Clinical Trial

Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

Recruiting Early Phase 1
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Record status
This record was last updated July 9, 2024 (before its estimated June 2025 completion). Its status may not reflect the trial's current state.
Summary
This is a single-arm, open label, single-dose clinical study to evaluate the safety, tolerability and efficacy of BBM-F101 injection in the pediatric Fabry disease participants up to 52 weeks after infusion, and the long-term safety and efficacy of BBM-F101 injection up to 5 years after infusion. BBM-F101 injection is an adeno-associated virus (AAV) gene therapy product for the treatment of pediatric Fabry disease.
Protocol Amendment History 2 amendments
This ClinicalTrials.gov record has been amended 2 times since 2024-01-04; most recent amendment 2024-07-07.
Status change: Not Yet Recruiting → Recruiting 2024-07-07
Trial Details
NCT Number NCT06207552
Lead Sponsor Children's Hospital of Fudan University
Conditions Fabry Disease
Enrollment 6 participants
Start Date 2024-02-20
Primary Completion 2025-06 (estimated)
Study Completion 2029-06 (estimated)
Updated on ClinicalTrials.gov 2024-07-09