Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
Launched by GENECRADLE INC · Apr 25, 2024
Trial Information
Current as of July 01, 2025
Recruiting
Keywords
ClinConnect Summary
This clinical trial is investigating a new gene therapy drug called GC301, which is designed to treat late-onset Pompe disease (LOPD). Pompe disease is a rare genetic condition that affects how the body breaks down certain sugars, leading to muscle weakness and other health issues. The study aims to determine if this new treatment is safe and effective for patients aged 6 years and older who have been diagnosed with LOPD.
To participate in the trial, patients must meet certain criteria. They need to be at least 6 years old and have a specific level of lung function and walking ability. It's also important that patients do not have certain other health conditions that could make them unsuitable for the study. Those who join the trial can expect to receive the gene therapy and undergo regular evaluations to monitor their health and the treatment's effects. Additionally, participants will need consent from a legal guardian if they are under 18, and they will be informed about the potential risks and benefits before joining the study.
Gender
ALL
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);
About Genecradle Inc
GeneCradle Inc. is a pioneering biotechnology company dedicated to advancing genetic therapies and innovative solutions in personalized medicine. With a robust focus on harnessing cutting-edge genomic technologies, GeneCradle aims to improve patient outcomes through the development of novel therapeutics targeting a range of genetic disorders. Committed to rigorous scientific research and ethical practices, the company collaborates with leading research institutions and healthcare professionals to translate breakthrough discoveries into effective clinical applications. Through its comprehensive clinical trials, GeneCradle seeks to establish new standards in treatment efficacy and safety, ultimately enhancing the quality of life for patients worldwide.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Beijing, , China
Patients applied
Trial Officials
Guang Yang
Study Chair
Chinese PLA General Hospital
Guang Yang
Principal Investigator
Chinese PLA General Hospital
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported