The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease

Hangzhou Jiayin Biotech

Status and phase: Not yet enrolling; Phase 2; Phase 1

Conditions: Fabry Disease

Treatments: Drug: EXG110 Injection

Study type: Interventional

Funder types: Industry

Identifiers

NCT06819514

EXG110-111

Details and patient eligibility

About

A phase 1/2, multicenter, open-label, Dose-escalation study to evaluate the safety and efficacy of intravenous EXG110 in patients with Fabry disease

Full description

Phase 1: Dose -escalation，2 Groups Phase 2: Dose- expansion，1 Group

Enrollment: 16 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Age ≥18 years old, 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
5. The participant voluntarily participate and are fully informed, fully understood the study, can comply with the requirements of the protocol, 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) Alkaline phosphatase > 2× upper limit of normal (ULN);d) Hypoalbuminemia ≥ grade 2
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
3. Current or have a history of serious cardiovascular disease and surgical history
4. Current underlying liver disease or history of liver disease, as assessed by the investigator, that may affect the safety assessment of the product
5. Acute/chronic infection or other chronic disease that the investigator evaluated will increase the risk of participants in the study
6. Those who have a history of epilepsy, mental illness (such as schizophrenia, depression, mania or anxiety, etc.) or obvious mental disorders, and are incapacitated or cognitively incapacitated due to other reasons.
7. Participant with a history of malignant tumor or currently suffering from any malignant tumor (except for the following tumor diseases: skin basal cell carcinoma, cervical carcinoma in situ, breast carcinoma in situ , skin squamous cell carcinoma has been controlled after treatment);
8. Participant with active autoimmune diseases (such as rheumatoid arthritis, systemic lupus erythematosus, multiple sclerosis, immune vasculitis, inflammatory bowel disease, etc.);
9. known history of allergy to the components of the investigational products
10. Participant with a history of drug use or drug abuse or alcoholism
11. Has received, or currently receiving, a clinical trial of another investigational drug/medical device or treatment (other than vitamins and minerals) within 3 months prior to signing the informed consent form (or within 5 half-lives of the investigational drug, whichever is longer)
12. Previous treatment with gene therapy products
13. Those who had received live attenuated vaccine/vaccine within 12 weeks prior to screening or planned to receive it during the study
14. Other clinical conditions that the investigators evaluated needed to be excluded

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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16 participants in 2 patient groups

Low dose arm

Experimental group

Description:

EXG110 injection, use once by intravenous

Treatment:

Drug: EXG110 Injection

High dose arm

Experimental group

Description:

EXG110 injection, use once by intravenous

Treatment:

Drug: EXG110 Injection