Research study to look at how well the drug concizumab works in your body if you have haemophilia with inhibitors

Phase 1

• Conditions: Haemophilia A with inhibitors Haemophilia B with inhibitors; MedDRA version: 20.0 Level: LLT Classification code 10053751 Term: Hemophilia A with anti factor VIII System Organ Class: 100000004850; MedDRA version: 20.0 Level: LLT Classification code 10053752 Term: Hemophilia B with anti factor IX System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2018-004889-34-FI
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-11-18
• Last Update Posted: 11 March 2020

Recruitment & Eligibility

• Status: A
• Sex: Not specified
• Target Recruitment: 133

Inclusion Criteria

- 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

Exclusion Criteria

- 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;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);<br> 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<br>

Secondary Outcome Measures

Name	Time	Method
<br> Secondary end point(s): 1. Change in SF36v2 bodily pain<br> 2. Change in SF36v2 physical functioning<br> ;Timepoint(s) of evaluation of this end point: 1-2. From baseline (week 0) to week 24