This is a single-center, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of CRG003 injection in participants with late onset Pompe disease (LOPD), with a long-term follow-up period of 5 years.
CRG003 (BBM-G102) injection is an adeno-associated virus (AAV) gene therapy product for treating Pompe disease to stably express active GAA enzyme in the liver on a long-term basis after the injection.
Participants voluntarily sign informed consent form;
Clinically diagnosed with LOPD;
Males or females aged ≥ 18 years;
Undergone enzyme replacement treatment (ERT) with recombinant human acid alpha-glucosidase (rhGAA) previously, and has been discontinued for at least four weeks before screening;
Severe cardiomyopathy was defined as left ventricular ejection fraction (LVEF) < 45% or New York Heart Association (NYHA) functional class 3 or above;
Require invasive mechanical ventilation, or rely on noninvasive ventilation during the day;
Intolerance to ERT, prior experience of serious infusion-associated reactions (IARs), prior experience of serious allergic reactions or investigator-assessed intolerance to ERT;
Have received any systemic immunosuppressants (except inhalation or topical use) other than glucocorticoids or investigator-recommended immunosuppressants 30 days prior to screening, and known intolerance to immunosuppressants such as glucocorticoids;
Single group
CRG003 injection
GeneticThe dose of CRG003 will be calculated according to the participant's weight with single intravenous infusion.
Incidence of dose limited toxicities
Incidence of dose limited toxicities (DLTs) as determined by the safety review committee (SRC) within 12 weeks following CRG003 infusion;
Incidence of adverse events and serious adverse events
Incidence of adverse events (AEs) and serious adverse events (SAEs) within 26 weeks and 52 weeks following CRG003 infusion
The change of hepatic enzyme concentration
Alkaline phosphatase (ALP)
Changes from baseline in liver function
Changes from baseline in liver function \[Alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transpeptidase (GGT) and total bilirubin (TBIL)\] within 26 weeks and 52 weeks following CRG003 infusion
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