NCT06207552

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

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

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Children's Hospital of Fudan University · Academic / Other
• Sex: All
• Age: 7 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
GENETIC	BBM-F101 injection	The dose of BBM-F101 injection will be calculated according to the participant's weight with single intravenous infusion.

Timeline

• Start date: 2024-02-20
• Primary completion: 2025-06-01
• Completion: 2029-06-01
• First posted: 2024-01-17
• Last updated: 2024-07-09

Locations

1 site across 1 country: China