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IV 人血浆来源的 C1 酯酶抑制剂浓缩物治疗先天性 C1-INH 缺乏症患者的 3 期研究，用于治疗和术前预防急性遗传性血管性水肿发作

2026年9月25日 更新者：Octapharma

对先天性 C1-INH 缺乏症参与者进行静脉注射人血浆 C1 酯酶抑制剂 (C1-INH) 浓缩物进行治疗的前瞻性、多中心、随机、双盲、平行组、安慰剂对照、疗效和安全性 3 期研究急性遗传性血管性水肿发作的术前预防和预防

Prospective, multicenter, randomized, double-blind, parallel group, placebo- controlled, efficacy and safety phase 3 study of an intravenous human plasma- derived C1 esterase inhibitor (C1-INH) concentrate in participants with congenital C1-INH deficiency for the treatment and pre-procedure prevention of acute hereditary angioedema attacks

研究概览

地位

招聘中

条件

干预/治疗

研究类型

介入性

注册 (估计的)

124

阶段

  • 第三阶段

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

  • 乌克兰
      • Kyiv、乌克兰、03057
        • 招聘中
        • Octapharma Research Site
      • Lviv、乌克兰、79035
        • 尚未招聘
        • Octapharma Research Site
      • Lviv、乌克兰、79010
        • 撤销
        • Octapharma Research Site
  • 亚美尼亚
      • Yerevan、亚美尼亚
        • 招聘中
        • Octapharma Research Site
  • 保加利亚
      • Sofia、保加利亚、1431
        • 招聘中
        • Octapharma Research Site
  • 印度
      • Bangalore、印度、560017
        • 尚未招聘
        • Octapharma Research Site
      • Chhatrapati Sambhajinagar、印度、431003
        • 尚未招聘
        • Octapharma Research Site, Chhatrapati Sambhajinagar
      • Patna、印度、801507
        • 尚未招聘
        • Octapharma Research Site
    • Maharashtra
      • Pune、Maharashtra、印度、411040
        • 尚未招聘
        • Octapharma Research Site
  • 土耳其（türkiye）
      • Ankara、土耳其（türkiye）
        • 尚未招聘
        • Octapharma Research Site
      • Istanbul、土耳其（türkiye）
        • 尚未招聘
        • Octapharma Research Site
      • Izmir、土耳其（türkiye）
        • 撤销
        • Octapharma Research Site
      • Sakarya、土耳其（türkiye）
        • 尚未招聘
        • Octapharma Research Site
  • 塞尔维亚
      • Kragujevac、塞尔维亚、11221
        • 撤销
        • Octapharma Research Site
  • 墨西哥
      • Mexico City、墨西哥、06720
        • 尚未招聘
        • Octapharma Research Site
      • México、墨西哥
        • 尚未招聘
        • Octapharma Research Site
  • 秘鲁
      • Lima、秘鲁、15001
        • 招聘中
        • Octapharma Research Site 5103
      • Lima、秘鲁、15001
        • 招聘中
        • Octapharma Research Site 5102
  • 罗马尼亚
      • Cluj-Napoca、罗马尼亚、400162
        • 撤销
        • Octapharma Research Site
  • 美国
    • Colorado
      • Centennial、Colorado、美国、80112
        • 撤销
        • Octapharma Research Site
    • Michigan
      • Farmington Hills、Michigan、美国、48334
        • 撤销
        • Octapharma Research Site
    • Ohio
      • Toledo、Ohio、美国、43617
        • 撤销
        • Octapharma Research Site
  • 阿尔巴尼亚
      • Tirana、阿尔巴尼亚
        • 撤销
        • Octapharma Research Site
  • 阿根廷
      • Rosario、阿根廷
        • 招聘中
        • Octapharma Research Site
  • 黑山
      • Podgorica、黑山
        • 撤销
        • Octapharma Research Site

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

2年 及以上 (孩子、成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

描述

Inclusion Criteria:

  1. Is at least 18 years of age (applicable for 1st study phase) or is at least 2 years of age (applicable for 2nd study phase)

  2. Has confirmed diagnosis of HAE type I or II

  3. Has had at least 3 moderate or severe HAE attacks (excluding extremity attacks) in the last 3 months before the Screening Visit. For participants ≥2 and ≤12 years of age, has had at least 1 moderate or severe HAE attack (excluding extremity attacks) in the last 6 months before Screening Visit

  4. Has a documented congenital C1-INH functional activity <50% with or without C1-INH deficiency and C4 antigen level below the laboratory reference range

  5. Participant or the participant's legally authorized representative(s) has signed informed consent (as required by local law), with the assent of participants legally capable of providing it, as applicable

  6. States willingness to comply with all study procedures and availability for the duration of the study

  7. If the participant is of childbearing potential (CBP), has a negative pregnancy test and must have been using a highly effective method of contraception and continue to do so until at least 2 weeks after their last dose (for both blinded and open-label doses of IMP). Not of CBP is defined as surgically sterilized (hysterectomy, bilateral oophorectomy) or who are postmenopausal (defined as women with no menses for 12 months without an alternative medical cause). Highly effective methods of contraception:

    • Combined hormonal contraception (estrogens and progesterone) methods such as oral, implantable, intravaginal, injectable, or transdermal contraceptives at a stable dose for a minimum of 1 full cycle (hormonal contraceptives must inhibit ovulation) and for at least 4 weeks before screening
    • Progesterone only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable)
    • Intrauterine device
    • Intrauterine hormone-releasing system inserted at least 4 weeks before screening
    • Bilateral tubal ligation/occlusion or vasectomized partner (with surgical success confirmed by medical assessment) OR Agrees to abstain from heterosexual intercourse during study participation and to use a highly effective contraceptive (as described above) as backup if they become sexually active during the study. Abstinence is only acceptable if this is the participant's usual lifestyle. Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method are not acceptable methods of contraception
    • Note: If a participant of CBP has a positive or suspected positive urine pregnancy test within 72 hours prior to treatment, a serum pregnancy test will be required
    • Male participants must not plan to father a child or donate sperm for 90 days after their last dose of study drug (for both blinded and open-label doses of the IMP). However, there are no official contraception requirements for male participants during the study.

Inclusion Criteria for IMP Dosing for QAT:

  1. Has confirmed QAT per definition criteria
  2. Has a swelling episode that is new and not the continuation of a previous HAE attack

Exclusion Criteria:

  1. Has a history of clinically relevant antibody development against C1-INH
  2. Has a medical history consistent with Type 3 HAE (i.e., onset at age above 40 year, no family history, no known HAE mutation, low C1q level in plasma)
  3. Has a history of allergic reaction to C1-INH or other blood/plasma product
  4. Has a history of B-cell malignancy that was unresolved in the past 5 years
  5. Has a narcotic and/or alcoholic addiction
  6. Has participated in any other investigational drug evaluation within 30 days before screening
  7. Is pregnant or breastfeeding
  8. Has any clinically significant medical or psychiatric condition that, in the investigator's opinion would interfere with the participant's ability to participate in the study
  9. Has a history of thromboembolic events (TEEs), myocardial infarction, unstable angina pectoris, critical aortic stenosis, cerebrovascular accident, transient ischemic attack, severe peripheral vascular disease, or disseminated intravascular coagulation within one year before screening
  10. (applicable until IDMC review of the interim preliminary safety and efficacy data): has clinically significant derangement in measurements of cardiovascular status (i.e. uncontrolled arterial hypertension, cardiac insufficiency New York Heart Association (NYHA) class III-IV), pulmonary status (i.e., COPD GOLD classification 3 and 4, severe asthma) and renal status (i.e., eGFR below 90 ml/min per 1.73 m2)

Exclusion Criteria for IMP Dosing for QAT:

  1. Has received blood or a blood product for prophylactic or acute treatment with any C1-INH (Berinert®, Cinryze®, HAEgarda®, Ruconest®, etc.), non-biological bradykinin and kallikrein pathway inhibitors (e.g., ecallantide, icatibant, berotralstat), or treatment with tranexamic acid within 14 days before dosing with the IMP (or is not willing to abstain from these medications throughout the study)
  2. started or changed hormone replacement therapy or selective estrogen receptor modulators (e.g., tamoxifen) within 14 days before IMP dosing
  3. Started or changed androgen therapy (e.g. testosterone, dehydro- epiandrosterone/androstenedione, oxandrolone, danazol, stanozolol) within 14 days before IMP dosing or is not willing to maintain a stable dose throughout the study
  4. Started or changed the dose of monoclonal antibodies e.g. lanadelumab within 11 weeks before dosing or not willing to maintain a stable dose throughout the study
  5. Has used narcotic pain medications or non-opioid analgesics within 7 days before IMP dosing for a QAT
  6. Has received OCTA-C1-INH within 14 days before IMP dosing

Exclusion Criteria for IMP Dosing for PK:

  1. Has received blood or a blood product for prophylactic or acute treatment with any C1-INH (Berinert®, Cinryze®, HAEgarda®, Ruconest®, etc.), non-biological bradykinin and kallikrein pathway inhibitors (e.g., ecallantide, icatibant, berotralstat), or treatment with tranexamic acid within 14 days before dosing with the IMP (or is not willing to abstain from these medications throughout the study)
  2. Is receiving hormone replacement therapy or selective estrogen receptor modulators (e.g., tamoxifen) and has had their dose changed within 14 days before IMP dosing
  3. Is receiving or has received androgen therapy (e.g., testosterone, dehydroepiandrosterone/androstenedione, oxandrolone, danazol, stanozolol) IN ANY DOSE within 14 days before dosing
  4. Started or changed the dose of monoclonal antibodies e.g lanadelumab within 11 weeks before dosing or not willing to maintain a stable dose throughout the study
  5. Has used narcotic pain medications or non-opioid analgesics within 7 days before IMP dosing
  6. Has received IMP within 14 days before IMP dosing
  7. Has planned dental, medical, or surgical procedures during the PK Period that will require pre-procedural prevention

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：双倍的

武器和干预

参与者组/臂
干预/治疗
实验性的：OCTA-C1-INH

OCTA-C1-INH injection, 20IU/kg BW after first qualifying attack. Treatment to be administered to blinded as well as open-label subjects.
药品：OCTA-C1-INH

OCTA-C1-INH is a stable, sterile, virus-inactivated, nano-filtered, highly purified concentrate of human C1-INH prepared from pooled human plasma. After reconstitution in 2.5mL water for injection, the solution can be administered as a slow IV injection. OCTA-C1-INH is given as a dose of 20 IU/kg body weight (BW)
安慰剂比较：Placebo

0.1 mL/kg BW 0.9% sodium chloride solution injection after first qualifying attack. Only blinded subjects to receive.
其他：Placebo

0.1 mL/kg BW 0.9% sodium chloride injection

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
Time (h) to beginning of unequivocal symptom relief at the defining site in blinded participants.
大体时间：Within 4 hours after injection

Patient will rate symptom relief for the defining attack site (site of swelling or pain) from 15 minutes after start of the IMP injection every 15 minutes over 4 hours. Unequivocal relief is defined as having 3 consecutive reports of "absent now but present before," "absent now and absent before", or "present, symptoms better" on the 5-grade SRRS.

This measure will also be used for the secondary outcomes in a different context.
Within 4 hours after injection

次要结果测量

结果测量
措施说明
大体时间
Percentage of participants responding to treatment
大体时间：within 4 hours after injection

Percentage of patients responding to treatment, defined as beginning of unequivocal symptom relief at the defining site within 4 hours after injection (once per participant after first qualifying attack in the study). Unequivocal relief is defined as having 3 consecutive reports of "absent now but present before," "absent now and absent before", or "present, symptoms better" on the 5-grade symptom relief rating.
within 4 hours after injection
Time to beginning of unequivocal symptom relief at all sites involved
大体时间：Within 4 hours after injection

Time to the beginning of unequivocal symptom relief at all sites involved within 4 hours after injection. Unequivocal relief is defined as having 3 consecutive reports of "absent now but present before," "absent now and absent before", or "present, symptoms better" on the 5-grade symptom relief rating.
Within 4 hours after injection
Changes in symptom severity at the defining site by VAS severity rating
大体时间：Within 4 hours after injection

Changes in symptom severity at the defining site by visual analog scale (VAS) rating from pre-injection over 4 hours after injection. The VAS is a self-reported 0-100 pain and swelling rating scale.
Within 4 hours after injection

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。

赞助

Octapharma

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4月30日

初级完成 (估计的)

2026年12月1日

研究完成 (估计的)

2027年6月1日

研究注册日期

首次提交

2024年1月11日

首先提交符合 QC 标准的

2024年4月5日

首次发布 (实际的)

2024年4月12日

研究记录更新

最后更新发布 (实际的)

2026年9月29日

上次提交的符合 QC 标准的更新

2026年9月25日

最后验证

2026年8月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • CONE-02

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

是的

研究美国 FDA 监管的设备产品

不

在美国制造并从美国出口的产品

不

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.

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