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Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug

NCT05793307 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2025-07-03

No results posted yet for this study

Summary

This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are younger than 6 months old will be studied.

Conditions

  • Pompe Disease Infantile-Onset

Interventions

GENETIC

GC301

GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene

Sponsors & Collaborators

  • GeneCradle Inc

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Max Age
6 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-06-02
Primary Completion
2025-12-31
Completion
2026-12-31

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 NCT05793307 on ClinicalTrials.gov