NCT06114056

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD)

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: West China Hospital · Academic / Other
• Sex: Male
• Age: 5 Years – 10 Years
• Healthy volunteers: Not accepted

Summary

This study is a single-center, single-arm, non-randomized, open-label, non-controlled, dose-escalation, prospective clinical trial designed to assess the safety, tolerability, and preliminary efficacy of JWK007 injection in pediatric patients with Duchenne Muscular Dystrophy (DMD).

Detailed description

DMD is a rare genetic disorder that primarily affects males. This disease is closely associated with mutations in the DMD gene located on the X chromosome. The DMD gene encodes a protein known as dystrophin, which plays a crucial role in providing essential structural and protective support within the muscles. Gene therapy drugs using Adeno-Associated Virus (AAV) as a vector hold the promise of offering a convenient, effective, and safe treatment option for DMD patients. Therefore, we have independently developed and designed the JWK007 injection. The study was originally designed as a '3+3' dose-escalation trial with two cohorts. However, due to recruitment difficulties at the higher dose level, only the low-dose cohort (N=3) will be enrolled. No further enrollment or dose escalation will be conducted, and all safety, tolerability, and preliminary efficacy endpoints will be assessed in this single low-dose cohort.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
BIOLOGICAL	JWK007 Single intravenous infusion administration	Each patient receives a single intravenous infusion of JWK007 at a dose of 1.0 × 10\^14 vg/kg.

Timeline

• Start date: 2024-01-31
• Primary completion: 2029-11-13
• Completion: 2029-11-13
• First posted: 2023-11-02
• Last updated: 2026-03-17

Locations

1 site across 1 country: China