Status:
TERMINATED
Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
Lead Sponsor:
Genetix Biotherapeutics Inc.
Conditions:
X-Linked Adrenoleukodystrophy (X-ALD)
Cerebral Adrenoleukodystrophy (CALD)
Eligibility:
MALE
Up to 17 years
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.
Eligibility Criteria
Inclusion
- 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.
- 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.
- Have a confirmed diagnosis of CALD as defined by abnormal VLCFA profile and cerebral lesion on brain MRI.
- 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
- Previous treatment with a gene therapy product.
- Receipt of an experimental transplantation procedure.
Key Trial Info
Start Date :
April 1 2015
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 6 2019
Estimated Enrollment :
59 Patients enrolled
Trial Details
Trial ID
NCT02204904
Start Date
April 1 2015
End Date
December 6 2019
Last Update
May 21 2020
Active Locations (13)
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1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
2
Stanford University
Palo Alto, California, United States, 94304
3
Boston Children's Hospital/Massachusetts General Hospital
Boston, Massachusetts, United States, 02141
4
University of Minnesota
Minneapolis, Minnesota, United States, 55455