Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 5:19 AM
Ignite Modification Date:
2025-12-25 @ 5:19 AM
NCT ID:
NCT05567627
Eligibility Criteria:
Inclusion Criteria:
* 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;
* The patient must be no older than 6 months;
* The patient must be diagnosed with infantile-onset Pompe disease.
Exclusion Criteria:
* Class IV patient based on Modified Ross Heart Failure Classification for Children;
* Aspartate aminotransferase (AST), alanine aminotransferase (ALT) \> 3x upper limit of normal (ULN), alkaline phosphatase (ALP) \> 2x ULN (with the exception of liver abnormalities 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 gastrointestinal symptoms; clinical examination found prolonged prothrombin time, prothrombin activity less than 40%; Neuropsychiatric symptoms, such as restlessness, changes in personality and behavior, lethargy, coma, etc.; Toxic tympanic bowel, ascites, multiple organ dysfunction, etc.; hyperalbuminemia exceeding 171 μmol/L, hypoalbuminemia. Renal failure: creatinine exceeding 110 μmol/L, or glomerular filtration rate less than 100 mL/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 B surface 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 other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study.
Healthy Volunteers:
False
Sex:
ALL
Maximum Age:
6 Months
Study:
NCT05567627
Study Brief:
Protocol Section:
NCT05567627