A research study on how well concizumab works for you if you have haemophilia A or B with or without inhibitors (explorer10)
- Conditions
- Haemophilia A with or without inhibitorsHaemophilia B with our without inhibitorsMedDRA version: 20.0Level: LLTClassification code 10053752Term: Hemophilia B with anti factor IXSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 20.0Level: LLTClassification code 10053751Term: Hemophilia A with anti factor VIIISystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 20.0Level: LLTClassification code 10053753Term: Hemophilia A without inhibitorsSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 20.1Level: LLTClassification code 10053754Term: Hemophilia B without inhibitorsSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2020-000504-11-SE
- Lead Sponsor
- ovo Nordisk A/S
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 127
- Informed consent/assent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
- Diagnosis of congenital severe haemophilia A (FVIII less than 1%) or moderate/severe congenital haemophilia B (FIX equal to or less than 2%), or congenital haemophilia with inhibitors.
- For arm 1 only: Male aged less than <12 years of age at the time of signing informed consent.
- For arm 1 only: Patients with inhibitors (HAwI or HBwI):
a) Patients with HAwI with historical medical records of a total of at least 26 weeks of on-demand treatment within the last 52 weeks prior to
enrolment
b) Patients with HBwI with historical medical records of a total of at least 26 weeks of on-demand treatment within the last 52 weeks prior to
enrolment
c) Patients with HBwI regardless of the regimen and duration of previous haemophilia treatment
- For arm 1 only: Patients without inhibitors (HA or HB):
a) Patients with historical medical records of at least 52 weeks of on-demand treatment during the last year prior to enrolment and with at
least 3 documented treated bleeds during this period
b) Patients with historical medical records of a total of at least 26 weeks of PPX treatment within the last 52 weeks prior to enrolment
- For arm 2 only: Male patients (regardless of age) previously treated with concizumab via compassionate use.
Are the trial subjects under 18? yes
Number of subjects for this age range: 125
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 2
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Known or suspected hypersensitivity to study intervention or related products.
- Known inherited or acquired coagulation disorder other than congenital haemophilia.
- Ongoing or planned Immune Tolerance Induction treatment.
- History of thromboembolic disease(a). Current clinical signs of or treatment for thromboembolic disease. Patients who in the judgement of the investigator are considered at high risk of thromboembolic events(b)
(a) Includes arterial and venous thrombosis including myocardial infarction, pulmonary embolism, cerebral infarction/thrombosis, deep vein thrombosis, other clinically significant thromboembolic events and peripheral artery occlusion.
(b) Thromboembolic risk factors could include, but are not limited to, hypercholesterolemia, diabetes mellitus, hypertension, obesity, smoking, family history of thromboembolic events, arteriosclerosis, other conditions associated with increased risk of thromboembolic events.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method