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Gene Therapy Trial Report

Summary

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


NCTID NCT06819514 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Fabry Disease
Disease Ontology Term DOID:14499
Compound Name EXG110
Sponsor Guangzhou Jiayin Biotech Ltd
Funder Type Industry
Recruitment Status
Not yet recruiting
Enrollment Count 16 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant GLA
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type proprietary AAV
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed high dose

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2025-01-22
Completion Date 2028-03-15
Last Update 2025-02-11

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 3
Locations China

Regulatory Information


Has US IND False
FDA Designations Orphan Drug Designation
Recent Updates First patient has been dosed in China, company plans to initiate US clinical trials

Resources/Links