A Single-arm, Open Label, Single-dose Clinical Study to Evaluate the Safety, Tolerability and Efficacy of BBM-F101 Injection in the Treatment of Pediatric Fabry Disease
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.
• 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 informed consent;
• Decreased α-Gal A (α-galactosidase A) and confirmed diagnosis of Fabry Disease by genetic testing;
• Males or females aged ≥7 years and \<18 years old;
• Acceptable eGFR (estimated Glomerular Filtration Rate) result in screening period;
• Participants had at least one of the clinical manifestations for Fabry disease;
• Acceptable capsid antibody titers;
• Acceptable anti α-Gal A antibody titers;
• Acceptable laboratory values;
• Participant's legal guardian and participant with good cooperation and compliance;
⁃ Use of reliable contraception methods during the study for adolescence.