Brief Title
Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
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
Brief Summary
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.
Study Type
Observational
Primary Outcome
Incidence of transplant-related mortality (TRM).
Secondary Outcome
Incidence of Major Functional Disabilities (MFDs).
Condition
X-Linked Adrenoleukodystrophy (X-ALD)
Intervention
Allo-HSCT
Study Arms / Comparison 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.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Genetic
Estimated Enrollment
59
Start Date
April 2015
Completion Date
December 6, 2019
Primary Completion Date
December 6, 2019
Eligibility Criteria
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.
Gender
Male
Ages
N/A - 17 Years
Accepts Healthy Volunteers
No
Contacts
Elizabeth McNeil, MD MSc, ,
Location Countries
Argentina
Location Countries
Argentina
Administrative Informations
NCT ID
NCT02204904
Organization ID
ALD-103
Responsible Party
Sponsor
Study Sponsor
bluebird bio
Study Sponsor
Elizabeth McNeil, MD MSc, Study Director, bluebird bio, Inc.
Verification Date
December 2019