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如果您患有血友病，则同情地使用 Concizumab

2026年10月26日 更新者：Novo Nordisk A/S

针对先天性血友病患者的 Concizumab 同情使用计划

The compassionate use programme will give participants concizumab for free, even though it is not yet approved by health authorities. This is because participants need this medicine to treat their haemophilia properly. The programme will check that participants are safe and that the medicine works for them. The programme may last for years. Participants will take one injection under their skin every day. Participants will have 4-5 visits with the study doctor for the first half year. After that they will have 1 visit every half year. At all clinic visits participants will have blood samples taken. Participants will fill in a diary between the visits.

A patient is considered to have completed the programme when any of the following criteria occurred first: 1) when the patient is included in a clinical trial with concizumab or 2) up to 6 months after concizumab is commercially available in the patient's country and approved for the patient (The time span of 6 months should provide ample time for the patient to obtain concizumab commercially) or 3) the sponsor decides to discontinue concizumab clinical development for the patient's population.

研究概览

地位

不再可用

条件

干预/治疗

研究类型

扩展访问

扩展访问类型

  • 中型人口

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

  • 保加利亚
      • Plovdiv、保加利亚、4002
        • UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics
  • 瑞典
      • Solna、瑞典、171 64
        • Koagulationsmottagningen
  • 美国
    • California
      • Los Angeles、California、美国、90027
        • Children's Hospital Los Angeles - Endocrinology
      • San Francisco、California、美国、94158
        • University of California San Francisco UCSF
    • Connecticut
      • Hartford、Connecticut、美国、06106
        • Connecticut Children's Medical Center
    • District of Columbia
      • Washington D.C.、District of Columbia、美国、20007
        • Georgetown University Medical Center
    • Georgia
      • Augusta、Georgia、美国、30912
        • Augusta University
      • Savannah、Georgia、美国、31404
        • Memorial Health University Medical Center
    • Idaho
      • Boise、Idaho、美国、83712
        • St. Luke's Mountain States Tumor Institute
    • Illinois
      • Chicago、Illinois、美国、60611
        • Childrens Hospital of Chicago
    • Indiana
      • Indianapolis、Indiana、美国、46260
        • Indiana Hemophilia-Thromb Ctr
    • Louisiana
      • New Orleans、Louisiana、美国、70118
        • Children's Hosp-New Orleans
    • Michigan
      • Detroit、Michigan、美国、48201
        • Children's Hospital of Michigan
    • Mississippi
      • Flowood、Mississippi、美国、39232
        • Southern Specialty Clinic
      • Madison、Mississippi、美国、39110
        • Louisiana Ctr for Adv Med-LCAM
    • Missouri
      • Kansas City、Missouri、美国、64108
        • The Children's Mercy Hospital
    • Nebraska
      • Omaha、Nebraska、美国、68114
        • Children's Nebraska
    • North Carolina
      • Greenville、North Carolina、美国、27834
        • ECU Sickle Cell Comp Clinic
    • Ohio
      • Columbus、Ohio、美国、43205
        • Nationwide Children's Hospital
    • Pennsylvania
      • Hershey、Pennsylvania、美国、17033
        • Penn State Hershey Medical Center
    • South Carolina
      • Greenville、South Carolina、美国、29605
        • BI-LO Chrt Childn's Cancer Ctr
    • Texas
      • Fort Worth、Texas、美国、76104
        • Cook Children's Hospital-Hematology-Oncology
      • Houston、Texas、美国、77030
        • Texas Children's Hospital_Houston
      • Houston、Texas、美国、77030
        • Univ TX Hlth Sci Ctr Houston
    • Virginia
      • Richmond、Virginia、美国、23298-0461
        • Virginia Cwealth Univ Richmond

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

  • 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:

  • Informed consent obtained before any programme-related activities. Programme-related activities are any procedures that are carried out as part of the programme.

  • Patients with congenital haemophilia:

    1. severe haemophilia A (coagulation factor VIII (FVIII) less than 1%) or moderate/severe haemophilia B (coagulation factor IX (FIX) less than or equal to 2%) without inhibitors or
    2. any haemophilia severity with documented history of inhibitors (more than or equal to 0.6 bethesda unit (BU)) who cannot be treated satisfactorily with authorised and marketed medicines (example: due to inhibitors or allergic reactions to factor-containing products, or due to poor venous access), and who are not able to enrol in clinical trials designed to support the development and registration of concizumab medicines (example: due to inhibitors or allergic reactions to factor-containing products, or due to poor venous access) as per investigator and Novo Nordisk assessment.

  • The potential benefit for the individual patient justifies the potential risks of treatment.

Exclusion Criteria:

  • Known or suspected hypersensitivity to investigational medicinal product or related products.
  • Any condition (current or medical history), which in the investigator's or Novo Nordisk's opinion might jeopardise patient's safety or compliance with the protocol.

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？

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

Novo Nordisk A/S

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。

研究注册日期

首次提交

2021年6月4日

首先提交符合 QC 标准的

2021年6月4日

首次发布 (实际的)

2021年6月10日

研究记录更新

最后更新发布 (实际的)

2026年10月28日

上次提交的符合 QC 标准的更新

2026年10月26日

最后验证

2026年10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

  • NN7415-4807

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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