NCT05793307View on ClinicalTrials.gov

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

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

16

Participants

Timeline

Start Date

June 2, 2023

Primary Completion Date

December 31, 2025

Study Completion Date

December 31, 2026

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

Trial Locations (5)

100005

Peking Union Medical College, Beijing

Unknown

301 Chinese PLA General Hospital, Beijing

Central South University, Xiangya Hospital, Changsha

Zhejiang University, School of Medicine, The Children's Hospital, Hangzhou

The First Affiliated Hospital of Zhengzhou University, Zhengzhou

All Listed Sponsors
lead

GeneCradle Inc

INDUSTRY