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

Bluebird Bio

Status: Terminated

Conditions: Adrenoleukodystrophy (ALD); Cerebral Adrenoleukodystrophy (CALD); X-Linked Adrenoleukodystrophy (X-ALD)

Treatments: Genetic: Allo-HSCT

Study type: Observational

Funder types: Industry

Identifiers

NCT02204904

ALD-103

Details and patient eligibility

About

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.

Enrollment: 59 patients

Sex: Male

Ages: Under 17 years old

Volunteers: No Healthy Volunteers

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.

Trial design

59 participants in 3 patient groups

Allo-HSCT prospective

Description:

Subjects who will be consented before they received an allo-HSC infusion. They will be consented and enrolled on the study during the Screening Period.

Treatment:

Genetic: Allo-HSCT

Allo-HSCT partial prospective/retrospective

Description:

Subjects who will be consented after they received an allo-HSC infusion but before they reach 24 months post-infusion on study. Subjects in this cohort will participate prospectively in at least the Month 24 Visit in order to obtain prospective on-study data for this and all visits after Month 24

Treatment:

Genetic: Allo-HSCT

Allo-HSCT retrospective

Description:

Subjects who received an allo-HSC infusion on or after January 1, 2013 and died before study data collection.

Treatment:

Genetic: Allo-HSCT