Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug
Pompe Disease Infantile-Onset
About this trial
This is an interventional treatment trial for Pompe Disease Infantile-Onset
Eligibility Criteria
Inclusion Criteria: Age < 6 months Patient has diagnosis of infantile onset Pompe disease 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: Left ventricle ejection fraction (LVEF) < 40%; Patient who has AAV9 neutralizing antibody titer ≥ 1:100; Patient who has received enzyme replacement therapy (ERT) more than twice; Patient who has respiratory dysfunction before enrollment, including the blood oxygen (O2) saturation level < 90%, or the partial pressure of carbon dioxide (PCO2) in venous blood > 55 mmHg, or PCO2 in arterial blood > 40 mmHg; Patient who has laboratory abnormalities of: creatinine > Upper Limit of Normal (ULN), hemoglobin < 90 g/L; Patient with congenital organ absence; Patient with a history of glucocorticoid allergy; Patient who is positive for human immunodeficiency (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody; Patient who has participated in a previous gene therapy research trial; Patient who has any concurrent clinically significant major disease or any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study.
Sites / Locations
- Peking Union Medical CollegeRecruiting
- 301 Chinese PLA General Hospital
- Central South University, Xiangya Hospital
- Zhejiang University, School of Medicine, The Children's HospitalRecruiting
- The First Affiliated Hospital of Zhengzhou University
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Experimental
Cohort 1
Cohort 2
Cohort 3
Single intravenous administration of GC301 at a dose of 8.0 x 10^13 vector genomes per kilogram body weight
Single intravenous administration of GC301 at a dose of 1.2 x 10^14 vector genomes per kilogram body weight
Single intravenous administration of GC301 at a dose of 1.8 x 10^14 vector genomes per kilogram body weight