• study
  • Signed informed consent by the patient and/or parents/legal guardian was obtained as soon as it is confirmed that the patient met inclusion/exclusion criteria and the parents/guardian would agree for the child to participate in the study. (clinicaltrials.gov)
  • Compare the frequency and severity of neurological disability of study patients with untreated historical controls. (clinicaltrials.gov)
  • While this study will be registered with clinicaltrials.gov, no specific patient recruitment materials will be utilized. (clinicaltrials.gov)
  • months
  • Patients are followed at 3, 6, and 12 months and then may be followed every 6 months thereafter. (clinicaltrials.gov)
  • This revised transplant protocol will test the following: 1) the ability to achieve engraftment with the reduced intensity protocol, 2) the mortality associated with transplant by day 100, 3) patient outcomes, based on differential neurologic, neuropsychologic, imaging and biologic evaluations prior to transplantation and at designated points after transplantation (day 100, 6 months, 1, 2 and 5 years). (clinicaltrials.gov)
  • Patients are followed monthly for 6 months, then every 3 months until death. (clinicaltrials.gov)
  • Patients are assessed at 3 and 6 months for liver function response, neurologic status, and nutritional status. (clinicaltrials.gov)