NCT06178432

Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)

A Single-arm, Open-label, Single-dose Study to Evaluate the Safety, Tolerability, and Efficacy of CRG003 Injection in the Treatment of Late Onset Pompe Disease

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Huashan Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a single-center, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of CRG003 injection in participants with late onset Pompe disease (LOPD), with a long-term follow-up period of 5 years. CRG003 (BBM-G102) injection is an adeno-associated virus (AAV) gene therapy product for treating Pompe disease to stably express active GAA enzyme in the liver on a long-term basis after the injection.

Conditions

• Pompe Disease (Late-onset)

Interventions

Type	Name	Description
GENETIC	CRG003 injection	The dose of CRG003 will be calculated according to the participant's weight with single intravenous infusion.

Timeline

• Start date: 2023-12-01
• Primary completion: 2024-12-01
• Completion: 2028-12-01
• First posted: 2023-12-21
• Last updated: 2023-12-21

Locations

1 site across 1 country: China