Actively Recruiting
Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease
Led by The Children's Hospital of Zhejiang University School of Medicine · Updated on 2026-02-27
12
Participants Needed
2
Research Sites
129 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Objective: To explore the safety and tolerability of different doses of EXG110 with Fabre disease
CONDITIONS
Official Title
Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 7 years or older at the time of signing informed consent, male or female
- Clinical symptoms of Fabry disease and genetic diagnosis
- Prior or no prior enzyme replacement therapy treatment
- Renal or cardiac involvement for adults
- Use of effective contraception for subjects of reproductive age from screening until 52 weeks after dosing
- Voluntary participation with informed consent and willingness to comply with study protocol and provide biological samples
You will not qualify if you...
- Elevated liver enzymes (AST or ALT > 1.5 times upper limit of normal)
- Total bilirubin > 1.5 times upper limit of normal
- Alkaline phosphatase > 2 times upper limit of normal
- Low albumin below normal range
- Clinically significant increase in AFP during screening
- Positive hepatitis B surface antigen with high HBV-DNA
- Positive hepatitis C antibody with high HCV-RNA
- Positive syphilis screening and specific antibodies
- Known or positive HIV infection
- Positive anti-AGA antibody or AVT917 (>1:50) levels
- Abnormal complement levels (low C3, high C5b-9) or positive anti-AVT917 IgM
- Current or history of serious cardiovascular disease or surgery
- Current or past liver disease affecting safety assessment
- Kidney disease with decline >5 mL/min/1.73m²/year in adults
- Poorly controlled diabetes (HbA1c ≥ 8%)
- Acute or chronic infections or other diseases increasing risk
- History or current malignant tumors except certain controlled skin or cervical cancers
- Active autoimmune diseases
- Known allergy to study drug components
- History of drug abuse or alcoholism
- Use of systemic immunomodulators within past 6 months
- Recent changes in blood pressure medications affecting proteinuria
- Participation in another investigational drug/device trial within 3 months
- Previous gene therapy treatment
- Receipt or planned receipt of live attenuated vaccines within 12 weeks
- Other clinical conditions deemed exclusionary by investigators
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 2 locations
1
Shanghai Children's Medical Center
Shanghai, Shanghai Municipality, China
Actively Recruiting
2
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Actively Recruiting
Research Team
J
Jianhua Mao, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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