Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors
- Conditions
- Diseases of the blood and blood-forming organs and certain disorders involving the immune mechanism
- Registration Number
- KCT0004497
- Lead Sponsor
- ovo Nordisk
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Active, not recruiting
- Sex
- Male
- Target Recruitment
- 2
1. Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
2. Male aged = 12 years at the time of signing informed consent.
3. Body weight >25 kg at screening.
4. Congenital Haemophilia A or B of any severity with documented history of inhibitor (= 0.6 BU).
5. Patient has been prescribed, or in need of, treatment with bypassing agents in the last 24 weeks prior to screening (for patients not previously enrolled in NN7415-4310).
1. Known or suspected hypersensitivity to monoclonal antibodies.
2. Previous participation in this trial. Participation is defined as signed informed consent.
3. Participation in any clinical trial of an approved or non-approved investigational medicinal product within 5 half-lives or 30 days from screening, whichever is longer (not applicable for patients from NN7415-4310).
4. Platelets = 100x109/L at screening.
5. Fibrinogen below laboratory lower normal limit at screening.
6. Hepatic dysfunction defined as AST and/or ALT > 3 times the upper limit combined with total bilirubin > 1,5 times the upper limit at screening.
7. Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) = 30 ml/min/1.73 m2 for serum creatinine measured at screening.
8. Known inherited or acquired coagulation disorder other than congenital haemophilia.
9. History of thromboembolic disease*. 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.
10. A known systemic inflammatory condition requiring systemic treatment at screening.
11. Treatment with emicizumab within 180 days before screening.
12. Ongoing or planned Immune Tolerance Induction treatment.
13. Any disorder, except for conditions associated with haemophilia, which in the investigator’s opinion might jeopardise patient’s safety or compliance with the protocol.
* 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.
Study & Design
- Study Type
- Interventional Study
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The number of treated bleeding episodes (spontaneous and traumatic)
- Secondary Outcome Measures
Name Time Method Change in SF36v2 bodily pain, Change in SF36v2 physical functioning;Number of treated spontaneous bleeding episodes, Number of treated joint bleeds, Number of treated target joint bleeds;Number of thromboembolic events, Number of hypersensitivity type reactions, Number of injection site reactions, Number of patients with antibodies to concizumab;Concizumab plasma concentrations prior to the last prophylaxis dose administration in main part, Peak thrombin generation prior to the last prophylaxis dose administration in main part, Free TFPI concentration value prior to the last prophylaxis dose administration in main part;Concizumab plasma concentration (Cmax), Concizumab plasma concentration AUC