials During Pandemics Andre C Kalil 1 ... Compassionate use of off-label drugs: need for a change in Spain]. Avendaño Solá C. Avendaño Solá C. Med Clin (Barc). 2009 Sep ...
Transparency of Clinical Trial Data, Regulatory Data and Prices Plus a Review of the Clinical Trials Regulation" and " ... Compassionate Use & Named Patient Use in the EU Presentation and Speech November 13, 2015, Off-label Use in Europe ... Clinical Trials - What to expect from the New Regulation? Presentation and Speech ... Clinical Trials - What to Expect From the New Regulation? Presentation and Speech ...
Genervon Announces ALS and PD Phase 2a Trial Results. Both clinical and biomarker data suggested that a six-dose treatment of ... ALS Compassionate Use: An ALS patient was first diagnosed with the disease in Q1 2005 and by Q3 2008 was quadriplegic and on a ... Genervon ALS and PD Trials: The two trials were designed to determine whether a six-dose treatment of GM6 would begin the ... patients in both trials, but was delighted to find that both clinical and biomarker data in the ALS and the PD trials reported ...
compassionate use trial. A way to provide an investigational therapy to an individual who is not eligible to receive that ... Compassionate use trials allow individuals to receive promising but not yet fully studied or approved cancer therapies, when no ... A Community Advisory Board may review and monitor clinical trials and help teach the community about the trials. ... clinical trial. Also called clinical study. A type of research study that tests how well new medical approaches work in people ...
Transplant where a trial reduction of immunosuppressive drug treatment is not feasible. ... Ganciclovir: Compassionate Use in Patients With Serious or Life-Threatening Cytomegalovirus Infections. The safety and ... Ganciclovir: Compassionate Use in Patients With Serious or Life-Threatening Cytomegalovirus Infections. ... Ganciclovir: Compassionate Use in Patients With Serious or Life-Threatening Cytomegalovirus Infections. ...
Bioscience-sponsored clinical trials for trimetrexate would not be eligible for this Compassionate Use protocol. ... Keywords provided by NIH AIDS Clinical Trials Information Service: Trimetrexate. Pneumonia, Pneumocystis carinii. Leucovorin. ... A Compassionate Treatment Protocol for the Use of Trimetrexate Glucuronate With Leucovorin Protection for Patients With ... A Compassionate Treatment Protocol for the Use of Trimetrexate Glucuronate With Leucovorin Protection for Patients With ...
Keywords provided by NIH AIDS Clinical Trials Information Service: Acquired Immunodeficiency Syndrome. Cachexia. Thalidomide. ... Compassionate Use Study of Two Dose Levels of Thalidomide in Adults With HIV Wasting Syndrome. The safety and scientific ... Compassionate Use Study of Two Dose Levels of Thalidomide in Adults With HIV Wasting Syndrome. ...
Interventional (Clinical Trial) Actual Enrollment :. 22 participants. Intervention Model:. Single Group Assignment. ... Compassionate Use of Nitazoxanide for the Treatment of Clostridium Difficile Infection. The safety and scientific validity of ... Compassionate Use of Nitazoxanide for the Treatment of Clostridium Difficile Colitis in Patients Who Have Failed Conventional ... The purpose of this study is for compassionate use of nitazoxanide in the treatment of diarrheal disease due to Clostridium ...
Interventional (Clinical Trial) Actual Enrollment :. 32 participants. Intervention Model:. Single Group Assignment. ... Active Juvenile Idiopathic Arthritis (JIA) Compassionate Use. The safety and scientific validity of this study is the ... Compassionate Use Study of Adalimumab in Children 2 to , 4 Years Old or Age 4 and Above Weighing Less Than 15 kg With Active ...
must not be enrolled in another clinical trial involving an investigational agent (unless approved by the designated physicians ... Compassionate Use of Omegaven IV Fat Emulsion. The safety and scientific validity of this study is the responsibility of the ... Compassionate Use of Omegaven IV Fat Emulsion in Infants With Life Threatening Parenteral Nutrition-associated Liver Disease ( ...
Interventional (Clinical Trial) Actual Enrollment :. 79 participants. Allocation:. Non-Randomized. Intervention Model:. Single ... Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid. The safety and scientific ... I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified ... A Phase III, open label, single arm, nonrandomized, non-comparative, compassionate treatment study of cholic acid in the ...
Trial record 30 of 36 for: Brain , Adrenomyeloneuropathy Previous Study , Return to List , Next Study ... Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid. The safety and scientific ... I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified ... A Phase III, open label, single arm, nonrandomized, non-comparative, compassionate treatment study of cholic acid in the ...
... conditions that limit their capacity to comply with the cross-linking treatment procedures required by ongoing clinical trials. ... This is a compassionate treatment protocol for the use of the UV-X system for corneal collagen cross-linking (CXL) in eyes with ... Safety and Effectiveness of the UV-X System for Corneal Collagen Cross-Linking for Compassionate Treatment in Pediatric ...
Learn more about our current liver cancer clinical trials.. Compassionate Care Every Step of the Way. Our team focuses on ... Our trials include the use of chemotherapy, radiation and targeted therapies, as well quality of life/symptom management trials ... Clinical Trials. Virginia Mason is relentlessly devoted to providing excellent health care. This commitment includes conducting ...
Compassionate Counsel from a Premier Trial Lawyer. We believe there are few things more valuable than justice. Though we have ... Being prepared and ready for trial or arbitration takes time. Being prepared, ready and willing to go to trial/arbitration also ... Trial Lawyer of the Year. Award-Winning San Diego Personal Injury Attorney. At the Law Offices of Robert Vaage, we have modeled ... Vaage is not only recognized as a top trial lawyer but is also a preferred source of legal advocacy for victims and their ...
  • While this approach may be available to a patient who does not have a specific disease or condition that is being investigated in clinical trials, there must always be sufficient data available to believe that the medicine will do more good than harm. (gene.com)
  • At NewYork-Presbyterian, bone marrow transplant candidates benefit from the world-class care and experience, in a compassionate environment. (nyp.org)
  • You may have the opportunity to participate in a clinical trial studying new, safer methods of preparing for, receiving, and recovering from a bone marrow transplant. (nyp.org)
  • Brincidofovir (CMX001) was the subject of widespread social media campaigning which was then picked up by national news sources when a young boy with an adenovirus infection following a bone marrow transplant was denied compassionate use of the drug. (wikipedia.org)
  • The US FDA has also cleared Pluristem to commence patient enrollment in a Phase I trial of its PLX-R18 cells to treat insufficient hematopoietic recovery following bone marrow transplant. (wikipedia.org)
  • PASADENA, Calif.--( BUSINESS WIRE )--Genervon Biopharmaceuticals LLC ("Genervon") today announced that it has completed the analysis of its Phase 2a double blinded, randomized, placebo controlled clinical trials of Amyotrophic Lateral Sclerosis ("ALS")(NCT01854294) and Parkinson's disease ("PD")(NCT 01850381) for its drug candidate GM6 (also known as GM604 in the ALS trial and GM608 in the PD trial). (businesswire.com)
  • This strategic collaboration strongly reinforces ImmmunoSites position as a world leader in this field and will allow us to provide significant expertise in coordinating clinical trials and monitoring a patients response to drugs that have the best potential to interact with the immune system to prevent disease. (upmc.com)
  • Multiple delamanid trials have evaluated safety, tolerability, and early bactericidal activity ( 1 , 2 ). (cdc.gov)
  • Tilray currently supplies pharmaceutical-grade medical cannabis products for three different clinical trials in partnership with hospitals and universities in Canada and Australia: The first trial is being conducted in partnership with the University of British Columbia to examine the safety and tolerability of medical cannabis for Canadian military veterans and other patients suffering from PTSD. (wikipedia.org)
  • In the 1990s, Genervon realized that the many failures of clinical trials for central nervous system ("CNS") diseases can be attributed to the fact that the classic drug development paradigm - designing single-target drugs - simply cannot account for the multifactorial nature of these complex diseases. (businesswire.com)
  • In the 1990s, doctors in the US, on behalf of Muscular Dystrophy Association, approached a small family-owned manufacturer of active pharmaceutical ingredients in New Jersey, Jacobus Pharmaceuticals, about manufacturing amifampridine so they could test it in clinical trials. (wikipedia.org)
  • Based on the encouraging results of our recent Phase II trial, in which Namodenoson demonstrated clinical benefits in patients with underlying CPB7 cirrhosis, Can-Fite is committed to providing Namodenoson to fulfill the unmet medical need in this population," stated Can-Fite CEO Dr. Pnina Fishman. (benzinga.com)
  • The company is planning on returning trials of the drug for use in stem cell transplant patients to Phase II, while continuing to advance a late-stage trial for use against adenovirus infections in patients suffering from weakened immune systems. (wikipedia.org)
  • Pluristem is in the process of clinically testing the use of its PLX (PLacental eXpanded) cells in Phase I, Phase II and Phase III trials. (wikipedia.org)
  • Tilray is currently supplying pharmaceutical-grade medical cannabis products - including whole flower, oils and capsules - to thousands of patients, physicians, pharmacies, hospitals, governments and researchers around the world for commercial, compassionate access and research purposes. (wikipedia.org)
  • Information from the National Cancer Institute about clinical trials. (gene.com)
  • 1 - Pediatric Neural Tumor Shrinkage Shown in Selumetinib Clinical Trial - NIH/National Cancer Institute . (disabled-world.com)
  • 3 - Evaluating FDA Approach to Cancer Clinical Trials - Richard Pazdur, M.D. . (disabled-world.com)
  • PITTSBURGH, March 8, 2004 The University of Pittsburgh Cancer Institute (UPCI) has selected local biotechnology company, ImmunoSite, as its exclusive partner for monitoring patients on clinical trials of a special class of drugs known as immuno-potentiators drugs specifically designed to attack cancer cells by modifying the patients immune system. (upmc.com)
  • In his introductory columns, Craig already red-flagged the first-in-human cancer trial of CRISPR gene editing technology. (bioworld.com)
  • Governments should lead, not follow on prioritizing drug development Most recently, Craig challenged the ethical justifications for randomized clinical trials (RCT) involving those wildly popular targeted cancer drugs, calling RCTs "outdated" because they overlook the most significant players in the high-stakes experiments: patients. (bioworld.com)
  • CUSP9 is related several other trials using a similar conceptual approach: The COMBAT regimen for treating various advanced pediatric cancers that uses two re-purposed non-cytotoxic drugs to augment two traditional cytotoxic drugs, or the GLAD regimen that uses one traditional anti-cancer drug, gefitinib, with three re-purposed non-cancer drugs. (wikipedia.org)
  • this antibody and several others were brought into clinical trials funded by the National Cancer Institute. (wikipedia.org)
  • Wouldn't you want to know what other people are experiencing in that particular trial? (medindia.net)
  • People over 55 (or a similar cutoff age) are often excluded from trials because their greater health issues and drug use complicate data interpretation, and because they have different physiological capacity than younger people. (wikipedia.org)
  • Dutch law still theoretically applied to the area, but, barring an emergency, the Dutch authorities were banned from entering the premises and the Court had the authority to enact regulations that superseded Dutch law when necessary for the execution of the trial, and to jail people for contempt of court. (wikipedia.org)
  • The court itself, as well as people involved in the trial (officials of the court, accused, witnesses, solicitors, etc.), also enjoyed total or partial immunity from Dutch law. (wikipedia.org)
  • in that same year it was administered to six people as part of a compassionate-use protocol for the victims of cesium irradiation from the Goiânia accident. (wikipedia.org)
  • While Defoe's protagonist remains condescending towards the native people that he encounters and easily deserts his companions for personal salvation, Winkfield's protagonist is compassionate and benevolent towards the indigenous community, embracing its practices. (wikipedia.org)
  • This course introduces the student to the structure of the trial process and the skills used by trial lawyers. (bu.edu)
  • Trial Lawyers and the FDA: What Happened Behind Closed Doors? (wsj.com)
  • Brewster held a fellowship with the American College of Trial Lawyers, was an associate of the American Board of Trial Advocates and served as President and chancellor: Louis M. Welsh Inn of Court. (wikipedia.org)
  • Cowart has been a frequent participant and speaker at The Trial Lawyers College in Dubois, Wyoming, with Gerry Spence. (wikipedia.org)