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An Open-label, Multicenter Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetic/Pharmacodynamic (PK/PD) Characteristics of SR604 Injection in Patients With Hemophilia A/B and Congenital Factor VII Deficiency

2026년 6월 8일 업데이트: Shanghai RAAS Blood Products Co., Ltd.
The purpose of this study is to evaluate the safety, tolerability, immunogenicity , PK, and PD of a single dose of SR604 in participants with Hemophilia A or Hemophilia B, with or without inhibitors (Part A)and to evaluate the safety, PK, PD, and efficacy of multiple doses of SR604 in participants with Hemophilia A or Hemophilia B, or Factor VII (FVII) deficiency, with or without inhibitors (Part B and Part C).

연구 개요

연구 유형

중재적

등록 (추정된)

76

단계

  • 2 단계
  • 1단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 연락처

연구 장소

      • Changsha, 중국
        • 모병
        • Xiangya Hospital of Central South University
      • Hefei, 중국
        • 모병
        • The First Affiliated Hospital of University of Science and Technology of China
      • Jinan, 중국
        • 모병
        • Jinan Central Hospital
      • Lanzhou, 중국
        • 모병
        • The First Hospital of Lanzhou University
      • Shanghai, 중국
        • 완전한
        • Ruijin Hospital Shanghai Jiaotong University School of Medicine
      • Taiyuan, 중국
        • 모병
        • The Second Hospital of Shanxi Medical University
      • Tianjin, 중국
        • 완전한
        • Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
      • Xi'an, 중국
        • 모병
        • Xian Central Hospital
      • Zhengzhou, 중국
        • 모병
        • Zhengzhou People's Hospital

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

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  • 고령자

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아니

설명

Inclusion Criteria:

  1. Age ≥18 years and ≤65 years at the time of signing informed consent, regardless of sex;
  2. Clinically diagnosed with Hemophilia A or B or congenital coagulation Factor VII deficiency, and must meet the following criteria:

    1. Hemophilia A or B patients with historical or screening FVIII activity level <1% or FIX activity level ≤2%; Note: Hemophilia A or B patients with or without inhibitors may be enrolled. For patients without inhibitors (inhibitor titer <0.6 BU/mL), they must have previously received coagulation factor treatment with exposure days (EDs) >50 days.
    2. Congenital coagulation Factor VII deficiency patients with historical or screening FVII activity <10%;
  3. Part A only: Received on-demand treatment with FVIII, FIX, recombinant human coagulation Factor VIIa (rFVIIa), or PCC for bleeding events within 1 month prior to screening;
  4. Part B/Part C only: Accessible bleeding and treatment records (factor replacement or bypassing agent therapy) for at least 3 months prior to enrollment. Hemophilia A or B patients must have received on-demand treatment with ≥3 treated de novo bleeding episodes within 3 months prior to enrollment. Congenital coagulation Factor VII deficiency patients must have ≥2 treated de novo bleeding episodes within 3 months prior to enrollment;
  5. No active bleeding symptoms prior to first dosing;
  6. The subject or a legally acceptable representative has a full understanding of and can comply with the protocol requirements, has the willingness to complete the study as planned, and voluntarily agrees to provide biological samples for testing as required by the protocol;
  7. The subject is able to understand the procedures and methods of this clinical trial, has been fully informed, and voluntarily participates in the trial by personally signing the informed consent form.

Exclusion Criteria:

  1. Subjects with a known history of hypersensitivity to the investigational medicinal product or any of its components;
  2. Intolerance to subcutaneous injection or presence of other local skin abnormalities or dermatological conditions that may affect administration and safety assessment;
  3. Subjects meeting any of the following criteria at screening:

    1. Hemoglobin <60 g/L;
    2. Platelet count <100 × 10^9/L;
    3. Hepatic or renal impairment: 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;
  4. Positive result(s) for hepatitis B virus surface antigen (HBsAg), anti-human immunodeficiency virus (HIV) antibody, and/or Treponema pallidum-specific antibody;
  5. Clinically diagnosed with active hepatitis C;
  6. Any other bleeding disorder or any other disease causing significant coagulation abnormalities (e.g., platelet disorders, vitamin K deficiency, etc.) other than Hemophilia A or B and congenital coagulation Factor VII deficiency;
  7. Protein C deficiency or protein S deficiency;
  8. History of or current thrombosis, family history of thrombosis, or history of thrombophilia prior to signing informed consent;
  9. Intracranial hemorrhage due to Hemophilia A or B or congenital coagulation Factor VII deficiency within 2 years prior to screening;
  10. Severe cardiac disease, such as unstable angina, congestive heart failure (New York Heart Association Class ≥III), severe arrhythmia (QTc interval >450 ms, corrected by Fridericia's formula), or uncontrolled hypertension (systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥95 mmHg);
  11. Received recombinant human coagulation Factor VIIa (rFVIIa) within 48 hours prior to first dosing; received any FVIII-containing product within 72 hours prior to first dosing; received any FIX-containing product within 96 hours prior to first dosing; long-acting products of the above have not completed a washout of 5 half-lives;
  12. Used or requires use of any anticoagulant, antifibrinolytic agent, or chemical drug, biological product, or traditional Chinese medicine affecting platelet function, including nonsteroidal anti-inflammatory drugs (NSAIDs) such as aspirin, within 1 week prior to first dosing or during the trial;
  13. Received whole blood or plasma therapy within 2 weeks prior to first dosing;
  14. Received emicizumab treatment within 6 months prior to first dosing;
  15. Received or planned to receive vaccination within 4 weeks prior to first dosing or during the trial;
  16. Underwent major surgery (e.g., orthopedic surgery, abdominal surgery) within 1 month prior to first dosing, or planned to undergo surgery during the study;
  17. Enrolled in another clinical trial within 1 month prior to first dosing;
  18. History of drug abuse or alcoholism (alcoholism criteria: long-term drinking history exceeding 5 years, equivalent to ethanol intake ≥40 g/day, or heavy drinking within 2 weeks, equivalent to ethanol intake >80 g/day. Ethanol amount (g) conversion formula = alcohol volume (mL) × ethanol content (%) × 0.8);
  19. Psychiatric illness or significant mental impairment, or incapacity or lack of cognitive ability due to other reasons;
  20. Plans to have children or donate sperm during the entire trial period up to 6 months after the last dose, or unwilling to use effective physical contraceptive measures (e.g., condoms);
  21. Subjects with clinically significant disease or other conditions that the investigator considers unsuitable for participation in the clinical trial (e.g., the patient cannot benefit from the clinical trial);
  22. Subjects deemed by the investigator to have poor compliance, rendering efficacy evaluation impossible or with low likelihood of completing the planned treatment course and follow-up.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 무작위
  • 중재 모델: 병렬 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: Part A: Dose escalation trial consists of 6 cohorts
Participants with Hemophilia A or Hemophilia B will receive single subcutaneous (SC) dose from dose 1 to dose 6
SR604는 SC 주사로 투여됩니다.
SR604 will be administered as SC injection
실험적: Part B: Multiple-dose exploratory efficacy trial consists of 2 cohorts
Participants with Hemophilia A or Hemophilia B or FVII deficiency will receive SR604 dose 1/2 as multiple SC injections every 2-weeks
SR604는 SC 주사로 투여됩니다.
SR604 will be administered as SC injection
실험적: Part C: Multiple-dose exploratory efficacy trial consists of 3 cohorts
Participants with Hemophilia A or Hemophilia B will receive SR604 dose 5 as multiple SC injections every 4-weeks/6-weeks/8-weeks.
SR604는 SC 주사로 투여됩니다.
SR604 will be administered as SC injection

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Part A: Incidence of AEs/SAEs/AESI
기간: Part A: From Baseline (Day 1) up to Day 85
Assessed through clinical signs and symptoms, vital signs, physical examination, laboratory tests (complete blood count, urinalysis, and blood biochemistry), coagulation function [PT, TT, INR, FIB, APTT, D-dimer], FDP, 12-lead electrocardiogram, injection site reactions, hypersensitivity/allergic reactions, thrombotic events, etc.;Safety and Immunogenicity of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
Part A: From Baseline (Day 1) up to Day 85
PartA: Incidence of drug-related AEs/SAEs/AESIs
기간: Part A: From Baseline (Day 1) up to Day 85
Safety and Immunogenicity of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
Part A: From Baseline (Day 1) up to Day 85
Part A: Number and incidence of patients with anti-drug antibodies (ADA) and neutralizing antibodies
기간: Part A: From Baseline (Day 1) up to Day 85
Safety and Immunogenicity of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
Part A: From Baseline (Day 1) up to Day 85
Part B/ Part C:Treated total annualized bleeding rate (ABR)
기간: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393

2차 결과 측정

결과 측정
측정값 설명
기간
Part A:Single-dose pharmacokinetic (PK) parameters:Peak Plasma Concentration (Cmax)
기간: Part A: From Baseline (Day 1) up to Day 85
PK of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
Part A: From Baseline (Day 1) up to Day 85
Part B/ Part C:Treated spontaneous annualized bleeding rate
기간: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B/ Part C:Treated total annualized joint bleeding rate
기간: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B/ Part C:Treated annualized menorrhagia bleeding rate (applicable only to reproductive-age female patients with congenital FVII deficiency and active menstruation)
기간: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B/ Part C:Change from baseline in Hemophilia Joint Health Score (HJHS) (for hemophilia A/B patients)
기간: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B/ Part C:Change from baseline in EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) score
기간: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B/ Part C:Multiple-dose pharmacokinetic parameters-Time to Peak Plasma Concentration (Tmax)
기간: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B/ Part C:Safety and Immunogenicity
기간: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Incidence of AEs/SAEs/AESI, Incidence of drug-related AEs/SAEs/AESIs, Number and incidence of patients with anti-drug antibodies (ADA) and neutralizing antibodies
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part A:Single-dose pharmacokinetic (PK) parameters:Time to Peak Plasma Concentration (Tmax)
기간: Part A: From Baseline (Day 1) up to Day 85
PK of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
Part A: From Baseline (Day 1) up to Day 85
Part A:Single-dose pharmacokinetic (PK) parameters:Area Under the Concentration-Time Curve from Zero to Last Quantifiable Time Point (AUC0-t)
기간: Part A: From Baseline (Day 1) up to Day 85
PK of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
Part A: From Baseline (Day 1) up to Day 85
Part B/ Part C:Multiple-dose pharmacokinetic parameters-Peak Plasma Concentration (Cmax)
기간: Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393

기타 결과 측정

결과 측정
기간
Part A, part B and part C: Pharmacodynamic parameters-protein C
기간: Part A: From Baseline (Day 1) up to Day 85;Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part A: From Baseline (Day 1) up to Day 85;Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part A, part B and part C: Pharmacodynamic parameters-prothrombin time (PT)
기간: Part A: From Baseline (Day 1) up to Day 85;Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
Part A: From Baseline (Day 1) up to Day 85;Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2024년 5월 31일

기본 완료 (추정된)

2026년 12월 31일

연구 완료 (추정된)

2026년 12월 31일

연구 등록 날짜

최초 제출

2026년 5월 26일

QC 기준을 충족하는 최초 제출

2026년 6월 8일

처음 게시됨 (실제)

2026년 6월 12일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 6월 12일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 6월 8일

마지막으로 확인됨

2026년 5월 1일

추가 정보

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아니요

약물 및 장치 정보, 연구 문서

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아니

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

혈우병 A에 대한 임상 시험

SR604에 대한 임상 시험

구독하다