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

Recruiting Early Phase 1 Interventional Study
Fabry Disease
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
7 – 18
Sex
Any
Trial phase
Early Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
6 (estimated)
Sponsor
Children's Hospital of Fudan University · Other
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About This Trial
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 pedia…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: 1. The participant's legal guardian fully understands the objectives, nature, methods and potential risks of the study and signs a written informed consent; If the participant is \>= 8 years old, the participant must also agree to participate in the study and sign a written info…
Contacts

Qian Shen, MD,PhD

+86 13701923307

shenqian@shmu.edu.cn

CONTACT