A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Infantile-onset Pompe Disease
Status: Active_not_recruiting
Location: See all (5) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Maximum Age: 6 months
Healthy Volunteers: f
View:
• Age \< 6 months
• Patient has diagnosis of infantile onset Pompe disease
• The patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated informed consent prior to any study-related procedures being performed.
Locations
Other Locations
China
301 Chinese PLA General Hospital
Beijing
Peking Union Medical College
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
Time Frame
Start Date: 2023-06-02
Completion Date: 2026-12
Participants
Target number of participants: 16
Treatments
Experimental: Cohort 1
Single intravenous administration of GC301 at a dose of 8.0 x 10\^13 vector genomes per kilogram body weight
Experimental: Cohort 2
Single intravenous administration of GC301 at a dose of 1.2 x 10\^14 vector genomes per kilogram body weight
Experimental: Cohort 3
Single intravenous administration of GC301 at a dose of 1.8 x 10\^14 vector genomes per kilogram body weight
Related Therapeutic Areas
Sponsors
Leads: GeneCradle Inc