drugset / Trial / NCT07398872
Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV
Phase 1
Enrolling by invitation
1 enrolled
The Children's Hospital of Zhejiang University School of Medicine
NaSingle-groupOpen-labelTreatment
Summary
Safety and Efficacy of AAV9.hMCOLN1co for patients with Mucolipidosis Type IV(MLIV): A Single-Center, Interventional, Open-Label, Single-Arm Clinical Study. The goal of this clinical trial is to evaluate whether a gene therapy can safely treat children with MLIV.
Timeline
- Start
- 2026-01-13
- Primary completion
- 2027-01-20
- Completion
- 2031-01-20
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAV9.hMCOLN1co | Gene therapy (AAV / viral vector) | 2e+14 vg | Intrathecal |