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
2026年6月8日 更新者: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).
研究概览
研究类型
介入性
注册 (估计的)
76
阶段
- 阶段2
- 阶段1
联系人和位置
本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。
学习联系方式
- 姓名:Research and Development
- 电话号码:862122130888
- 邮箱:hanyu@raas-corp.com
学习地点
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Changsha、中国
- 招聘中
- Xiangya Hospital of Central South University
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Hefei、中国
- 招聘中
- The First Affiliated Hospital of University of Science and Technology of China
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Jinan、中国
- 招聘中
- Jinan Central Hospital
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Lanzhou、中国
- 招聘中
- The First Hospital of Lanzhou University
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Shanghai、中国
- 完全的
- Ruijin Hospital Shanghai Jiaotong University School of Medicine
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Taiyuan、中国
- 招聘中
- The Second Hospital of Shanxi Medical University
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Tianjin、中国
- 完全的
- Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
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Xi'an、中国
- 招聘中
- Xian Central Hospital
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Zhengzhou、中国
- 招聘中
- Zhengzhou People's Hospital
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参与标准
研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
不
描述
Inclusion Criteria:
- Age ≥18 years and ≤65 years at the time of signing informed consent, regardless of sex;
Clinically diagnosed with Hemophilia A or B or congenital coagulation Factor VII deficiency, and must meet the following criteria:
- 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.
- Congenital coagulation Factor VII deficiency patients with historical or screening FVII activity <10%;
- 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;
- 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;
- No active bleeding symptoms prior to first dosing;
- 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;
- 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:
- Subjects with a known history of hypersensitivity to the investigational medicinal product or any of its components;
- Intolerance to subcutaneous injection or presence of other local skin abnormalities or dermatological conditions that may affect administration and safety assessment;
Subjects meeting any of the following criteria at screening:
- Hemoglobin <60 g/L;
- Platelet count <100 × 10^9/L;
- 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;
- Positive result(s) for hepatitis B virus surface antigen (HBsAg), anti-human immunodeficiency virus (HIV) antibody, and/or Treponema pallidum-specific antibody;
- Clinically diagnosed with active hepatitis C;
- 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;
- Protein C deficiency or protein S deficiency;
- History of or current thrombosis, family history of thrombosis, or history of thrombophilia prior to signing informed consent;
- Intracranial hemorrhage due to Hemophilia A or B or congenital coagulation Factor VII deficiency within 2 years prior to screening;
- 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);
- 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;
- 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;
- Received whole blood or plasma therapy within 2 weeks prior to first dosing;
- Received emicizumab treatment within 6 months prior to first dosing;
- Received or planned to receive vaccination within 4 weeks prior to first dosing or during the trial;
- Underwent major surgery (e.g., orthopedic surgery, abdominal surgery) within 1 month prior to first dosing, or planned to undergo surgery during the study;
- Enrolled in another clinical trial within 1 month prior to first dosing;
- 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);
- Psychiatric illness or significant mental impairment, or incapacity or lack of cognitive ability due to other reasons;
- 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);
- 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);
- 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.
学习计划
本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:随机化
- 介入模型:并行分配
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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实验性的: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
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SR604 将作为 SC 注射给药。
SR604 will be administered as SC injection
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实验性的: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
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SR604 将作为 SC 注射给药。
SR604 will be administered as SC injection
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实验性的: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.
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SR604 将作为 SC 注射给药。
SR604 will be administered as SC injection
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研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Part A: Incidence of AEs/SAEs/AESI
大体时间:Part A: From Baseline (Day 1) up to Day 85
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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.
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Part A: From Baseline (Day 1) up to Day 85
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PartA: Incidence of drug-related AEs/SAEs/AESIs
大体时间:Part A: From Baseline (Day 1) up to Day 85
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Safety and Immunogenicity of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
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Part A: From Baseline (Day 1) up to Day 85
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Part A: Number and incidence of patients with anti-drug antibodies (ADA) and neutralizing antibodies
大体时间:Part A: From Baseline (Day 1) up to Day 85
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Safety and Immunogenicity of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
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Part A: From Baseline (Day 1) up to Day 85
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Part B/ Part C:Treated total annualized bleeding rate (ABR)
大体时间:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Part A:Single-dose pharmacokinetic (PK) parameters:Peak Plasma Concentration (Cmax)
大体时间:Part A: From Baseline (Day 1) up to Day 85
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PK of a single ascending SC dose of SR604 inparticipants with Hemophilia A or Hemophilia B will be evaluated.
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Part A: From Baseline (Day 1) up to Day 85
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Part B/ Part C:Treated spontaneous annualized bleeding rate
大体时间:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B/ Part C:Treated total annualized joint bleeding rate
大体时间:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B/ Part C:Treated annualized menorrhagia bleeding rate (applicable only to reproductive-age female patients with congenital FVII deficiency and active menstruation)
大体时间:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B/ Part C:Change from baseline in Hemophilia Joint Health Score (HJHS) (for hemophilia A/B patients)
大体时间:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B/ Part C:Change from baseline in EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) score
大体时间:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B/ Part C:Multiple-dose pharmacokinetic parameters-Time to Peak Plasma Concentration (Tmax)
大体时间:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B/ Part C:Safety and Immunogenicity
大体时间:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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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
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part A:Single-dose pharmacokinetic (PK) parameters:Time to Peak Plasma Concentration (Tmax)
大体时间: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
|
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Part A:Single-dose pharmacokinetic (PK) parameters:Area Under the Concentration-Time Curve from Zero to Last Quantifiable Time Point (AUC0-t)
大体时间:Part A: From Baseline (Day 1) up to Day 85
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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
|
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Part B/ Part C:Multiple-dose pharmacokinetic parameters-Peak Plasma Concentration (Cmax)
大体时间:Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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Part B: From Baseline (Day 1) up to Day 211;Part C: From baseline (Day 1) up to Day 393
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其他结果措施
结果测量 |
大体时间 |
|---|---|
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Part A, part B and part C: Pharmacodynamic parameters-protein C
大体时间: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
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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
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Part A, part B and part C: Pharmacodynamic parameters-prothrombin time (PT)
大体时间: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
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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
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (实际的)
2024年5月31日
初级完成 (估计的)
2026年12月31日
研究完成 (估计的)
2026年12月31日
研究注册日期
首次提交
2026年5月26日
首先提交符合 QC 标准的
2026年6月8日
首次发布 (实际的)
2026年6月12日
研究记录更新
最后更新发布 (实际的)
2026年6月12日
上次提交的符合 QC 标准的更新
2026年6月8日
最后验证
2026年5月1日
更多信息
此信息直接从 clinicaltrials.gov 网站检索,没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求,请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改,我们的网站上也会自动更新.