Education and Training
Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
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.
Stanford is currently accepting patients for this trial.
Stanford Investigator(s):
Intervention(s):
- genetic: Allo-HSCT
Eligibility
Inclusion Criteria:
1. 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.
2. 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.
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 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:
1. Previous treatment with a gene therapy product.
2. Receipt of an experimental transplantation procedure.
Ages Eligible for Study
N/A - 17 Years
Genders Eligible for Study
Male
Now accepting new patients
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Recruiting