Clinical trial NCT06819514: The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease
Official title: A Phase 1/2, Multicenter, Open-label,Dose-escalation Study to Evaluate the Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Fabry Disease
Lead sponsor: Hangzhou Jiayin Biotech Ltd
Sponsor class: INDUSTRY
First posted: 2025-02-11
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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

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

Primary outcomes:
measure: Phase 1: To evaluate the safety and tolerability of EXG110 following a single intravenous infusion
timeFrame: 52 weeks
measure: Phase 2: The changes from the baseline of kidney function
timeFrame: 6 months
measure: Phase 2: The changes from the baseline of cardiac function
timeFrame: 6 months
measure: Phase 2: The changes from the baseline of pain
timeFrame: 6 months
measure: Phase 2: The changes from the baseline of gastrointestinal function
timeFrame: 6 months
Secondary outcomes:
measure: eGFR
timeFrame: 52 weeks
measure: Cardiac function
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Nanjing
country: China
facility: General Hospital of Eastern Theater Command
state: Jiangsu
city: Hangzhou
country: China
facility: Children's Hospital Zhejiang University School of Medicine
state: Zhejiang
city: Hangzhou
country: China
facility: First Affiliated Hospital of Zhejiang University
state: Zhejiang
Study officials:
affiliation: The General Hospital of Eastern Theater Command
name: Zhihong Liu, MD
role: PRINCIPAL_INVESTIGATOR
References: