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

NCTID	NCT06207552 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Active
Indication	Fabry Disease
Disease Ontology Term	DOID:14499
Compound Name	BBM-F101
Sponsor	Children's Hospital of Fudan University
Funder Type	Other
Recruitment Status	Recruiting
Enrollment Count	6
Results Posted	Not Available

Target Gene/Variant	GLA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV
Editor Type	none
Dose 1	Undisclosed dose
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China