Objective: To explore the safety and tolerability of different doses of EXG110 with Fabre disease
An open-label, multicenter, single-arm, non-randomized, dose-escalation, and recommended dose-extension clinical design was used to evaluate the safety and efficacy of a single intravenous administration of different doses of EXG110 in patients
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
12
EXG110 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.
Shanghai Children's Medical Center
Shanghai, Shanghai Municipality, China
RECRUITINGChildren's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
RECRUITINGIncidence 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
Time frame: 52 weeks following EXG110 administration
eGFR change from baseline in mL/min/(1.73m^2);
eGFR change from baseline in mL/min/(1.73m\^2)
Time frame: 52 weeks following EXG110 administration
NYHA cardiac function grade changed from baseline;
NYHA cardiac function grade changed from baseline;
Time frame: 52 weeks following EXG110 administration
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
Time frame: 52 weeks following EXG110 administration
Change from baseline in serum AGA activity
Change from baseline in serum AGA activity
Time frame: 52 weeks following EXG110 administration
Change from baseline serum lysoGb3
Change from baseline serum lysoGb3
Time frame: 52 weeks following EXG110 administration
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