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임상시험 NCT06539624은(는) 파브리병에 대해 모집중 상태입니다. 모든 세부 정보를 보려면 임상시험 레이더 카드 뷰와 AI 발견 도구를 확인하거나 여기에서 무엇이든 물어보세요. | ||
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카드 뷰
Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease 12
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임상시험 NCT06539624은(는) 파브리병에 대해 알아보는 중재연구입니다. 현재 상태는 모집중이며, 연구는 2024년 10월 16일에 시작되어 12명의 참여자를 모집하고 있습니다. The Children's Hospital of Zhejiang University School of Medicine이(가) 진행하며, 2027년 4월 9일까지 완료될 예정입니다. ClinicalTrials.gov의 가장 최근 정보는 2026년 2월 27일에 갱신되었습니다.
간단한 개요
Objective: To explore the safety and tolerability of different doses of EXG110 with Fabre disease
상세한 설명
An open-label, multicenter, single-arm, non-randomized, dose-escalation, and recommended dose-extension clinical design was used to evaluate the safety and efficacy of a single intravenous administration of different doses of EXG110 in patients
공식 제목
A Multicenter, Non-randomized, Open-label, Dose-finding Study to Evaluate the Safety and Preliminary Efficacy of Gene Therapy With EXG110 in Subjects With Fabry Disease
질환명
파브리병기타 연구 식별자
- EXG110-011
NCT 번호
실제 연구 시작일
2024-10-16
최신 업데이트 게시
2026-02-27
예상 연구 완료일
2027-04-09
계획된 등록 인원
12
연구종류
중재연구
단계/상
해당 없음
상태
모집중
주요 목적
치료
설계 할당
비랜덤화 배정
중재 모델
순차설계
맹검 (마스킹)
없음 (오픈 라벨)
시험군 / 개입
| 참가자 그룹/시험군 | 개입/치료 |
|---|---|
실험적Dose escalation-Cohort 1 Genetic : EXG110 | EXG110 injection EXG110 is a recombinant adeno-associated virus (rAAV) that not only significantly increases plasma AGA activity, but is also highly expressed in target organs such as the heart and kidneys.EXG110 will be administered in a single dose by intravenous infusion. |
실험적Dose escalation-Cohort 2 Genetic : EXG110 | EXG110 injection EXG110 is a recombinant adeno-associated virus (rAAV) that not only significantly increases plasma AGA activity, but is also highly expressed in target organs such as the heart and kidneys.EXG110 will be administered in a single dose by intravenous infusion. |
실험적Dose escalation-Cohort 3 Genetic : EXG110 | EXG110 injection EXG110 is a recombinant adeno-associated virus (rAAV) that not only significantly increases plasma AGA activity, but is also highly expressed in target organs such as the heart and kidneys.EXG110 will be administered in a single dose by intravenous infusion. |
실험적Dose escalation-Cohort 4 Genetic : EXG110 | EXG110 injection EXG110 is a recombinant adeno-associated virus (rAAV) that not only significantly increases plasma AGA activity, but is also highly expressed in target organs such as the heart and kidneys.EXG110 will be administered in a single dose by intravenous infusion. |
주요결과변수
이차결과변수
| 결과변수 | 측정값 설명 | 시간 범위 |
|---|---|---|
Incidence and severity of adverse events | Safety and tolerability of EXG110 following a single IV dose, as assessed by incidence and severity of adverse events, serious adverse events and dose limiting toxicities, including clinically significant changes from baseline to scheduled time points in safety parameters | 52 weeks following EXG110 administration |
| 결과변수 | 측정값 설명 | 시간 범위 |
|---|---|---|
eGFR change from baseline in mL/min/(1.73m^2); | eGFR change from baseline in mL/min/(1.73m\^2) | 52 weeks following EXG110 administration |
NYHA cardiac function grade changed from baseline; | NYHA cardiac function grade changed from baseline; | 52 weeks following EXG110 administration |
Changed from baseline: region and area in mm^2 of skin angiokeratoma The number of Gb3 deposition in skin biopsy under the microscope | Changed from baseline: region and area in mm\^2 of skin angiokeratoma The number of Gb3 deposition in skin biopsy under the microscope | 52 weeks following EXG110 administration |
Change from baseline in serum AGA activity | Change from baseline in serum AGA activity | 52 weeks following EXG110 administration |
Change from baseline serum lysoGb3 | Change from baseline serum lysoGb3 | 52 weeks following EXG110 administration |
참여 도우미
적격성 기준
연령대
어린이, 성인, 노인
최소 연령
7 Years
참여 가능한 성별
전체
- At the time of signing the informed consent, age ≥7, 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 (adults only)
- All subjects of reproductive age voluntarily took effective contraception and prohibited sperm donation from entering the screening period until 52 weeks after dosing (main study period)
- The subjects voluntarily participate and are fully informed, fully understand the research, can comply with the requirements of the research protocol, and are willing to complete the research as planned, and voluntarily provide biological samples for testing according to the requirements of the protocol
- 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) Albumin < lower limit of normal (LLN)
- There was a clinically significant increase in AFP during the screening period
- 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
- AVT917 (>1:50), anti-AGA antibody positive(>1:2560)
- C3 lower than the normal range, C5b-9 higher than the normal range, anti-AVT917 IgM 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 drug
- Renal disease in adult and the slope of kidney >5 mL/min/1.73m²/year
- Subjects with poorly controlled diabetes after drug treatment (e.g., HbA1c≥8%);
- Acute/chronic infection or other chronic disease that the investigator determines will increase the risk of participants participating in the study
- Patients 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);
- Have malignancy cancer
- Patients 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
- Patients with a history of drug use or drug abuse or alcoholism
- Use of systemic (intravenous or oral) immunomodulators within the past 6 months or currently
- Initiation of treatment with blood pressure lowering drugs that affect proteinuria levels (such as angiotensin-converting enzyme inhibitors, angiotensin-receptor blockers, or angiotensin-receptor/enkephalin inhibitors) within 4 weeks prior to screening, or changes in the therapeutic dose of these drugs within 4 weeks prior to screening;
- Has received, or is 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 (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 felt needed to be ruled out
The Children's Hospital of Zhejiang University School of Medicine연구 책임자
Mao Jianhua, 책임연구자, Vice President of the hospital, The Children's Hospital of Zhejiang University School of Medicine
연구 대표 연락처
연락처: Jianhua Mao, PhD, 13516819071, [email protected]
2 1개국에 임상시험 장소
Shanghai Municipality
Shanghai Children's Medical Center, Shanghai, Shanghai Municipality, China
Lei Yin, PhD, 연락처, 13641673203, [email protected]
Lei Yin, PhD, 책임연구자
모집중
Zhejiang
Children's Hospital, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
Mao Jianhua, MD, 연락처, 13616819071, [email protected]
모집중