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Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease

NCT05567627 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2023-11-01

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

  • Infantile-onset Pompe Disease

Interventions

BIOLOGICAL

Genetic: GC301

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

Sponsors & Collaborators

  • GeneCradle Therapeutics, Inc

    collaborator UNKNOWN

  • Seventh Medical Center of PLA General Hospital

    lead OTHER

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
6 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-08-01
Primary Completion
2024-09-30
Completion
2025-09-30

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 NCT05567627 on ClinicalTrials.gov