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Recruiting NCT06539624

Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease

Trial Parameters

Condition Fabry Disease
Sponsor The Children's Hospital of Zhejiang University School of Medicine
Study Type INTERVENTIONAL
Phase N/A
Enrollment 12
Sex ALL
Min Age 7 Years
Max Age N/A
Start Date 2024-10-16
Completion 2027-04-09
Interventions
EXG110 injection

Brief Summary

Objective: To explore the safety and tolerability of different doses of EXG110 with Fabre disease

Eligibility Criteria

Inclusion Criteria: 1. At the time of signing the informed consent, age ≥7, male or female 2. Clinical symptoms (at least one Fabry disease related symptom) and genetic diagnosis of Fabry disease, 3. Prior or no prior ERT treatment 4. Have renal or cardiac involvement (adults only) 5. All subjects of reproductive age voluntarily took effective contraception and prohibited sperm donation from entering the screening period until 52 weeks after dosing (main study period) 6. The subjects voluntarily participate and are fully informed, fully understand the research, can comply with the requirements of the research protocol, and are willing to complete the research as planned, and voluntarily provide biological samples for testing according to the requirements of the protocol Exclusion Criteria: 1. 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) Al

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