Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

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.