NCT06391736 Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
| NCT ID | NCT06391736 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | GeneCradle Inc |
| Condition | Pompe Disease (Late-onset) |
| Study Type | INTERVENTIONAL |
| Enrollment | 33 participants |
| Start Date | 2024-04-19 |
| Primary Completion | 2026-12 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 33 participants in total. It began in 2024-04-19 with a primary completion date of 2026-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 Criteria
Inclusion Criteria: * 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 Criteria: * 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 requires invasive mechanical ventilation, or rely on noninvasive non-non-invasive assisted ventilation when sitting upright; * 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 has AAV9 neutralizing antibody titer ≥ 1:100； * Patient who has participated in a previous gene therapy research trial; * 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);
Contact & Investigator
Guang Yang
STUDY CHAIR
Chinese PLA General Hospital
Frequently Asked Questions
Who can join the NCT06391736 clinical trial?
This trial is open to participants of all sexes, aged 6 Years or older, studying Pompe Disease (Late-onset). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06391736 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06391736 currently recruiting?
Yes, NCT06391736 is actively recruiting participants. Contact the research team at ind@bj-genecradle.com for enrollment information.
Where is the NCT06391736 trial being conducted?
This trial is being conducted at Beijing, China.
Who is sponsoring the NCT06391736 clinical trial?
NCT06391736 is sponsored by GeneCradle Inc. The principal investigator is Guang Yang at Chinese PLA General Hospital. The trial plans to enroll 33 participants.