Trials / Enrolling By Invitation
Enrolling By InvitationNCT06888661
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy
An Open-label, Dose-escalation Clinical Trial to Assess the Safety and Efficacy of EXG001-307 After Intrathecal Injection in Patients With Spinal Muscular Atrophy
- Status
- Enrolling By Invitation
- Phase
- EARLY_Phase 1
- Study type
- Interventional
- Enrollment
- 4 (estimated)
- Sponsor
- Guangzhou Jiayin Biotech Ltd · Industry
- Sex
- All
- Age
- 1 Day – 2 Years
- Healthy volunteers
- Not accepted
Summary
The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of EXG001-307 as a treatment of spinal muscular atrophy .
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | EXG001-307 injection | non-replicating, rAAV vector based on AAV9 containing cDNA encoding the human SMN protein. |
Timeline
- Start date
- 2025-03-10
- Primary completion
- 2026-03-01
- Completion
- 2026-12-30
- First posted
- 2025-03-21
- Last updated
- 2025-03-21
Locations
2 sites across 1 country: China
Source: ClinicalTrials.gov record NCT06888661. Inclusion in this directory is not an endorsement.