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

NCT ID: NCT06819514

Last Updated: 2025-02-11

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-03-01
• Study Completion Date: 2028-03-15

Brief Summary

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

Detailed Description

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

Conditions

Fabry Disease

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Low dose arm

EXG110 injection, use once by intravenous

Group Type: EXPERIMENTAL

EXG110 Injection

Intervention Type: DRUG

EXG110 Injection is gene therapy for Fabry Disease , uses a proprietary AAV capsid with improved liver and muscle specificity.

High dose arm

EXG110 injection, use once by intravenous

Group Type: EXPERIMENTAL

EXG110 Injection

Intervention Type: DRUG

EXG110 Injection is gene therapy for Fabry Disease , uses a proprietary AAV capsid with improved liver and muscle specificity.

Interventions

EXG110 Injection

EXG110 Injection is gene therapy for Fabry Disease , uses a proprietary AAV capsid with improved liver and muscle specificity.

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Hangzhou Jiayin Biotech Ltd

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Zhihong Liu, MD

Role: PRINCIPAL_INVESTIGATOR

The General Hospital of Eastern Theater Command

Locations

General Hospital of Eastern Theater Command

Nanjing, Jiangsu, China

Children's Hospital Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

First Affiliated Hospital of Zhejiang University

Hangzhou, Zhejiang, China

Countries

China

Central Contacts

Zhihong Liu, MD

Role: CONTACT

chenpl166@163.com

+86 137 0158 9469

Facility Contacts

Peiling Chen, MD

Role: primary

chenpl166@163.com

+86 137 0158 9469

Jianhua Mao, PhD

Role: primary

maojh88@zju.edu.cn

+86 13616819071

Xiaogang Guo, PhD

Role: primary

michelleyan2000@126.com

+86 13867741856

Other Identifiers

EXG110-111

Identifier Type: -

Identifier Source: org_study_id