Single Arm, Multicenter, Open and Dose-escalation Clinical Study on Safety, Tolerance, and Efficacy of GC301, an AAV-Delivered Gene Transfer Therapy in Patients With Infantile-onset Pompe Disease
Status: Recruiting
Location: See location...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Not Applicable
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:
• 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;
• The patient must be no older than 6 months;
• The patient must be diagnosed with infantile-onset Pompe disease.
Locations
Other Locations
China
Bayi Children's Hospital, Seventh Medical Center, PLA general hospital
RECRUITING
Beijing
Contact Information
Primary
Zhichun Feng
zhichunfeng81@163.com
+86(10) 66721786
Time Frame
Start Date: 2022-08-01
Estimated Completion Date: 2025-09
Participants
Target number of participants: 6
Treatments
Experimental: Initial dose cohort
1.2x10\^14 vg/kg of GC301 administered via intravenous infusion
Related Therapeutic Areas
Sponsors
Collaborators: GeneCradle Therapeutics, Inc
Leads: Seventh Medical Center of PLA General Hospital