A Prospective and Retrospective Data Collection Study to Evaluate Outcomes in males <= 17 years of age undergoing Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Cerebral Adrenoleukodystrophy.
- Conditions
- Adrenoleukodystrophybronze Schilder disease10029299
- Registration Number
- NL-OMON47132
- Lead Sponsor
- bluebird bio
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 6
Subjects must:
1. Provide informed consent from a competent custodial parents or guardian with legal capacity to execute a local Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved consent. In addition, informed assent will be sought from capable subjects, in accordance with the directive of the IRB/IEC and with local requirements.
2. Be male and <=17 years of age at the time of treatment, for retrospective and partial prospective/retrospective subjects, or at time of parental/guardian consent and, where appropriate, subject assent.
3. Have a confirmed diagnosis of CALD as defined by abnormal VLCFA profile and cerebral lesion on brain MRI.
4. Depending on the cohort, the subject must:
- Be scheduled for allo-HSCT evaluation/procedure at a study site (prospective cohort only)
- Received an allo-HSC infusion and to be consented in time to complete the Month 24 Visit on study (partial prospective/retrospective cohort only) or
- Have received their most recent allo-HSC infusion on or after January 1, 2013 (retrospective cohort only).
Subjects are excluded if they meet any of the following criteria.
1. Previous treatment with a gene therapy product.
2. Receipt of an experimental transplant procedure.
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method