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Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV

NCT07398872 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2026-02-10

No results posted yet for this study

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

  • Mucolipidosis Type IV

Interventions

BIOLOGICAL

AAV9.hMCOLN1co

A single intrathecal infusion of 10 mL at 2E13 vg/mL for a total dose of 2E14 vg

Sponsors & Collaborators

  • The Children's Hospital of Zhejiang University School of Medicine

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Months
Max Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-01-13
Primary Completion
2027-01-20
Completion
2031-01-20

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07398872 on ClinicalTrials.gov