Clinical trial designs. Medical search. Frequent questions

Assessing disability outcomes in MS in clinical trials is a complex task. (msif.org)
Cohort-based enrollment can thus slow down dose-finding trials since the outcomes of the previous cohort must be fully evaluated before the next cohort can be enrolled. (cytel.com)
This unmet medical need was inversely proportional to the clinical trial size in previous years, using the conventional cardiovascular outcomes trial design. (worldwide.com)
The sizes of typical cardiovascular outcomes clinical trials have become unmanageable, untenable, and very expensive. (worldwide.com)
The conventional cardiovascular drug development program employs a large, cumbersome, Phase 3 cardiovascular outcomes trial after completion of a Phase 2 clinical trial. (worldwide.com)
For example, instead of a conventional Phase 3 outcomes trial, the PARADIGM-HF trial employed a long run-in period for safety with stopping points at completion of a certain number of patients in the run-in period. (worldwide.com)
We consider a design problem for a clinical trial with multiple treatment arms and time-to-event primary outcomes that are modeled using the Weibull family of distributions. (eurekamag.com)
The proposed optimal allocation designs are studied theoretically and are implemented using response-adaptive randomization for a clinical trial with censored Weibull outcomes. (eurekamag.com)
The frequency of migraine aura and other outcomes may also be considered, they wrote, but these "need to be defined prior to trial initiation. (neurologylive.com)
A decision-theoretic framework is proposed for designing sequential dose-finding trials with multiple outcomes. (nus.edu.sg)
In particular, we present the framework for phase I/II trials with multiple outcomes. (nus.edu.sg)
[ 2 , 3 ] There was significant improvement in angiographic results (in stent late lumen loss: 0.17 mm vs 1.00 mm) as well as clinical outcomes (binary in-lesion restenosis: 8.9% vs 36.3%) in the sirolimus stent treatment group relative to the BMS group. (medscape.com)
In addition, full access to trial data reduces the potential for incomplete reporting of study outcomes and improves the medical evidence base, which should ultimately improve clinical decision making. (bmj.com)
To date, data on clinical outcomes are scarce and on biomechanical outcomes of the dynamic MPFL reconstruction are lacking. (springer.com)
Here, we present the protocol of a randomized clinical trial for comparing clinical and biomechanical outcomes of dynamic versus static medial patellofemoral ligament reconstruction. (springer.com)
Preoperative magnetic resonance imaging, upright radiographs, surgical reports and patient records will be evaluated, and clinical and functional outcomes will be measured. (springer.com)
A pragmatic clinical trial (PCT), sometimes called a practical clinical trial (PCT), is a clinical trial that focuses on correlation between treatments and outcomes in real-world health system practice rather than focusing on proving causative explanations for outcomes, which requires extensive deconfounding with inclusion and exclusion criteria so strict that they risk rendering the trial results irrelevant to much of real-world practice. (wikipedia.org)
The pragmatic versus explanatory distinction is a spectrum or continuum rather than a dichotomy (each study can fall toward one end or the other), but the distinction is nonetheless important to evidence-based medicine (EBM) because physicians have found that treatment effects in explanatory clinical trials do not always translate to outcomes in typical practice. (wikipedia.org)
The movement toward supporting pragmatic randomized controlled trials (pRCTs) hopes to make sure that money spent on RCTs is well spent by providing information that actually matters to real-world outcomes, regardless of conclusively tying causation to particular variables. (wikipedia.org)
A randomized trial of medical care with and without physical therapy and chiropractic care with and without physical modalities for patients with low back pain: 6-month follow-up outcomes from the UCLA low back pain study. (cdc.gov)
In general, using a combination of study designs, with different strengths and weaknesses, increases the strength and validity of scientific evidence of the relationship between exposures and disease outcomes. (cdc.gov)

A new guidebook explains how rethinking Alzheimer's clinical trials design and delivery can improve the experiences of participants and their study partners. (roche.com)
It can be really tough recruiting people to clinical trials in Alzheimer's and, once on a trial, keeping to a trial schedule can be very challenging for participants. (roche.com)
Although this view was initially for pRED, it provided the team with some unexpected glimpses into the impact of clinical trials on participants and their study partners, which actually had much broader implications across all Roche Alzheimer's trials. (roche.com)
For phase 3 trials, up to 2 doses are acceptable to be administered to participants, while dosing in trials of migraine devices and non-pharmaceutical products should be adjusted to match individual participants' stage of development. (neurologylive.com)
The acceptability of the study intervention and RCT design will be explored through qualitative methods with 20 participants (patients and informal caregivers) and 12 health professionals. (ox.ac.uk)
This innovative approach has the potential to reduce resource use, decrease time to trial completion, limit allocation of participants to inferior interventions, and improve the likelihood that trial results will be scientifically or clinically relevant. (mcmaster.ca)
Participants: N= 1000 participants aged 50 or older will be included in total, with a clinical dementia rating scale (CDR) score of 0 or 0.5. (lu.se)
To conduct a clinical trial, researchers must first develop a study plan, or protocol , in which they describe the study's aims, the characteristics of the participants, the scientific approach, the outcome measures, and the plan for statistical evaluation of the data. (britannica.com)
Interventional trials assign participants to a procedure or treatment and then measure the effects of that intervention. (britannica.com)
Observational trials collect a group of participants with a specific condition who receive a procedure or treatment as part of routine care and then measure the outcome results in this group. (britannica.com)
In both types of trials, the outcome in the participants undergoing the procedure or treatment is often compared with another group called the control. (britannica.com)
Participants in both VRE and PE showed similar meaningful clinical improvement with no difference between the treatment groups. (nature.com)
Participants Clinical trialists who were corresponding authors of clinical trials published in 2010 or 2011 in one of six general medical journals with the highest impact factor in 2011. (bmj.com)
Depending on the choice of outcome, two-armed full-scale trials may require fewer than 1,000 participants (continuous outcome) or 2,000 participants (categorical outcome). (biomedcentral.com)
Its design was approved by the Institutional Review Board of Wake Forest University and all participants signed an informed consent. (biomedcentral.com)
Dr. Roy pointed in the press meet, in its effort to disseminate & build strong scientific temperament among participants (scientists), the pioneers in the field of scientific research are roped in to train the participants in designing & conducting clinical trials. (myarogya.in)
It is a phase 3 randomized, double-blind, placebo-controlled trial, where participants were randomized to bamlanivimab (4200 mg) or placebo and then followed up for 24 weeks. (biomedcentral.com)

Results on the sequential construction of optimum experimental designs yield a very general procedure for generating response-adaptive designs for the sequential allocation of treatments to patients in clinical trials. (cam.ac.uk)
Traditional group sequential design has been used in cardiovascular clinical trials over decades as the trials can potentially be stopped early, therefore, it can reduce pre-planned sample size and trial resources . (bvsalud.org)
In this systematic review , we aim to explore characteristics of adaptive and traditional group sequential trials in practice and to gain an understanding how these trial designs are currently being reported in cardiology . (bvsalud.org)
Randomised controlled phase 2/3 trials with either adaptive or traditional group sequential design in patients with cardiovascular disease were included. (bvsalud.org)
Of 456 articles found in the initial search, 56 were identified including 43 (76.8%) trials with traditional group sequential design and 13 (23.2%) with adaptive. (bvsalud.org)

The conduct of a clinical trial requires substantial investment from research funders, demands considerable time and effort from investigators, and may expose human volunteers to health risks. (bmj.com)
Lévy V., Zohar S., Porcher R., Chevret S. (2001): Alternate designs for conduct and analysis of phase I cancer trials. (sciendo.com)
We have previously led the design and conduct of large, multicenter clinical trials for PSP. (vivli.org)
Depending on the choice of outcome measures, the necessary sample sizes to conduct four-year trials appear to be feasible. (biomedcentral.com)
Before definitive trials are mounted, it is important to conduct pilot studies that address important practical concerns. (biomedcentral.com)
We also know that a phase III trial, although challenging to organize and conduct among persons at high risk, is doable. (cdc.gov)
4. In supporting Member States to conduct clinical trials in the WHO African Region, WHO in 2004 published guidelines for clinical study of traditional medicines in the African Region. (who.int)

Summarise the role and key features of feasibility studies and pilot trials to inform complex trials and illustrate how qualitative methods can be used to more efficiently answer research questions. (birmingham.ac.uk)
Clinical data captured via these methods are helping answer questions about the safety, effectiveness, and costs of new treatments. (appliedclinicaltrialsonline.com)
The mixed-methods approach will permit in-depth examination of the intervention and trial design parameters. (ox.ac.uk)
Ideally, before new drugs and other treatments, diagnostic tests, or preventive measures are accepted for general use, they should be studied in clinical trials to determine whether they have advantages over existing methods in health benefits, safety, or cost. (britannica.com)
Methods: This study was an examiner-blind, randomized controlled clinical trial using a split-mouth design. (tue.nl)
Le Tourneau C., Lee J.J., Siu L.L. (2009): Dose escalation methods in phase I cancer clinical trials. (sciendo.com)
Although none of the therapies that we tested showed any benefit, we have improved our methods of designing clinical trials. (vivli.org)
Methods Randomised double-blind, placebo-controlled clinical trial comparing a 12-week programme of using a GTN or placebo patch in combination with eccentric squats on a decline board. (bmj.com)
Methods Exploratory analysis from a 2-year, double-masked, randomised trial. (bmj.com)

In its endeavour to augment & integrate Traditional Medicine with Modern Medicine, Belagavi based premier Research Institution, Indian Council of Medical Research-National Institute of Tradition Medicine (ICMR-NITM) is conducting two days training program in ""Designing & Conducting Clinical Trials" on 14th & 15th Sept, 2023. (myarogya.in)

The authors of the article were organizers or presented during this workshop, and this article presents their consensus on the design of clinical trials for WD, addressing trial population, treatment comparators, inclusion and exclusion criteria, and treatment endpoints. (nih.gov)
The primary study endpoint should be clinical or a composite endpoint until appropriate surrogate endpoints are validated. (nih.gov)
Results The commentary includes 18 statements for guiding data extrapolation from adults, eligibility criteria to PIBD trials, use of placebo, dosing, endpoints and recommendations for feasible trials. (bmj.com)
However, for normally distributed endpoints, the determination of a design typically depends on the assumption that the patient variance in response is known. (cam.ac.uk)

Such results can be used to prioritize drugs for further investigation in observational studies or randomized clinical trials. (cdc.gov)

For certain types of adaptive design trials it is advisable to meet and discuss the trial design with regulatory agencies. (iconplc.com)
The US Food and Drug Administration recently issued guidance on adaptive trial designs, which highlighted general principles and different types of adaptive clinical trials but did not provide concrete guidance about important considerations in designing such trials. (mcmaster.ca)

In the present report, we evaluated the efficacy of oral Cu supplementation in the treatment of AD in a prospective, randomized, double-blind, placebo-controlled phase 2 clinical trial in patients with mild AD for 12 months. (springer.com)

Interpretation of clinical trial results can be challenging, as weaknesses in trial design, data collection, analysis or reporting, can compromise the usefulness of results. (nih.gov)
1 In a study of trials of multiple myeloma the authors' conclusions-that is, the authors' reported interpretation of the overall trial results-were more positive towards the benefit of experimental interventions in those trials that were funded by the pharmaceutical industry compared with trials that were funded by non-profit organisations. (bmj.com)

Teams from Roche Patient Partnerships, Product Development and Roche's Pharma Research and Early Development (pRED) unit started by asking people living with Alzheimer's, carers and patient organisations to share their feedback on Roche trials in Alzheimer's. (roche.com)
From new clinical research professionals just starting out to industry veterans looking for ways to move ahead in their careers, ACRP is where success starts - and grows. (acrpnet.org)
FREE for ACRP Members-Gain the foundational knowledge clinical research professionals need to implement digital health components in clinical trials. (acrpnet.org)
Interested in a career in clinical research? (acrpnet.org)
Explore career options, resources, and more to help you find your first opportunity in clinical research. (acrpnet.org)
Through our gold-standard education, the most rigorous certification process in the industry, and strong comment connections - ACRP prepares clinical research professionals to be the study team members employers can trust to get it right. (acrpnet.org)
I hope we recognize the need to build quality into clinical research from the initial concept all the way through to operations," Burrow says. (acrpnet.org)
The overarching goal of this R25 program is to support educational activities that complement and/or enhance the training of a workforce to meet the nation's biomedical, behavioral and clinical research needs. (nih.gov)
This FOA seeks to support programs that include innovative approaches to enhance biomedical engineering (BME) design education to ensure a future workforce that can meet the nation's needs in biomedical research and healthcare technologies. (nih.gov)
His research interests lie in the area of adaptive designs, mainly from a Bayesian perspective, as well as hierarchical model testing and checking although his secret passion is Text Mining. (cytel.com)
Click here for Part 1 of our two-part series on innovative and alternative trial design in heart failure research. (worldwide.com)
So if we think of Sisyphus rolling that large boulder up the hill and watching it come back to hit him for all eternity, we can imagine the concept of insanity as continuing to do the same thing over and over again within cardiovascular clinical research , while expecting a different result. (worldwide.com)
Collaborating with academic research organizations (AROs) and clinical research organizations (CROs) can prove beneficial in the development of trial designs. (worldwide.com)
It is important to have the academic research organization (ARO) and the contract research organization (CRO) partnering with the sponsor throughout the trial design process to define and refine the strategy. (worldwide.com)
In this position paper we aimed to provide guidance from the global clinical research network (Pediatric Inflammatory Bowel Disease Network, PIBDnet) for designing clinical trials in PIBD in order to facilitate drug approval for children. (bmj.com)
Many research questions cannot be effectively answered in a simple parallel trial design or can be more efficiently answered within an advanced design. (birmingham.ac.uk)
The course also considers feasibility studies and pilot trials (often needed to inform a definitive trial in the evaluation of a complex intervention or for difficult to recruit to trials), and how qualitative research can be embedded within trials to more efficiently answer research questions. (birmingham.ac.uk)
Jared Baeten, Vice President, Clinical Development, HIV Franchise Head at Gilead Sciences highlights how awareness and clinical innovation have transformed research in the HIV space. (appliedclinicaltrialsonline.com)
In chapter 3.3 we performed a survey among European neurologists with specialisation in neurodegenerative diseases and assessed the attitude towards the utility of the concept MCI and usefulness of research criteria in clinical practice. (vu.nl)
The MRC Biostatistics Unit is starting the new year hosting an Adaptive Designs Research Workshop sponsored by the MRC Network of Hubs for Trials Methodology Research. (cam.ac.uk)
Conclusion: The outcome of PROSPECT-AD may support AD drug development research as well as primary or tertiary prevention of dementia by providing a validated tool using a remote approach for identifying individuals at risk of dementia and monitoring individuals over time, either in a screening context or in clinical trials. (lu.se)
Objective To investigate clinical trialists' opinions and experiences of sharing of clinical trial data with investigators who are not directly collaborating with the research team. (bmj.com)
1 In clinical research, data sharing involves a research team making trial data available to individuals with whom they are not collaborating. (bmj.com)
In recent years, several major clinical research funders have adopted policies supporting or mandating data sharing. (bmj.com)
The Seniors Health and Activity Research Program Pilot trial (SHARP-P) was designed to serve these purposes for trials assessing cognitive and physical activity training. (biomedcentral.com)
Canadian Association of Clinical Research Specialists (CACRS) is a Canadian not-for-profit Corporation established in Canada in 2011. (cacrs.com)
Clancy, CM (2003), "Practical clinical trials: increasing the value of clinical research for decision making in clinical and health policy", JAMA, 290 (12): 1624-1632, doi:10.1001/jama.290.12.1624, PMID 14506122. (wikipedia.org)
2010), "Generating evidence for comparative effectiveness research using more pragmatic randomized controlled trials", Pharmacoeconomics, 28 (10): 969-976, doi:10.2165/11536160-000000000-00000, PMID 20831305, S2CID 33592391. (wikipedia.org)
The Multiplex Luminex assay was conducted at Merck Research Laboratories using their protocol established for vaccine clinical trials. (cdc.gov)
Published abstracts were accessed by online searching on site of SBPqO and organized according to: year, code research, authors (surname), banner category and the study design. (bvsalud.org)
States to strengthen health research, including clinical trials.2 Furthermore, the Regional Strategy on Regulation of Medical Products in the African Region, 2016-2025,3 calls for close collaboration with regional and international platforms, such as AVAREF, for review of clinical trial applications and to reduce the current lengthy timelines for review and authorizations of applications. (who.int)

Introduction The optimal trial design for assessing novel therapies in paediatric IBD (PIBD) is a subject of intense ongoing global discussions and debate among the different stakeholders. (bmj.com)
Decentralized clinical trials have the potential to expand the number of people who can benefit from clinical trials while accelerating therapies to more patients in need. (appliedclinicaltrialsonline.com)
Multi-arm platform trials are clinical trials that allow the testing of multiple therapies in the same clinical trial, which offers many economies of scale, and reduces costs and time to test new therapies. (vivli.org)
As adjuvant therapies have cular biology of breast cancer, clinical decision-making costs and side-effects associated with them, various still largely relies on pathological staging, grading and prognostic criteria have been established to guide the steroid receptor status. (lu.se)

The D-optimal design for the most precise estimation of model parameters is derived, along with compound optimal allocation designs that provide targeted efficiencies for various estimation problems and ethical considerations. (eurekamag.com)
This article reviews important considerations for researchers who are designing adaptive clinical trials. (mcmaster.ca)
Several factors and considerations shaped the design, planning, and implementation of the study. (biomedcentral.com)

SHARP-P was a single-blinded pilot randomized controlled trial that involved the delivery of a cognitive training intervention and/or a physical training intervention in a 2 × 2 factorial design. (biomedcentral.com)

An increase of therapeutical trials is anticipated. (nih.gov)
1967), "Explanatory and pragmatic attitudes in therapeutical trials", J Chronic Dis, 20 (8): 637-648, doi:10.1016/0021-9681(67)90041-0, PMID 4860352. (wikipedia.org)

We annotated targets and eligibility criteria for 3238 neurodegenerative disease trials registered at ClinicalTrials.gov from 2000 to 2020. (nature.com)
Trial registration The study protocol was registered at clinicaltrials.gov (NCT04849130). (springer.com)

These sites should therefore be adequately resourced and compliant with Good Clinical Practices (GCP) standards. (who.int)

The focus will be on confirmatory clinical trials. (nih.gov)
This is particularly true for confirmatory trials. (iconplc.com)

So, basket and umbrella trials are biomarker or precision medicine-based designs, and we'll go back to this in a minute. (hstalks.com)

Our results are, with some exceptions, in accordance to the hypothetical model of dynamic changes and may be helpful to determine stage specific outcome measures for clinical trials. (vu.nl)
The results of the study will clarify whether the reported surgery success for patella stabilization via dynamic MPFL reconstruction is due to muscle contraction or to the passive tenodesis effect combined with clinical outcome measures. (springer.com)
From outcome measures to entry criteria, this webinar will explore the lessons we have learned from the failed trials of the past, and how those have been integrated in to our optimization of clinical trials of the future. (als-mnd.org)

The first Wilson Disease Aarhus Symposium (May 2019) included a workshop on randomized clinical trial design. (nih.gov)

Clinical trials are studies or experiments designed to assess the safety and efficacy of new interventions such as drugs, medical devices, or biologics. (childrenshospital.org)
To avoid this frustrating recurrence in heart failure clinical trials, we will want to look at inserting novelty into our cardiovascular drug development plans to achieve drug approval quicker - but with the same if not better evaluation of efficacy and safety as in the conventional trial design - and when possible, with reduced costs. (worldwide.com)
Health authorities recognize the need to reduce cardiovascular outcome trial size and are thus open to new and innovative trial designs that will demonstrate efficacy and evaluate for safety. (worldwide.com)
These groups will have trialists who are well versed in designing trials of all types and styles, and who are familiar with what regulatory agencies are looking for related to efficacy, safety evaluation and labeling. (worldwide.com)
Another alternative trial design is to combine a Phase 2 and Phase 3 program into one fluid clinical trial, with continued enrollment held between the Phase 2 and Phase 3 periods for evaluation of data for safety and efficacy as well as a re-evaluation of sample size based on the Phase 2 period treatment effect. (worldwide.com)
The SIRIUS trial, which included 1058 patients with workhorse lesions who were randomly assigned to either sirolimus-eluting stents or BMS, demonstrated the long-term efficacy and safety of sirolimus-eluting stents. (medscape.com)
Lilly, in collaboration with the National Institute of Allergy and Infectious Diseases, conducted the BLAZE-2 trial to evaluate the efficacy and safety of the monoclonal antibody bamlanivimab (LY3819253) in preventing SARS-CoV-2 infection and COVID-19, defined as symptomatic infection, in skilled nursing and assisted living facilities. (biomedcentral.com)
Sponsored by Eli Lilly and Company, the BLAZE-2 trial, which evaluated the efficacy and safety of the monoclonal antibody bamlanivimab (LY3819253) in preventing SARS-CoV-2 infection and COVID-19 in skilled nursing and assisted living facilities, began enrollment in August 2020 and was conducted in collaboration with the National Institute of Allergy and Infectious Diseases. (biomedcentral.com)
67% of the trials stopped early, in which 14 (32.6%) were due to efficacy , and 5 (11.6%) for futility . (bvsalud.org)
For trials with adaptive designs, 54% of the trials stopped early, in which 4 (30.8%) were due to futility , and 2 (15.4%) for efficacy . (bvsalud.org)
Based on controlled efficacy trials conducted in the 1940s and on subsequent observational efficacy studies, a primary series comprising four doses of whole-cell DTP vaccine is considered 70%-90% effective in preventing serious pertussis disease (1-4). (cdc.gov)
1. Clinical trials are important for evaluating the safety and efficacy of new products prior to wider human utilization. (who.int)
The target populations for trials should present sufficiently high disease burdens to permit estimation of efficacy from a reasonable number of subjects, within a short time frame and at reasonable cost. (who.int)
2015. Efficacy and effectiveness of an rVSV-vectored vaccine expressing Ebola surface glycoprotein: interim results from the Guinea ring vaccination cluster-randomised trial. (who.int)

In addition to diagnostic testing, frequent phlebotomy is requisite for many pediatric allergy clinical trials. (frontiersin.org)

25% of the trials reported "adaptive" in abstract or title of the studies. (bvsalud.org)

these were depleted for drug trials and enriched for behavioral interventions. (nature.com)
Therefore, combining automated annotation and deep manual curation of clinical trial registrations from ClinicalTrials.gov, we set out to map the landscape of clinical trials and their therapeutic interventions and to test correlations and trends over the past 2 decades. (nature.com)
Before definitive trials are mounted, important design questions on participation/adherence, training and interventions effects must be answered to more fully inform a full-scale trial. (biomedcentral.com)
Authors' conclusions in randomised clinical trials significantly favoured experimental interventions if financial competing interests were declared. (bmj.com)
In 2014, during the largest ever outbreak of Ebola virus disease (EVD), clinical trials of candidate vaccines against EVD were conducted in the Region, resulting in a safe and highly effective vaccine.1 Achieving universal health coverage requires overcoming inequities in access to health interventions, and this, in part entails clinical trials of new or existing medicinal products, particularly those meant for poor populations. (who.int)

To achieve adequate recruitment of patients with this rare disorder, the study groups should include all clinical phenotypes and treatment-experienced as well as treatment-naïve patients. (nih.gov)
Standardization of clinical trials will permit pooling of data and allow for better treatment comparisons, as well as reduce the future numbers of patients needed per trial. (nih.gov)
2008) is a rule-based design that extends the 3+3 design with the aim to reduce the occurrence of accrual suspension after enrolling each set of three patients, thereby accelerating the trial. (cytel.com)
The aims, both now and aspirationally are, increased efficiencies for these targeted drug approaches, and we're moving ever more towards personalized medicines, and how are we going to be able to study what may be one or a very small numbers of patients in clinical trials. (hstalks.com)
So, in the picture here, you see the patients are clustered by the histologic type and the patients have undergone testing with an assay, and if you have the mutation of interest, the ones, the stick figures in red, then those patients can be entered into the trial and receive the same treatment. (hstalks.com)
After a number of patients were enrolled without significant safety signals, the trial was allowed to continue enrolling unfettered (3) . (worldwide.com)
This can save costs, decrease the overall duration of the trial, and reduce the total number of patients needed between the Phase 2 and 3 trials. (worldwide.com)
The designs will be considered in the wider context of decisions about treatment allocation to patients within the study and to the population of future patients. (cam.ac.uk)
Evaluate the impact of a specific trial design on patients and investigate what aspects of the design may or may not encourage participation. (birmingham.ac.uk)
Aiding in patient recruitment 4, 5 by identifying patients that meet clinical protocol criteria. (appliedclinicaltrialsonline.com)
KEYLYNK-008 trial finds that Keytruda in combination with chemotherapy followed by Keytruda plus Lynparza did not produce an improved overall survival benefit in patients with metastatic squamous non-small cell lung cancer. (appliedclinicaltrialsonline.com)
Clinical trials demonstrated superiority of Fabhalta to anti-C5s in hemoglobin improvement in the absence of transfusions and transfusion avoidance rate, showing clinically meaningful hemoglobin-level increases without the need for blood transfusions in patients with paroxysmal nocturnal hemoglobinuria. (appliedclinicaltrialsonline.com)
Simulation results for the two trials show that the computationally compromised strategy can perform well and appear to be ethical for allocating patients. (nus.edu.sg)
The findings will inform whether and how a definitive trial may be undertaken to test the effectiveness of this caregiver intervention for patients after hip fracture surgery. (ox.ac.uk)
Phlebotomy procedures required in food allergy (FA) diagnosis and clinical trials often induce fear and anxiety for pediatric patients. (frontiersin.org)
The primary aim of this study was to determine whether virtual reality (VR) applications were effective in reducing anxiety for pediatric FA patients undergoing phlebotomy during FA clinical trials. (frontiersin.org)
Conclusion It seems that continuous topical GTN treatment in addition to an eccentric exercise programme does not improve clinical outcome compared to placebo patches and an eccentric exercise programme in patients with chronic patellar tendinopathy. (bmj.com)
For this reason, taking into account patients' mind-sets, outlooks, and opinions becomes an imperative step in the logo design process. (gopraxis.com)
Low back pain patients presenting to a large managed care facility from October 30, 1995, through November 9, 1998, were randomly assigned in a balanced design to medical care with and without physical therapy and to chiropractic care with and without physical modalities. (cdc.gov)
The authors ask whether disparate results of recent consensus conferences, which ``good old'' clinical markers, if optimally analysed, might have attempted, using simple criteria, to define ``low- have a similar discriminating power with regard to breast risk'' patients for whom adjuvant chemotherapy should cancer prognosis as the microarray gene expression pro- not be recommended. (lu.se)

Therefore, reducing the risk of a negative Phase 3 result is no longer a prescient rationale for a Phase 2 trial. (worldwide.com)

The designs provide balance across prognostic factors with a controllable amount of randomization and speci able skewing towards better treatments. (cam.ac.uk)
Ultimately, they wrote, "these guidelines should be consulted and used in designing and conducting clinical trials of preventive treatments in children and adolescents with migraine. (neurologylive.com)
The group recommended dosing for treatments to mirror what is known with regard to pharmacokinetics and pharmacodynamics, as well as results from trials in adults as a guide for phase 2 trials. (neurologylive.com)
Mendelian randomization studies have also been designed to suggest possible treatments for COVID-19. (cdc.gov)
A number of new vaccines and treatments against meningitis, rotavirus diarrhoea, pneumonia, malaria and HIV have been tested during clinical trials in the Region. (who.int)

Popular statistical designs, such as CRM (O'Quigley et al. (cytel.com)
Describe the statistical implications, in terms of design and analysis, of each trial design. (birmingham.ac.uk)
they are based on decision rules that have been rigorously examined via statistical simulations before the first trial participant is enrolled. (mcmaster.ca)
In 69% of the trials, simulation including Bayesian approach was used to define the statistical stopping rules. (bvsalud.org)

While current best practices such as multidisciplinary/interdisciplinary education, introduction to the regulatory pathway and other issues related to the commercialization of medical devices, and clinical immersion remain encouraged components of a strong BME program, this FOA also challenges institutions to propose other novel, innovative and/or ground-breaking activities that can form the basis of the next generation of biomedical engineering design education. (nih.gov)
You will explore the benefits and challenges of each design from different perspectives and be equipped with an understanding of the available designs and when and where they are applicable. (birmingham.ac.uk)
The benefits and challenges of each design will be considered from the perspectives of different stakeholders, including patient, funder, investigator, biostatistician and trial management staff. (birmingham.ac.uk)
Evaluate the impact of each trial design on investigators and trial management groups, the logistical challenges of such designs, and the trade-off between parsimonious and statistically efficient designs, incorporating the funder's perspective. (birmingham.ac.uk)
The International Headache Society has issued a number of recommendations for the proper design of trials for the prevention of pediatric migraine, hoping to address the challenges which are unique to pediatric patient populations. (neurologylive.com)
The International Headache Society (IHS) has released new guidelines for the preventive treatment of migraine in children and adolescents, focusing on issuing a number of recommendations to address what it determined to be the major challenges with clinical trials in this population. (neurologylive.com)
Conducting a trial in the midst of a pandemic in these facilities poses several challenges, including a vulnerable elderly population, travel restrictions, supply chain interruptions, and defining the target population. (biomedcentral.com)
This paper therefore reviews the status of clinical trials, identifies the challenges and proposes actions that can be taken by Member States and partners to improve timelines for authorizations and enhance oversight of clinical trials in the Region. (who.int)

Clinicians and other decision makers in healthcare use results from clinical trials to inform practice. (nih.gov)
In addition, we investigated the perceived usefulness of biomarkers in clinical practice. (vu.nl)
Use of AD biomarkers for clinical trials and clinical practice. (vu.nl)

To shorten the study duration of phase I trials and reduce the number of accrual suspensions, use of rolling-enrollment designs is recommended, which allows concurrent patient enrollment that is faster than cohort-base enrollment. (cytel.com)
This design can be applied to adult and pediatric phase I trials. (cytel.com)
Shortening the timeline of pediatric phase I trials: the rolling six design. (cytel.com)
Leung D.H.Y., Wang Y.G. (2001): Isotonic designs for phase I trials. (sciendo.com)

2002). Simulation for Designing Clinical Trials: A Pharmacokinetic-Pharmacodynamic Modeling Perspective (1st ed. (taylorfrancis.com)
ICON offers design, simulation and execution of adaptive clinical trials. (iconplc.com)

We compare the merits of our multiple-objective response-adaptive designs with traditional randomization designs and show that our designs are more flexible, realistic, generally more ethical, and frequently provide higher efficiencies for estimating different sets of parameters. (eurekamag.com)
Any trial can be either randomized or nonrandomized and have any degree of pragmatic and explanatory power, depending on its study design, with randomization being preferable if practicably available. (wikipedia.org)
To address this need, investigators used a study design that is rooted in human genetics, Mendelian randomization , an approach that has been used to identify important modifiable risk factors for common chronic diseases. (cdc.gov)

Journal of Clinical Oncology, 26(2):190-195. (cytel.com)
Simon R., Rubinstein L., Arbuck S.G., Christian M.C., Freidlin B., Collins J. (1997): Accelerated titration designs for phase I clinical trials in oncology. (sciendo.com)

Use of adaptive design has rapidly risen as sponsors capitalise on its ability to increase portfolio valuation by protecting good drugs from failure and improved decision-making at critical junctures in the development process. (iconplc.com)
The reporting of adaptive design needs improving. (bvsalud.org)

There have been significant changes in the way clinical trials are executed today-with a move towards eClinical where technology-based solutions are being widely used to drive automation and overall efficiency into the clinical trial end-to-end process. (appliedclinicaltrialsonline.com)
Adaptive designs allow changes to be made during a clinical trial for reasons of ethics and efficiency. (cam.ac.uk)
Multi-arm multi-stage trial designs can bring notable gains in efficiency to the drug development process. (cam.ac.uk)
Clinical trials take a considerable amount of time, resources, and funding, and as such there have been numerous efforts in the field of ALS/MND to optimize clinical trials to improve their efficiency and effectiveness. (als-mnd.org)
States to improve efficiency in the processing of clinical trial applications. (who.int)

Military personnel with PTSD ( n = 192) were recruited into a multisite double-blind randomized controlled trial to receive nine weeks of VRE or PE, with DCS or placebo. (nature.com)

This manuscript will give a brief overview of clinical trial design including the strengths and limitations of various approaches. (nih.gov)
Let's switch gears and now talk about some innovative or newer clinical trial approaches that are beginning to be used. (hstalks.com)
The majority of these current approaches are more siloed around systems such as CTMS, EDC/CDMS, IVR and submissions management (i.e., trial master file, CSR, etc. (appliedclinicaltrialsonline.com)
At times, approaches already in common use are compared in a clinical trial to determine if one is superior. (britannica.com)

A new and useful feature of PoD-TPI is that it allows investigators and regulators to balance the trade-off between enrollment speed and making risky decisions by tuning a pair of intuitive design parameters. (cytel.com)
At 3 months post-treatment, the clinical parameters were re-assessed. (tue.nl)
Chernoff H. (1953): Locally optimal designs for estimating parameters. (sciendo.com)

East Bayes is an enhanced web-based extension of East, that contains the most extensive range of Bayesian and Frequentist dose escalation and dose-finding designs available, including innovative designs such as PoD-TPI. (cytel.com)
Home / Insights / Heart Failure Clinical Trials and Alternative, Innovative. (worldwide.com)
Herein, we describe the design of the study, the analytics behind facility selection, and an innovative operational model. (biomedcentral.com)

At the very top of the evidence-based hierarchy is the randomized clinical trial, which by its very design offers a parallel control group, and often masking and allocation concealment too. (hstalks.com)
Many of the biases that can compromise the integrity of other designs are eliminated by randomized clinical trials, but some of these biases are eliminated by the add-ons of masking and allocation concealment, which do not necessarily apply to all randomized trials, or even to all randomized trials that are labeled as masked and with allocation concealment. (hstalks.com)

First, investigators may share trial data by depositing it in a repository, an archive of data with terms of access defined by the organisation that maintains it. (bmj.com)
Second, investigators may share trial data on their own terms in response to individual requests. (bmj.com)

Their role is crucial to support the participant throughout the trial, helping to ensure they are able to complete the trial and that meaningful data can be gathered. (roche.com)
These differ from conventional clinical trials because they allow and even enforce continual modifications to key components of trial design while data are being collected. (mcmaster.ca)
Design: PROSPECT-AD leverages ongoing cohorts such as EPAD (UK), DESCRIBE and DELCODE (Germany), and BioFINDER Primary Care (Sweden) and Beta-AARC (Spain) by adding a collection of speech data over the telephone to existing longitudinal follow-ups. (lu.se)
Conclusions We found strong support for sharing clinical trial data among corresponding authors of recently published trials in high impact general medical journals who responded to our survey, including a willingness to share data, although several practical concerns were identified. (bmj.com)
For these reasons, many have advocated data sharing to enhance the scientific value of trial data. (bmj.com)
The goal of this project is to use recent clinical trial data to model disease progression in PSP as a basis for the design of a multi-arm platform trial. (vivli.org)
A clinical trial that includes a prospectively planned opportunity for modification of one or more specified aspects of the study design and hypotheses based on analysis of data (usually interim data) from subjects in the study. (trilogywriting.com)
The analysis of NHANES laboratory data must be conducted with the key survey design and basic demographic variables. (cdc.gov)
Data were organized in a table from the first publication and analyzed in relation to the frequency of publications per year and study design. (bvsalud.org)

A feasibility study to assess the design of a multicentre randomized controlled trial of the clinical and cost-effectiveness of a caregiving intervention for people following hip fracture surgery. (ox.ac.uk)
AIMS: This study aims to assess the feasibility of conducting a pragmatic, multicentre randomized controlled trial (RCT) to test the clinical and cost-effectiveness of an informal caregiver training programme to support the recovery of people following hip fracture surgery. (ox.ac.uk)

Trials became more selective as the mean number of inclusion and exclusion criteria increased and eligible score ranges shrank. (nature.com)

To assess the association between competing interests and authors' conclusions in randomised clinical trials. (bmj.com)
Authors' conclusions were significantly more positive towards the experimental intervention in trials funded by for profit organisations alone compared with trials without competing interests (mean difference 0.48 (SE 0.13), P=0.014), trials funded by both for profit and non-profit organisations (0.30 (SE 0.10), P=0.003), and trials with other competing interests (0.45 (SE 0.13), P=0.006). (bmj.com)
We analysed the association between financial and other competing interests and authors' conclusions in randomised clinical trials published in the BMJ . (bmj.com)

applications in cardiovascular clinical trials. (bvsalud.org)
The application of adaptive trials is increasingly popular in cardiovascular clinical trials. (bvsalud.org)

Decision-makers (including individual physicians deciding what to do next for a particular patient, developers of clinical guidelines, and health policy directors) hope to build a better evidence base to inform decisions by encouraging more PCTs to be conducted. (wikipedia.org)

In chapter 3.2 we investigated the influence of different inclusion criteria for preclinical and prodromal Alzheimer's disease (AD) on changes in biomarkers and cognitive markers and on trial sample size estimates. (vu.nl)
Collins J.M., Grieshaber C.K., Chabner B.A. (1990): Pharmacologically guided phase I clinical trials based upon preclinical drug development. (sciendo.com)

A good working knowledge of clinical trial design is essential to expertly interpret and determine the validity and generalizability of the results. (nih.gov)
Yet, it has become more the rule than the exception that the Phase 2 trial is discordant with the results of the Phase 3 trial. (worldwide.com)
It has not been uncommon for a potential therapy to show promise in Phase 2 trial results, then prove to be ineffective in the subsequent, larger-scale Phase 3 trial. (als-mnd.org)
In contrast, trials with adoptive designs provide greater flexibility and are more efficient due to the ability to modify trial design according to the interim analysis results. (bvsalud.org)
With trial results now available, we know that this vaccine was not efficacious. (cdc.gov)

It enables thorough examination and comparison of operating characteristics of different dose finding designs effected by convenient simulations. (cytel.com)
Reiner E., Paoletti X., O'Quigley J. (1999): Operating characteristics of the standard phase I clinical trial design. (sciendo.com)

R-TPI Design is a rolling enrollment design that combines the features in model-based designs such as mTPI-2 (Guo et al. (cytel.com)
All the rolling enrollment designs I speak about in this blog are currently available in East Bayes. (cytel.com)

Vital to the success of these programmes is the participation of people living with Alzheimer's, from the start of the trial to its completion, which can sometimes last several years. (roche.com)
When it comes to participation, they rely heavily on the availability of a study partner to support the person living with Alzheimer's throughout the trial. (roche.com)
Taking part in a clinical trial can be a very challenging experience both for the person living with Alzheimer's and their study partner supporting their participation," said Léa Proulx, Patient Voice Partner at Roche. (roche.com)
We wanted to get a fresh view of the wider needs of people living with Alzheimer's, their care partners and study partners and co-create solutions to improve their joint experiences and participation in Alzheimer's clinical trials. (roche.com)
Additionally, needle phobia may contribute to reluctance toward routine diagnostic testing and participation in FA trials that require phlebotomy. (frontiersin.org)

What has become apparent in the era of advanced health technology is that we also need to know about comparative effectiveness in real-world applications so that we can ensure the best use of our limited resources as we make countless instances of clinical decisions. (wikipedia.org)

By leveraging its powerful range of designs and reporting tools, East Bayes helps clinicians save time, gain confidence in their dose-escalation decisions, increase development productivity, and provides greater opportunity for trial success and patient safety. (cytel.com)

This module introduces advanced trial designs such as adaptive, cluster, factorial, and cross-over trials. (birmingham.ac.uk)
This short course introduces examples of advanced designs, such as adaptive, cluster, factorial and cross-over trials, building on the key concepts and features of clinical trial design and their delivery, considered in earlier courses within the programme. (birmingham.ac.uk)
SHARP-P was a single-blinded randomized controlled pilot trial of a 4-month physical activity training intervention (PA) and/or cognitive training intervention (CT) in a 2 × 2 factorial design with a health education control condition in 73 community-dwelling persons, aged 70-85 years, who were at risk for cognitive decline but did not have mild cognitive impairment. (biomedcentral.com)

Our lab group has expertise in the development, design, and implementation of randomized control clinical trials of analgesics and novel local anesthetics on pediatric and adult cohorts. (childrenshospital.org)
We consider several examples, provide R code for general implementation, and show that our designs confer a familywise error-rate and power close to the desired level. (cam.ac.uk)

We found that lower CSF Aβ 1-42 levels within the normal range are predictive for faster clinical progression in non-demented memory clinic subjects. (vu.nl)

We need to listen and better adapt to their needs in order to keep them on that trial," explains Ruth Croney, Clinical Programme Leader, Roche UK. (roche.com)
This is a core module on the MSc/PGDip Clinical Trials programme. (birmingham.ac.uk)

In August 2018, Szperka told NeurologyLive that one of the biggest steps in the pediatric migraine space was the fact that for preventive treatment, the field is just "getting there" in terms of understanding how to design trials properly. (neurologylive.com)
Second, while reports sometimes categorize trials as preventive versus symptomatic, we wished to examine more quantitative metrics of disease stage or severity. (nature.com)

Developed by accomplished study design experts, East creates clinical trials that best address key questions confronted by clinical trial sponsors. (cytel.com)
Incorporating CDISC-based libraries to store study components for study design and building eCRF pages. (appliedclinicaltrialsonline.com)
There still exists certain phases such as study design and study build within the clinical trial process that can have a significant impact based on reduction in overall time for getting trials ready for study execution across the clinical sites. (appliedclinicaltrialsonline.com)
This article provides a framework called clinical trial design automation that helps establish a recommended approach to manage the study design and study build phase of the overall clinical trial process. (appliedclinicaltrialsonline.com)
The approach includes process and technology to aid in speeding up critical elements of a clinical trial lifecycle-elements covered are from study design (protocol design) to study build. (appliedclinicaltrialsonline.com)
This recommended approach has significant value for key clinical trial stakeholders-sponsors, CROs, and eClinical vendors, each with the potential for driving efficiencies in the study design and study build phases of clinical trials resulting in the overall cost reduction of clinical trial execution while enabling flexibility to meet new regulations and clinical trial trends. (appliedclinicaltrialsonline.com)

Sixteen novel, genetically supported therapeutic hypotheses tested in drug trials represent a small, non-increasing fraction of trials, and the mean lag from genetic association to first trial was 13 years. (nature.com)
In this study, we sought to understand the disease stages and therapeutic hypotheses studied in clinical trials conducted in neurodegenerative diseases to date. (nature.com)

Diseases19

Chemicals and Drugs16

Analytical, Diagnostic and Therapeutic Techniques and Equipment89

Psychiatry and Psychology8

Phenomena and Processes8

Disciplines and Occupations12

Anthropology, Education, Sociology and Social Phenomena10

Technology, Industry, Agriculture2

Humanities2

Information Science12

Named Groups3

Health Care84

Geographicals2

Outcomes21

Participants17

Sequential5

Conduct7

Methods9

20231

Endpoints4

Observational studies1

Types of adaptive2

Placebo-controlled1

Interpretation2

Research31

Therapies4

Considerations3

Pilot Randomized1

Therapeutical trials2

Registered at ClinicalTrials.gov2

Good Clinical Practices1

Confirmatory2

Biomarker1

Outcome measures3

20191

Efficacy14

Pediatric allergy1

Abstract1

Interventions5

Patients20

Rationale1

Treatments5

Statistical4

Challenges8

Practice3

Phase I tria4

Simulation2

Randomization3

Oncology2

Adaptive design2

Efficiency5

Double-blind randomized1

Approaches4

Parameters3

Innovative3

Allocation2

Investigators2

Data9

Multicentre2

Inclusion and exclusion criteria1

Conclusions3

Cardiovascular2

Inform1

Preclinical2

Results5

Characteristics2

Rolling enrollment2

Participation5

Effectiveness1

Decisions1

Factorial3

Implementation2

Progression1

Programme2

Preventive2

Study design6

Hypotheses2