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CSL312 (Garadacimab) 预防性治疗遗传性血管性水肿发作的长期安全性和有效性

2026年5月6日 更新者：CSL Behring

一项评估 CSL312 (Garadacimab) 在遗传性血管性水肿预防性治疗中的长期安全性和有效性的开放标签研究

该 3b 期研究将评估 CSL312（也称为 garadacimab）皮下给药 (SC) 的长期安全性和有效性

研究概览

地位

完全的

条件

干预/治疗

研究类型

介入性

注册 (实际的)

171

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。

学习地点

  • 以色列
      • Ashkelon、以色列、7830604
        • Barzilai University Medical Center
  • 俄罗斯
      • Moscow、俄罗斯、115522
        • NRC Institute of Immunology FMBA Russia
  • 加拿大
    • Alberta
      • Edmonton、Alberta、加拿大、T6G 2B7
        • University of Alberta - Research Transition Facility
    • Ontario
      • Hamilton、Ontario、加拿大、L8N 3Z5
        • McMaster University
      • Ottawa、Ontario、加拿大、K1H 1E4
        • Ottawa Allergy Research Corp
      • Toronto、Ontario、加拿大、M3B 3S6
        • Gordon Sussman Clinical Research
    • Quebec
      • Montreal、Quebec、加拿大、H2W 1R7
        • Montreal Clinical Research Institute
  • 匈牙利
      • Budapest、匈牙利、1088
        • Semmelweis Egyetem Altalanos Orvostudományi Kar Belgyógyászati és Hematológiai Klinika
  • 台湾
      • Taichung、台湾、407
        • Taichung Veterans General Hospital
  • 德国
      • Berlin、德国、10117
        • Charité - Universitätsmedizin Berlin
      • Frankfurt、德国、60590
        • Universitätsklinikum Frankfurt
      • Mainz、德国、55131
        • Johannes Gutenberg-Universität KöR, Hautklinik und Poliklinik der Universitätsmedizin, Clinical Research Center
      • Mörfelden-Walldorf、德国、64546
        • HZRM Haemophilie Zentrum Rhein Main GmbH
  • 捷克语
      • Brno、捷克语、65691
        • University hospital St. Anna Ustav klinicke imunologie a alergologie, Fakultní nemocnice u sv. Anny v Brně
      • Prague、捷克语、150 06
        • University Hospital Motol
  • 新西兰
    • Auckland
      • Grafton、Auckland、新西兰、1023
        • Auckland City Hospital
  • 日本
    • Daikakuji Yaizu-shi
      • Shizuoka、Daikakuji Yaizu-shi、日本、425-0088
        • Koga Community Hospital
    • Edobashi, Tsu-shi
      • Mie、Edobashi, Tsu-shi、日本、Edobashi, Tsu-shi
        • Mie University Hospital
    • Hongo Bunkyo-ku
      • Tokyo、Hongo Bunkyo-ku、日本、113-8431
        • Juntendo University Hospital
    • Kamoda Kawagoe-shi
      • Saitama、Kamoda Kawagoe-shi、日本、350-8550
        • Saitama Medical Center
    • Kawasaki-shi
      • Kanagawa、Kawasaki-shi、日本、216-8511
        • St. Marianna University School of Medicine Hospital
    • Kugenumaishigami, Fujisawa-shi
      • Kanagawa、Kugenumaishigami, Fujisawa-shi、日本、251-0025
        • Clover Hospital
    • Matsubara Soka-shi
      • Saitama、Matsubara Soka-shi、日本、340-0041
        • Saiyu Soka Hospital
    • Midoricho, Tachikawa-shi
      • Tokyo、Midoricho, Tachikawa-shi、日本、190-0014
        • National Hospital Organization Disaster Medical Center
    • Nebeshima, Saga-shi
      • Saga、Nebeshima, Saga-shi、日本、849-8501
        • Saga University Hospital
    • Osaka-shi
      • Miyakojima-ku、Osaka-shi、日本、534-0021
        • Local Incorporated Administrative Agency Osaka City Hospital Organization Osaka City General Hospital
  • 澳大利亚
    • New South Wales
      • Campbelltown、New South Wales、澳大利亚、2560
        • Campbelltown Hospital / Western Sydney University
    • Victoria
      • Melbourne、Victoria、澳大利亚、3004
        • The Alfred Hospital
    • Western Australia
      • Murdoch、Western Australia、澳大利亚、6150
        • Fiona Stanley Hospital, Department of Clinical Immunology
  • 美国
    • Alabama
      • Birmingham、Alabama、美国、35209
        • Clinical Research Center of Alabama
    • Arizona
      • Litchfield Park、Arizona、美国、85340
        • Research Solutions of Arizona
      • Scottsdale、Arizona、美国、85251
        • Medical Research of Arizona
    • Arkansas
      • Little Rock、Arkansas、美国、72205
        • Little Rock Allergy & Asthma Clinic
    • California
      • Orange、California、美国、92868
        • Donald S. Levy M.D.
      • Santa Monica、California、美国、90404
        • Raffi Tachdjian MD, Inc.
      • Walnut Creek、California、美国、94598
        • Allergy & Asthma Clinical Research
    • Maryland
      • Chevy Chase、Maryland、美国、20815
        • Institute for Asthma and Allergy PC
    • Ohio
      • Cincinnati、Ohio、美国、45236
        • Bernstein Clinical Research Center, LLC
    • Pennsylvania
      • Hershey、Pennsylvania、美国、17033
        • PennState Health Milton S. Hershey Medical Center
    • Texas
      • Dallas、Texas、美国、75231
        • AARA Research Center
  • 荷兰
      • Amsterdam、荷兰、1105
        • Amsterdam UMC, location AMC
  • 西班牙
      • Barcelona、西班牙、8035
        • Hospital Universitari General de La Vall d'Hebron
      • Madrid、西班牙、28007
        • Hospital Gregorio Marañón, Servicio de Alergia
  • 香港
      • Hong Kong、香港
        • The University of Hong Kong, Queen Mary Hospital

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

12年 及以上 (孩子、成人、年长者)

接受健康志愿者

不

描述

纳入标准：

  • ≥ 12 岁的男性和女性
  • 诊断为临床证实的 C1-INH HAE
  • 筛选前 3 个月内经历≥ 3 次 HAE 发作
  • 参加了至少 1 个月的磨合期（仅限 CSL312-naïve 受试者）
  • 在磨合期内平均每月至少经历 1 次 HAE 攻击

排除标准：

  • 另一种形式的血管性水肿的伴随诊断，例如特发性或获得性血管性水肿或与荨麻疹相关的复发性血管性水肿
  • 在磨合期第一天前至少 2 周，使用 C1-INH 产品、雄激素、抗纤维蛋白溶解药或其他小分子药物常规预防 HAE 发作
  • 在磨合期第一天前 3 个月使用单克隆抗体，例如 lanadelumab (Takhzyro®)。
  • 女性受试者在筛选前 4 周内使用含雌激素的口服避孕药或激素替代疗法
  • 在研究期间和收到最后一剂 CSL312 后 30 天内，有生育能力和性活跃的女性或男性受试者未使用或不愿意使用可接受的避孕方法来避免怀孕
  • 怀孕、哺乳或不愿意停止母乳喂养

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：预防
  • 分配：不适用
  • 介入模型：单组作业
  • 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：CSL312
全人免疫球蛋白 G 亚类 4/λ 重组抑制剂单克隆抗体皮下给药
生物：CSL312
全人免疫球蛋白G亚类4/λ重组抑制剂单克隆抗体
其他名称：
  • 加拉达昔单抗
  • 因子 XIIa 抑制剂单克隆抗体

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
Number of Participants With Treatment-emergent Adverse Events (TEAE)
大体时间：Approximately up to 54 months
Approximately up to 54 months
Percentage of Participants With TEAE
大体时间：Approximately up to 54 months
The percentage of participants was rounded to one place of decimal.
Approximately up to 54 months
Number of TEAE
大体时间：Approximately up to 54 months
Approximately up to 54 months
TEAE Rates Per Injection
大体时间：Approximately up to 54 months
The TEAE rate per injection was calculated as the number of TEAE/ total number of injections. The number of injections was defined as the total injections received by participants during the respective safety evaluation period.
Approximately up to 54 months
TEAE Rates Per Participant Year
大体时间：Approximately up to 54 months
The TEAE rate per participant year was calculated as the total number of TEAE/ participant years. Participant years was defined as the sum of the time (in years) that participant were exposed to study treatment during the respective safety evaluation period.
Approximately up to 54 months

次要结果测量

结果测量
措施说明
大体时间
The Time-normalized Number (Per Month) of Hereditary Angioedema (HAE) Attacks During the Run-in Period and Treatment Period
大体时间：Run-in Period: Up to Day 60 and Treatment Period: Approximately up to 52 months
Time-normalized number of HAE attacks per month during treatment was calculated per participant as: [number of HAE attacks / length of participant treatment in days]*30.4375.
Run-in Period: Up to Day 60 and Treatment Period: Approximately up to 52 months
The Time-normalized Number (Per Year) of HAE Attacks During Treatment Period
大体时间：Approximately up to 52 months
Time-normalized number of HAE attacks per year during treatment was calculated per participant as: [number of HAE attacks / length of participant treatment in days]*365.25.
Approximately up to 52 months
Percentage Reduction in the Attack Rate During the Treatment Period Compared to the Run-in Period
大体时间：Run-in Period: Up to 60 days and Treatment Period: Approximately up to 52 months
The percentage reduction in the time-normalized number of HAE attacks was calculated within a participant as: 100*(1- time normalized number of HAE attacks per month during Treatment Period/time-normalized number of HAE attacks per month during Run-in Period).
Run-in Period: Up to 60 days and Treatment Period: Approximately up to 52 months
Number of Participants With Percentage Reduction (of >=50%, >=70%, >=90%, and 100%) in HAE Attacks
大体时间：Run-in Period: Up to Day 60 and Treatment Period: Approximately up to 52 months
The number of participants who achieved a percentage reduction in HAE attacks of >=50% (also considered as responders), >=70%, >=90%, and 100% (attack free) during the Treatment Period compared with the Run-in Period. The percentage reduction in the time-normalized number of HAE attacks per month was calculated as 100*[1 - (time-normalized number of HAE attacks per month under CSL312 treatment / time-normalized number of HAE attacks per month during Run-in Period)].
Run-in Period: Up to Day 60 and Treatment Period: Approximately up to 52 months
The Time-normalized Number (Per Month) of HAE Attacks Requiring On-demand Treatment
大体时间：Approximately up to 52 months
Time-normalized number of HAE attacks per month requiring on demand treatment was calculated per participant as: [number of HAE attacks requiring on demand treatment during treatment period / length of participant treatment in days]*30.4375.
Approximately up to 52 months
The Time-normalized Number (Per Year) of HAE Attacks Requiring On-demand Treatment
大体时间：Approximately up to 52 months
Time-normalized number of HAE attacks requiring on demand treatment per year was calculated per participant as: [number of HAE attacks requiring on demand treatment during treatment period / length of participant treatment in days]*365.25.
Approximately up to 52 months
The Time-normalized Number (Per Month) of Moderate and/or Severe HAE Attacks
大体时间：Approximately up to 52 months
Time-normalized number of moderate or severe HAE attacks per month during treatment period was calculated per participant as: [number of moderate or severe HAE attacks / length of participant treatment in days]*30.4375.
Approximately up to 52 months
The Time-normalized Number (Per Year) of Moderate and/or Severe HAE Attacks
大体时间：Approximately up to 52 months
Time-normalized number of moderate or severe HAE attacks per year during treatment period was calculated per participant as: [number of moderate or severe HAE attacks /length of participant treatment in days]*365.25.
Approximately up to 52 months
Number of Participants Rating Their Response to Therapy as Good or Excellent
大体时间：At Months 12, 24, and 36
Number of participants rating their response to therapy as good or excellent was evaluated as per Subject's Global Assessment of Response to Therapy (SGART) questionnaire. SGART is a patient-reported outcome that represents the participant's overall response to treatment using the following ratings: (0) none: worse or no response at all, not acceptable, (1) poor: very little response, not acceptable, (2) fair: some response, acceptable but could be better, (3) good: good response, acceptable, and (4) excellent: excellent response, as good as can be imagined. Cumulative responses as "Good or Excellent" are reported for this outcome measure.
At Months 12, 24, and 36
Percentage of Participants Rating Their Response to Therapy as Good or Excellent
大体时间：At Months 12, 24, and 36
Percentage of participants rating their response to therapy as good or excellent was evaluated as per SGART questionnaire. SGART is a patient-reported outcome that represents the participant's overall response to treatment using the following ratings: (0) none: worse or no response at all, not acceptable, (1) poor: very little response, not acceptable, (2) fair: some response, acceptable but could be better, (3) good: good response, acceptable, and (4) excellent: excellent response, as good as can be imagined. Cumulative responses as "Good or Excellent" are reported for this outcome measure. The percentage of participants was rounded to one place of decimal.
At Months 12, 24, and 36
Number of Participants Experiencing Serious Adverse Events (SAE), Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation
大体时间：Approximately up to 54 months
Approximately up to 54 months
Percentage of Participants Experiencing SAE, Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation
大体时间：Approximately up to 54 months
The percentage of participants was rounded to one decimal place.
Approximately up to 54 months
Number of Participants Experiencing TEAE by Severity
大体时间：Approximately up to 54 months
Severity of AE was assessed by the investigator and categorized as mild, moderate and severe. A mild AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. A moderate AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. A severe AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention.
Approximately up to 54 months
Percentage of Participants Experiencing TEAE by Severity
大体时间：Approximately up to 54 months
Severity of AE was assessed by the investigator and categorized as mild, moderate and severe. A mild AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. A moderate AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. A severe AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention. The percentage of participants was rounded to one decimal place.
Approximately up to 54 months
Number of Participants Experiencing Adverse Events of Special Interest (AESI)
大体时间：Approximately up to 54 months
The AESI defined for this study were thromboembolic events, abnormal bleeding events, and severe hypersensitivity including anaphylaxis. The AESI reported have been identified by investigators.
Approximately up to 54 months
Percentage of Participants Experiencing AESI
大体时间：Approximately up to 54 months
The AESI defined for this study were thromboembolic events, abnormal bleeding events, and severe hypersensitivity including anaphylaxis. The AESI reported have been identified by investigators.
Approximately up to 54 months
Number of Participants With Laboratory Findings Reported as TEAE
大体时间：Approximately up to 54 months
Approximately up to 54 months
Percentage of Participants With Laboratory Findings Reported as TEAE
大体时间：Approximately up to 54 months
The percentage of participant was rounded to one place of decimal.
Approximately up to 54 months
Number of Participants With Normal C1-esterase Inhibitor (nC1-INH) Experiencing TEAE
大体时间：Approximately up to 54 months
Approximately up to 54 months
Percentage of Participants With nC1-INH Experiencing TEAE
大体时间：Approximately up to 54 months
Approximately up to 54 months
Number of Participants With Anti-CSL312 Antibodies
大体时间：At Day 1, Months 6, 12, 36 and 43 (end of treatment [EOT])
At Day 1, Months 6, 12, 36 and 43 (end of treatment [EOT])
Percentage of Participants With Anti-CSL312 Antibodies
大体时间：At Day 1, Months 6, 12, 36 and 43 (EOT)
The percentage of participants was rounded to one decimal place.
At Day 1, Months 6, 12, 36 and 43 (EOT)

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

CSL Behring

调查人员

  • 研究主任：Study Director、CSL Behring

出版物和有用的链接

负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。

一般刊物

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)

2021年3月29日

初级完成 (实际的)

2025年11月21日

研究完成 (实际的)

2025年11月21日

研究注册日期

首次提交

2021年2月1日

首先提交符合 QC 标准的

2021年2月1日

首次发布 (实际的)

2021年2月4日

研究记录更新

最后更新发布 (实际的)

2026年6月2日

上次提交的符合 QC 标准的更新

2026年5月6日

最后验证

2026年4月1日

更多信息

与本研究相关的术语

其他相关的 MeSH 术语

其他研究编号

  • CSL312_3002
  • 2020-003918-12 (EudraCT编号)

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

是的

IPD 计划说明

CSL 将考虑系统审查小组或真正的研究人员提出的共享患者个人数据 (IPD) 的请求。 有关提交 IPD 自愿数据共享请求的流程和要求的信息，请通过 clinicaltrials@cslbehring.com 联系 CSL。

将考虑适用的特定国家/地区的隐私和其他法律法规，并可能阻止共享 IPD。

如果请求获得批准并且研究人员已执行适当的数据共享协议，则可以使用已适当匿名化的 IPD。

IPD 共享时间框架

IPD 请求可在本研究结果通过公共网站上的文章发布后的 12 个月内提交给 CSL。

IPD 共享访问标准

请求只能由系统审查小组或真正的研究人员提出，他们对 IPD 的拟议用途本质上是非商业性的，并且已获得内部审查委员会的批准。

CSL 不会考虑 IPD 请求，除非提出的研究问题旨在回答 CSL 内部审查委员会确定的重要且未知的医学或患者护理问题。

在 IPD 可用之前，请求方必须执行适当的数据共享协议。

IPD 共享支持信息类型

  • 研究方案
  • 树液

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

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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