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

Trial ID or NCT#

NCT02204904

Status

not recruiting iconNOT RECRUITING

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

Eligibility Criteria

Sexes Eligible for Study: MALE
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. 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:
    1. * 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).
      1. Exclusion Criteria:
  2. 1. Previous treatment with a gene therapy product.2. Receipt of an experimental transplantation procedure.
Exclusion Criteria:
  1. 1. Previous treatment with a gene therapy product.2. Receipt of an experimental transplantation procedure.
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