Tests a gene therapy drug for late-onset Pompe disease
Tests the gene therapy GC301 in people aged 6 or older with late-onset Pompe disease.
Plain-language title generated from the registry record
Registry title: Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
Key facts
- Where
- Beijing, ChinaSee the site
- Phase
- Phase 1/2
Where in China
- Chinese PLA General HospitalBeijing, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- GeneCradle Inc
- Trial code
- NCT06391736
- Record updated
- 3 July 2025
- Original description
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.Read full original descriptionShow less
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
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.
Inclusion: 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.
Exclusion: Patient who has any history or concurrent clinical organic disease, including cardiovascular and liver diseases, respiratory system, nervous system disease, or any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study; Patient who has AAV9 neutralizing antibody titer ≥ 1:100；; Patient who has participated in a previous gene therapy research trial; Patient who is positive for human immunodeficiency (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody; Patient with a history of glucocorticoid allergy; Patient who has a contraindication to study drug or to corticosteroids, or has demonstrated hypersensitivity to any of the components of the study drug; Patient who requires invasive mechanical ventilation, or rely on noninvasive non-non-invasive assisted ventilation when sitting upright; Pregnant or lactating female participants; Patients who have fertility plans within 6 months from screening to the end of the study and are unwilling to take effective physical contraceptive measures (such as a condom, intrauterine device, contraceptive ring, ligation, abstinence, etc.) for contraception (including the subject's partner).