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

Stopped early

Conditions studied: X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)

In brief

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.

Key facts

Study ID

NCT02204904

Run by

Genetix Biotherapeutics Inc.

People needed

59

Starts

2015-04-01

Expected to finish

2019-12-06

Last updated by the study team

2020-05-21

Who can join

Age: any, up to 17. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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).

You may not qualify if…

• Previous treatment with a gene therapy product.
• Receipt of an experimental transplantation procedure.

Where it is running

• Children's Hospital Los Angeles — Los Angeles, California, United States
• Stanford University — Palo Alto, California, United States
• Boston Children's Hospital/Massachusetts General Hospital — Boston, Massachusetts, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Duke University Medical Center — Durham, North Carolina, United States
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Hospital Austral — Buenos Aires, Argentina
• McGill University Health Centre — Montreal, Quebec, Canada
• University Hospital Leipzig — Leipzig, Germany
• IRCCS Ospedale Pediatrico Bambine Gesú — Roma, Italy
• Princess Maxima Center for Pediatric Oncology (PMC) — Utrecht, Netherlands
• Great Ormond Street Hospital — London, United Kingdom
• Central Manchester University Hospitals NHS Foundation Trust — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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