Research study to look at how well the drug concizumab works in your body if you have haemophilia with inhibitors
- Conditions
- Haemophilia A with inhibitorsHaemophilia B with inhibitorsMedDRA version: 20.0Level: LLTClassification code 10053751Term: Hemophilia A with anti factor VIIISystem Organ Class: 100000004850MedDRA version: 20.0Level: LLTClassification code 10053752Term: Hemophilia B with anti factor IXSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2018-004889-34-GB
- Lead Sponsor
- ovo Nordisk A/S
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Male
- Target Recruitment
- 133
- 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
- Male aged 12 or older years at the time of signing informed consent
- Congenital Haemophilia A or B of any severity with documented history of inhibitor (0.6 BU or more)
- 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)
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 108
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10
- Known or suspected hypersensitivity to monoclonal antibodies
- Known inherited or acquired coagulation disorder other than congenital haemophilia
- Ongoing or planned Immune Tolerance Induction treatment
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To compare effect of concizumab prophylaxis to no prophylaxis (on-demand treatment with bypassing agents) in reducing the number of bleeding episodes in adult and adolescent patients with haemophilia A or B with inhibitors;Secondary Objective: 1. To compare the patient reported outcomes (PROs) after treatment with concizumab prophylaxis vs no prophylaxis in adult and adolescent patients with haemophilia A or B with inhibitors<br>2. To investigate the safety of concizumab prophylaxis in adult and adolescent patients with haemophilia A or B with inhibitors<br>3. To investigate the PK and PD parameters of concizumab prophylaxis in adult and adolescent patients with haemophilia A or B with inhibitors;Primary end point(s): The number of treated bleeding episodes (spontaneous and traumatic);Timepoint(s) of evaluation of this end point: From start of treatment (week 0) to the end of the main part (week 24)
- Secondary Outcome Measures
Name Time Method Secondary end point(s): 1. Change in SF36v2 bodily pain<br>2. Change in SF36v2 physical functioning;Timepoint(s) of evaluation of this end point: 1-2. From baseline (week 0) to week 24