NCT05567627View on ClinicalTrials.gov

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

NARecruitingINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

August 1, 2022

Primary Completion Date

September 30, 2024

Study Completion Date

September 30, 2025

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

Trial Locations (1)

100700

RECRUITING

Bayi Children's Hospital, Seventh Medical Center, PLA general hospital, Beijing

Sponsors
All Listed Sponsors
collaborator

GeneCradle Therapeutics, Inc

UNKNOWN

lead

Seventh Medical Center of PLA General Hospital

OTHER