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Clinical trial

Evaluating the Safety and Efficacy of AAV9.hMCOLN1co in Treating Mucolipidosis Type IV: A Single-Center, Interventional, Open-Label, Single-Arm Clinical Study

NCT07398872 is registered in ClinicalTrials.gov with The Children's Hospital of Zhejiang University School of Medicine as lead sponsor.

NCT NCT07398872
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Registry record

As filed in ClinicalTrials.gov.

Title
Evaluating the Safety and Efficacy of AAV9.hMCOLN1co in Treating Mucolipidosis Type IV: A Single-Center, Interventional, Open-Label, Single-Arm Clinical Study
Lead sponsor
The Children's Hospital of Zhejiang University School of Medicine
Phase
Phase 1
Overall status
Enrolling by invitation
Start date
January 13, 2026
Completion date
January 20, 2031
Enrollment
1 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

mucolipidosis type iv