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A Phase II Exploratory Study of SR604 Injection Evaluating the Safety and Efficacy in the Treatment of Congenital Coagulation Factor VII Deficiency

2026年7月14日 更新者:Shanghai RAAS Blood Products Co., Ltd.

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

This is an open-label, multicenter, exploratory Phase II clinical trial designed to evaluate the efficacy, safety, and pharmacokinetic characteristics of SR604 Injection in patients with congenital coagulation Factor VII deficiency.

研究概览

地位

尚未招聘

干预/治疗

研究类型

介入性

注册 (估计的)

12

阶段

  • 阶段2

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

学习地点

      • Tianjin、中国
        • Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

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:

  1. Age ≥12 years and ≤65 years at the time of signing the informed consent form; both genders eligible;
  2. 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;
  3. No active bleeding symptoms prior to first administration of SR604 Injection;
  4. 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;
  5. 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:

  1. Known history of hypersensitivity to the study drug formulation or any of its components;
  2. Intolerance to subcutaneous injection or other local skin abnormalities or dermatoses that may affect drug administration or safety assessment;
  3. Meeting any one of the following criteria during screening:

    1. Hemoglobin <60 g/L;
    2. Platelet count <100×10⁹/L;
    3. 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;
    4. Positive for anti-human immunodeficiency virus (HIV) antibodies.
  4. 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.);
  5. Protein C deficiency or Protein S deficiency;
  6. History of thrombosis, family history of thrombosis, or history of thrombophilia;
  7. Intracranial hemorrhage within 2 years prior to signing the informed consent form;
  8. 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.;
  9. Female patients with menstrual abnormalities caused by organic gynecological conditions (e.g., uterine fibroids, endometriosis, adenomyosis, etc.);
  10. 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;
  11. 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;
  12. 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;
  13. Enrolled in other clinical trials within 1 month prior to signing the informed consent form;
  14. Miscarriage or pregnancy termination within 3 months prior to signing the informed consent form; pregnant or breastfeeding women;
  15. Mental illness or significant psychiatric disorder, or incapacity or lack of cognitive ability due to other causes;
  16. 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.

    -

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:不适用
  • 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
实验性的: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
SR604 将作为 SC 注射给药。

研究衡量的是什么?

主要结果指标

结果测量
大体时间
Treated overall Annualized Bleeding Rate (ABR)
大体时间:Through 28 weeks of treatment.
Through 28 weeks of treatment.

次要结果测量

结果测量
大体时间
Treated spontaneous annualized bleeding rate
大体时间:over 28 weeks of treatment
over 28 weeks of treatment
Treated overall annualized joint bleeding rate
大体时间:over 28 weeks of treatment
over 28 weeks of treatment
Overall annualized bleeding rate including treated and untreated bleeding events
大体时间:over 28 weeks of treatment,
over 28 weeks of treatment,
Annualized menorrhagia bleeding rate (menstruating females only)
大体时间:over 28 weeks of treatment
over 28 weeks of treatment
Change in PBAC score (menstruating females only);
大体时间:From baseline over 28 weeks of treatment
From baseline over 28 weeks of treatment
Change in EQ-5D-5L health index score
大体时间:From pre-treatment over 28 weeks of treatment
From pre-treatment over 28 weeks of treatment
Change in EQ-VAS score
大体时间:From baseline over 28 weeks of treatment;
From baseline over 28 weeks of treatment;
Subject overall satisfaction score with treatment efficacy
大体时间:over 28 weeks of treatment
over 28 weeks of treatment

其他结果措施

结果测量
大体时间
Single-dose pharmacokinetic (PK) parameters:Time to Peak Plasma Concentration (Tmax)
大体时间:over 28 weeks of treatment
over 28 weeks of treatment
Single-dose pharmacokinetic (PK) parameters:Peak Plasma Concentration (Cmax)
大体时间:over 28 weeks of treatment
over 28 weeks of treatment
Single-dose pharmacokinetic (PK) parameters:Area Under the Concentration-Time Curve from Zero to Last Quantifiable Time Point (AUC0-t)
大体时间:over 28 weeks of treatment
over 28 weeks of treatment
Multiple-dose pharmacokinetic parameters-Time to Peak Plasma Concentration (Tmax)
大体时间:over 28 weeks of treatment
over 28 weeks of treatment
Multiple-dose pharmacokinetic parameters-Peak Plasma Concentration (Cmax)
大体时间:Over 28 weeks of treatment
Over 28 weeks of treatment
Safety and Immunogenicity:Incidence of AEs/SAEs/AESI,
大体时间:over 28 weeks of treatment
over 28 weeks of treatment
Incidence of drug-related AEs/SAEs/AESIs
大体时间:over 28 weeks of treatment
over 28 weeks of treatment
Safety and Immunogenicity: Anti-drug antibody (ADA) and neutralizing antibody (NAb) - number of subjects and incidence rate.
大体时间:over 28 weeks of treatment
over 28 weeks of treatment

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 (估计的)

2026年7月1日

初级完成 (估计的)

2027年2月1日

研究完成 (估计的)

2027年12月1日

研究注册日期

首次提交

2026年7月14日

首先提交符合 QC 标准的

2026年7月14日

首次发布 (实际的)

2026年7月17日

研究记录更新

最后更新发布 (实际的)

2026年7月17日

上次提交的符合 QC 标准的更新

2026年7月14日

最后验证

2026年5月1日

更多信息

与本研究相关的术语

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)?

不

药物和器械信息、研究文件

研究美国 FDA 监管的药品

不

研究美国 FDA 监管的设备产品

不

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