Last updated: October 30, 2023
Sponsor: Seventh Medical Center of PLA General Hospital
Overall Status: Active - Recruiting
Phase
N/A
Condition
Pompe Disease
Treatment
Genetic: GC301
Clinical Study ID
NCT05567627
JLJY-GC301-IOPD-003
Ages < 6 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
- The patient's legal guardian(s) must be able to understand the purpose and risks ofthe study and voluntarily provide signed and dated informed consent prior to anystudy-related procedures being performed;
- The patient must be no older than 6 months;
- The patient must be diagnosed with infantile-onset Pompe disease.
Exclusion
Exclusion Criteria:
- Class IV patient based on Modified Ross Heart Failure Classification for Children;
- Aspartate aminotransferase (AST), alanine aminotransferase (ALT) > 3x upper limit ofnormal (ULN), alkaline phosphatase (ALP) > 2x ULN (with the exception of liverabnormalities related to Pompe disease);
- Patient has severe organ dysfunction, such as liver and kidney failure (Liver failure:patients may have liver failure syndrome, including fatigue, severe gastrointestinalsymptoms; clinical examination found prolonged prothrombin time, prothrombin activityless than 40%; Neuropsychiatric symptoms, such as restlessness, changes in personalityand behavior, lethargy, coma, etc.; Toxic tympanic bowel, ascites, multiple organdysfunction, etc.; hyperalbuminemia exceeding 171 μmol/L, hypoalbuminemia. Renalfailure: creatinine exceeding 110 μmol/L, or glomerular filtration rate less than 100mL/min), congenital/acquired encephalopathy, etc.;
- Patient with congenital organ absence;
- Patient with primary immunodeficiency;
- Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis Bsurface antigen, hepatitis C antibody, or treponema pallidum antibody;
- Patient with a history of glucocorticoid allergy;
- Patient who has participated in a previous gene therapy research trial;
- Patient who has any concurrent clinically significant major disease or any othercondition that, in the opinion of the Investigator, makes the subject unsuitable forparticipation in the study.
Study Design
Total Participants: 6
Treatment Group(s): 1
Primary Treatment: Genetic: GC301
Phase:
Study Start date:
August 01, 2022
Estimated Completion Date:
September 30, 2025
Connect with a study center
Bayi Children's Hospital, Seventh Medical Center, PLA general hospital
Beijing, Beijing 100700
ChinaActive - Recruiting
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