A Phase II Exploratory Study of SR604 Injection Evaluating the Safety and Efficacy in the Treatment of Congenital Coagulation Factor VII Deficiency
An Open-Label, Multicenter, Phase II Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetic Characteristics of SR604 Injection in Patients With Congenital Coagulation Factor VII Deficiency
연구 개요
상태
상태
정황
정황
개입 / 치료
개입 / 치료
연구 유형
연구 유형
등록 (추정된)
등록
단계
단계
- 2 단계
연락처 및 위치
연구 연락처
연구 연락처
- 이름: Research and Development
- 전화번호: 862122130888
- 이메일: hanyu@raas-corp.com
연구 장소
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Tianjin, 중국
- Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
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참여기준
자격 기준
자격 기준
공부할 수 있는 나이
- 성인
- 고령자
건강한 자원 봉사자를 받아들입니다
설명
Inclusion Criteria:
- Age ≥12 years and ≤65 years at the time of signing the informed consent form; both genders eligible;
- Clinically diagnosed with congenital coagulation Factor VII deficiency, with historical or screening FVII activity <10%, and ≥2 treated new-onset bleeding events within 3 months prior to enrollment;
- No active bleeding symptoms prior to first administration of SR604 Injection;
- The subject and/or legal representative and impartial witness have signed the informed consent form, indicating voluntary agreement to participate in this trial, to provide biological samples for testing as required by the protocol, and to comply with the planned study visits;
- Female subjects (post-menarche) must have a negative serum pregnancy test (HCG) during the screening period; subjects with childbearing potential (females post-menarche or males post-spermarche) must agree to use highly effective contraceptive measures throughout the study period.
Exclusion Criteria:
- Known history of hypersensitivity to the study drug formulation or any of its components;
- Intolerance to subcutaneous injection or other local skin abnormalities or dermatoses that may affect drug administration or safety assessment;
Meeting any one of the following criteria during screening:
- Hemoglobin <60 g/L;
- Platelet count <100×10⁹/L;
- Abnormal hepatic or renal function: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥2.5× upper limit of normal (ULN), or total bilirubin ≥1.5× ULN; or serum creatinine (Cr) ≥1.5× ULN;
- Positive for anti-human immunodeficiency virus (HIV) antibodies.
- Any bleeding disorder other than congenital Factor VII deficiency, or other conditions causing significantly abnormal coagulation parameters (e.g., hemophilia A or B, von Willebrand disease, platelet disorders, vitamin K deficiency, etc.);
- Protein C deficiency or Protein S deficiency;
- History of thrombosis, family history of thrombosis, or history of thrombophilia;
- Intracranial hemorrhage within 2 years prior to signing the informed consent form;
- Severe cardiac disease, such as unstable angina, congestive heart failure (New York Heart Association class ≥III), serious arrhythmia (QTc interval >450 ms, corrected by Fridericia formula), uncontrolled hypertension (systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg), etc.;
- Female patients with menstrual abnormalities caused by organic gynecological conditions (e.g., uterine fibroids, endometriosis, adenomyosis, etc.);
- Received Factor VII-containing products within 48 hours prior to first administration of SR604 Injection; received whole blood or plasma transfusion within 2 weeks prior to first administration of SR604 Injection;
- Used any anticoagulants, antifibrinolytics, or agents affecting platelet function (including chemical drugs, biological products, or traditional Chinese medicine), including aspirin, within 1 week prior to screening, or required use of such agents during the treatment period;
- Underwent major surgery (defined as Grade III or IV surgery) within 1 month prior to signing the informed consent form, or planned to undergo surgery during the study period;
- Enrolled in other clinical trials within 1 month prior to signing the informed consent form;
- Miscarriage or pregnancy termination within 3 months prior to signing the informed consent form; pregnant or breastfeeding women;
- Mental illness or significant psychiatric disorder, or incapacity or lack of cognitive ability due to other causes;
Other conditions deemed by the investigator to be unsuitable for enrollment, such as alcoholism, anticipated poor subject compliance preventing completion of dosing and study follow-up, poorly controlled comorbid chronic diseases, or serious systemic diseases.
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공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 해당 없음
- 중재 모델: 병렬 할당
- 마스킹: 없음(오픈 라벨)
팔의 수
무기와 개입
참가자 그룹 / 팔참가자 그룹 / 팔 |
개입 / 치료개입 / 치료 |
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실험적: SR604:Multiple-dose exploratory efficacy trial consists of 2 cohorts
Participants with FVII deficiency will receive SR604 dose 1/2 as multiple SC injections every 4-weeks
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SR604는 SC 주사로 투여됩니다.
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연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
기간 |
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Treated overall Annualized Bleeding Rate (ABR)
기간: Through 28 weeks of treatment.
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Through 28 weeks of treatment.
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2차 결과 측정
2차 결과 측정
결과 측정 |
기간 |
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Treated spontaneous annualized bleeding rate
기간: over 28 weeks of treatment
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over 28 weeks of treatment
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Treated overall annualized joint bleeding rate
기간: over 28 weeks of treatment
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over 28 weeks of treatment
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Overall annualized bleeding rate including treated and untreated bleeding events
기간: over 28 weeks of treatment,
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over 28 weeks of treatment,
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Annualized menorrhagia bleeding rate (menstruating females only)
기간: over 28 weeks of treatment
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over 28 weeks of treatment
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Change in PBAC score (menstruating females only);
기간: From baseline over 28 weeks of treatment
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From baseline over 28 weeks of treatment
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Change in EQ-5D-5L health index score
기간: From pre-treatment over 28 weeks of treatment
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From pre-treatment over 28 weeks of treatment
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Change in EQ-VAS score
기간: From baseline over 28 weeks of treatment;
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From baseline over 28 weeks of treatment;
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Subject overall satisfaction score with treatment efficacy
기간: over 28 weeks of treatment
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over 28 weeks of treatment
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기타 결과 측정
기타 결과 측정
결과 측정 |
기간 |
|---|---|
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Single-dose pharmacokinetic (PK) parameters:Time to Peak Plasma Concentration (Tmax)
기간: over 28 weeks of treatment
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over 28 weeks of treatment
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Single-dose pharmacokinetic (PK) parameters:Peak Plasma Concentration (Cmax)
기간: over 28 weeks of treatment
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over 28 weeks of treatment
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Single-dose pharmacokinetic (PK) parameters:Area Under the Concentration-Time Curve from Zero to Last Quantifiable Time Point (AUC0-t)
기간: over 28 weeks of treatment
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over 28 weeks of treatment
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Multiple-dose pharmacokinetic parameters-Time to Peak Plasma Concentration (Tmax)
기간: over 28 weeks of treatment
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over 28 weeks of treatment
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Multiple-dose pharmacokinetic parameters-Peak Plasma Concentration (Cmax)
기간: Over 28 weeks of treatment
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Over 28 weeks of treatment
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Safety and Immunogenicity:Incidence of AEs/SAEs/AESI,
기간: over 28 weeks of treatment
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over 28 weeks of treatment
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Incidence of drug-related AEs/SAEs/AESIs
기간: over 28 weeks of treatment
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over 28 weeks of treatment
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Safety and Immunogenicity: Anti-drug antibody (ADA) and neutralizing antibody (NAb) - number of subjects and incidence rate.
기간: over 28 weeks of treatment
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over 28 weeks of treatment
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공동 작업자 및 조사자
연구 기록 날짜
연구 주요 날짜
연구 시작 (추정된)
연구 시작
기본 완료 (추정된)
기본 완료
연구 완료 (추정된)
연구 완료
연구 등록 날짜
최초 제출
최초 제출
QC 기준을 충족하는 최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
처음 게시됨
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
마지막 업데이트 게시됨
QC 기준을 충족하는 마지막 업데이트 제출
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
기타 연구 ID 번호
- LS-SR604-VII-II01
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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