Objective: To explore the safety and tolerability of different doses of EXG110 with Fabre disease
At the time of signing the informed consent, age ≥7, male or female
Clinical symptoms (at least one Fabry disease related symptom) and genetic diagnosis of Fabry disease,
Prior or no prior ERT treatment
Have renal or cardiac involvement (adults only)
Screening period laboratory test results: a) aspartate aminotransferase or alanine aminotransferase > 1.5× upper limit of normal (ULN);b) Total bilirubin > 1.5× upper limit of normal (ULN);c) Alkaline phosphatase > 2× upper limit of normal (ULN);d) Albumin < lower limit of normal (LLN)
There was a clinically significant increase in AFP during the screening period
Serum virology test: a) Hepatitis B: Hepatitis B virus surface antigen (HBsAg) positive, and hepatitis B virus-deoxyribonucleic acid (HBV-DNA) higher than the upper limit of normal detection;b) Hepatitis C: if the hepatitis C virus (HCV) antibody is positive, and the hepatitis C virus-ribonucleic acid (HCV-RNA) is higher than the upper limit of normal test value;c) Syphilis: positive for syphilis screening (Tp-Ab) and positive for syphile-specific antibodies;d) HIV: Known human immunodeficiency virus (HIV) positive history or HIV screening positive
AVT917 (>1:50), anti-AGA antibody positive(>1:2560)
Sequential
EXG110 injection
GeneticEXG110 is a recombinant adeno-associated virus (rAAV) that not only significantly increases plasma AGA activity, but is also highly expressed in target organs such as the heart and kidneys.EXG110 will be administered in a single dose by intravenous infusion.
Incidence and severity of adverse events
Safety and tolerability of EXG110 following a single IV dose, as assessed by incidence and severity of adverse events, serious adverse events and dose limiting toxicities, including clinically significant changes from baseline to scheduled time points in safety parameters
eGFR change from baseline in mL/min/(1.73m^2);
eGFR change from baseline in mL/min/(1.73m\^2)
NYHA cardiac function grade changed from baseline;
NYHA cardiac function grade changed from baseline;
Changed from baseline: region and area in mm^2 of skin angiokeratoma The number of Gb3 deposition in skin biopsy under the microscope
Changed from baseline: region and area in mm\^2 of skin angiokeratoma The number of Gb3 deposition in skin biopsy under the microscope
Change from baseline in serum AGA activity
Change from baseline in serum AGA activity
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