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

Официальное название

A Phase 1/2, Multicenter, Open-label,Dose-escalation Study to Evaluate the Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease

Обзор

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

Подробное описание

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

Вмешательства

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

Первичные конечные точки

• Phase 1: To evaluate the safety and tolerability of EXG110 following a single intravenous infusion [Срок оценки: 52 weeks]
• Phase 2: The changes from the baseline of kidney function [Срок оценки: 6 months]
• Phase 2: The changes from the baseline of cardiac function [Срок оценки: 6 months]
• Phase 2: The changes from the baseline of pain [Срок оценки: 6 months]
• Phase 2: The changes from the baseline of gastrointestinal function [Срок оценки: 6 months]

Вторичные конечные точки (2)

• eGFR [Срок оценки: 52 weeks]
• Cardiac function [Срок оценки: 52 weeks]

Критерии участия

Критерии включения

• 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
• 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

Критерии исключения

• 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
• Acute/chronic infection or other chronic disease that the investigator evaluated will increase the risk of participants in the study
• 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.
• 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);
• Participant with active autoimmune diseases (such as rheumatoid arthritis, systemic lupus erythematosus, multiple sclerosis, immune vasculitis, inflammatory bowel disease, etc.);
• known history of allergy to the components of the investigational products
• Participant with a history of drug use or drug abuse or alcoholism
• 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)
• Previous treatment with gene therapy products
• Those who had received live attenuated vaccine/vaccine within 12 weeks prior to screening or planned to receive it during the study
• Other clinical conditions that the investigators evaluated needed to be excluded

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Последовательный дизайн

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 3 центра

• General Hospital of Eastern Theater Command — Нанкин
• Children's Hospital Zhejiang University School of Medicine — Ханчжоу
• First Affiliated Hospital of Zhejiang University — Ханчжоу

Идентификаторы

NCT: NCT06819514 · EXG110-111

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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