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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
The dose of BBM-F101 injection will be calculated according to the participant's weight with single intravenous infusion.
Children's Hospital of Fudan University
Shanghai, China
RECRUITINGIncidence of dose limited toxicity
The incidence of dose limited toxicity (DLT) events as determined by the safety review committee (SRC) within DLT observation period following the BBM-F101 injection
Time frame: 12 weeks
Incidence of adverse events and serious adverse events
The incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks following the BBM-F101 injection
Time frame: 52 weeks
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