Trials / Enrolling By Invitation
Enrolling By InvitationNCT07398872
Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV
Evaluating the Safety and Efficacy of AAV9.hMCOLN1co in Treating Mucolipidosis Type IV: A Single-Center, Interventional, Open-Label, Single-Arm Clinical Study
- Status
- Enrolling By Invitation
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 1 (estimated)
- Sponsor
- The Children's Hospital of Zhejiang University School of Medicine · Academic / Other
- Sex
- All
- Age
- 18 Months – 8 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | AAV9.hMCOLN1co | A single intrathecal infusion of 10 mL at 2E13 vg/mL for a total dose of 2E14 vg |
Timeline
- Start date
- 2026-01-13
- Primary completion
- 2027-01-20
- Completion
- 2031-01-20
- First posted
- 2026-02-10
- Last updated
- 2026-02-10
Locations
1 site across 1 country: China
Source: ClinicalTrials.gov record NCT07398872. Inclusion in this directory is not an endorsement.