A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety, Tolerability, and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Late-onset Pompe Disease
Status: Recruiting
Location: See location...
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 late-onset Pompe disease （LOPD） who are ≥ 6 years old will be studied.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: f
View:
• Age ≥ 6 years, males or females;
• Patient has a diagnosis of LOPD;
• Patient has upright FVC ≥ 30% of predicted normal value;
• A 6MWT ≥ 40 meters, assistive device allowed;
• 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
Chinese PLA General Hospital
RECRUITING
Beijing
Contact Information
Primary
GeneCradle, Inc China
ind@bj-genecradle.com
86-13501380583
Time Frame
Start Date: 2024-04-19
Estimated Completion Date: 2026-12
Participants
Target number of participants: 33
Treatments
Experimental: Low dosage group
Single intravenous administration of GC301 at a dose of 3.0 x 10\^13 vector genomes per kilogram body weight
Experimental: High dosage group
Single intravenous administration of GC301 at a dose of 6.0 x 10\^13 vector genomes per kilogram body weight
Related Therapeutic Areas
Sponsors
Leads: GeneCradle Inc