Condition: Fabry Disease · Sponsor: Children's Hospital of Fudan University
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.
This description comes directly from the study's public registry record.
Qian Shen, MD,PhD · +86 13701923307 · shenqian@shmu.edu.cn
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| Children's Hospital of Fudan University | Shanghai, China | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06207552