NCT06539624

Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease

A Multicenter, Non-randomized, Open-label, Dose-finding Study to Evaluate the Safety and Preliminary Efficacy of Gene Therapy With EXG110 in Subjects With Fabry Disease

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: The Children's Hospital of Zhejiang University School of Medicine · Academic / Other
• Sex: All
• Age: 7 Years
• Healthy volunteers: Not accepted

Summary

Objective: To explore the safety and tolerability of different doses of EXG110 with Fabre disease

Detailed description

An open-label, multicenter, single-arm, non-randomized, dose-escalation, and recommended dose-extension clinical design was used to evaluate the safety and efficacy of a single intravenous administration of different doses of EXG110 in patients

Conditions

• Fabry Disease

Interventions

Type	Name	Description
GENETIC	EXG110 injection	EXG110 is a recombinant adeno-associated virus (rAAV) that not only significantly increases plasma AGA activity, but is also highly expressed in target organs such as the heart and kidneys.EXG110 will be administered in a single dose by intravenous infusion.

Timeline

• Start date: 2024-10-16
• Primary completion: 2027-04-09
• Completion: 2027-04-09
• First posted: 2024-08-06
• Last updated: 2026-02-27

Locations

2 sites across 1 country: China