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

Recruiting

Trial ID: NCT02204904

Purpose

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.

Official Title

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

Stanford Investigator(s)

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.

Intervention(s):

genetic: Allo-HSCT

Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305