Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

Korábbi kezelés

• Have a vaccination history within 30 days prior to screening, or have a vaccination plan during the screening period and the main study period;
• Have received gene therapy or used other investigational drugs within four weeks prior to dosing;

Más betegség

• Positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus DNA (HBV-DNA), positive for hepatitis C virus RNA (HCV-RNA), positive for HIV or syphilis;
• Have potential liver diseases;
• Heart failure and severe arrhythmias;
• Severe allergic reactions for enzyme replacement drugs or other medications;
• Acute/chronic infections;
• End-stage renal disease;
• Other conditions that make the participant not eligible for the study according to the investigator.

• The participant's legal guardian fully understands the objectives, nature, methods and potential risks of the study and signs a written informed consent; If the participant is >= 8 years old, the participant must also agree to participate in the study and sign a written informed consent;
• Decreased α-Gal A (α-galactosidase A) and confirmed diagnosis of Fabry Disease by genetic testing;
• Males or females aged ≥7 years and <18 years old;
• Acceptable eGFR (estimated Glomerular Filtration Rate) result in screening period;
• Participants had at least one of the clinical manifestations for Fabry disease;
• Acceptable capsid antibody titers;
• Acceptable anti α-Gal A antibody titers;
• Acceptable laboratory values;
• Participant's legal guardian and participant with good cooperation and compliance;
• Use of reliable contraception methods during the study for adolescence.