Summary
Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.
Conditions
X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
Recruitment Status
Terminated
Eligibility Criteria
Inclusion Criteria:
Provide informed consent from a competent custodial parent 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 institution's IRB/IEC and all other local requirements.
Be male and ≤17 years of age at the time of treatment, for retrospective and partial prospective/retrospective subjects, or at the time of parental/guardian consent and, where appropriate, subject assent, for prospective subjects.
Have a confirmed diagnosis of CALD as defined by abnormal VLCFA profile and cerebral lesion on brain MRI.
Depending on the cohort, the subject must:
Be scheduled for allo-HSCT evaluation at a study site (prospective cohort only),
Have received an allo-HSC infusion and 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).
Exclusion Criteria:
Previous treatment with a gene therapy product.
Receipt of an experimental transplantation procedure.
Intervention
Intervention Type
Intervention Name
Genetic
Allo-HSCT
Gender
Male
Min Age
N/A
Max Age
17 Years
Download Date
May 21, 2020
Principal Investigator
N/A
Primary Contact Information
For more information on this trial, visit clinicaltrials.gov.
Contact
For more information and to contact the study team: