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.
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