Actively Recruiting
Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
Led by GeneCradle Inc · Updated on 2025-07-03
33
Participants Needed
1
Research Sites
136 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 6 years or older, male or female
- Diagnosed with late-onset Pompe disease (LOPD)
- Upright forced vital capacity (FVC) is 30% or more of predicted normal value
- Able to complete a 6-minute walk test of at least 40 meters, assistive devices allowed
- Legal guardian can understand study purpose and risks and provide signed informed consent before any study procedures
You will not qualify if you...
- History or current serious organic disease including heart, liver, respiratory, or nervous system conditions that make participation unsuitable
- Requires invasive mechanical ventilation or depends on noninvasive assisted ventilation while sitting upright
- Positive for HIV antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody
- History of allergy to glucocorticoids
- Contraindication or hypersensitivity to the study drug or corticosteroids
- AAV9 neutralizing antibody titer equal or greater than 1:100
- Prior participation in a gene therapy research trial
- Pregnant or breastfeeding females
- Planning pregnancy within 6 months of screening to study end and unwilling to use effective physical contraception methods
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Chinese PLA General Hospital
Beijing, China, 100853
Actively Recruiting
Research Team
G
GeneCradle, Inc China
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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