The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease
ConditionFabry Disease
Trial statusNot yet recruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorGuangzhou Jiayin Biotech Ltd
About this trial
A phase 1/2, multicenter, open-label, Dose-escalation study to evaluate the safety and efficacy of intravenous EXG110 in patients with Fabry disease
Eligibility criteria
Qualifiers
Age ≥18 years old, 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
Disqualifiers
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) Hypoalbuminemia ≥ grade 2
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
Current or have a history of serious cardiovascular disease and surgical history
Current underlying liver disease or history of liver disease, as assessed by the investigator, that may affect the safety assessment of the product
Trial design
Treatments tested in this trial
- EXG110 Injection
Treatment groups
16 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov