Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug

Korábbi kezelés

• Patient who has AAV9 neutralizing antibody titer ≥ 1:100；
• Patient who has received enzyme replacement therapy (ERT) more than twice;
• 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.

Más betegség

• 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;

Laborértékek

• Left ventricle ejection fraction (LVEF) < 40%;
• Patient who has laboratory abnormalities of: creatinine > Upper Limit of Normal (ULN), hemoglobin < 90 g/L;

Egyéb

• 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 with congenital organ absence;

• 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.