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

Phase 1

Conditions: Haemophilia AHaemophilia A with inhibitorsHaemophilia BHaemophilia B with inhibitors
MedDRA version: 20.0Level: LLTClassification code 10018938Term: Haemophilia A (Factor VIII)System Organ Class: 100000004850
MedDRA version: 20.0Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 100000004850
MedDRA version: 20.0Level: LLTClassification code 10053752Term: Hemophilia B with anti factor IXSystem Organ Class: 100000004850
MedDRA version: 20.0Level: LLTClassification code 10053751Term: Hemophilia A with anti factor VIIISystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2019-002641-37-SE

Lead Sponsor: ovo Nordisk A/S

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: 80

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 years or older at the time of signing informed consent (18 years or older for the PK/PD cohort).
- Diagnosis of congenital severe haemophilia A (FVIII below 1%) or moderate/severe congenital haemophilia B (FIX equal to or below 2%), or congenital haemophilia with inhibitors.
- Patient has been prescribed, or is in need of, treatment with coagulation factor product or bypassing agent in the last 24 weeks prior to screening.
Are the trial subjects under 18? yes
Number of subjects for this age range: 25
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion Criteria

- Known or suspected hypersensitivity to any constituents of the trial product.
- 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 evaluate the effect of 1.75 mg/kg concizumab administered once-weekly in maintaining adequate control of bleeding in adult and adolescent patients with haemophilia A or B with or without inhibitors;Secondary Objective: 1. To investigate the safety of concizumab once-weekly prophylaxis in adult and adolescent patients with haemophilia A or B with or without inhibitors<br>2. To investigate the pharmacokinetics (PK) and pharmacodynamics (PD) of concizumab once-weekly prophylaxis in adult and adolescent patients with haemophilia A or B with or without 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. Number of thromboembolic events <br>2. Number of hypersensitivity type reactions<br>3. Number of injection site reactions <br>4. Patient developed antibodies to concizumab – yes/no<br>5. Patient developed antibodies to concizumab – yes/no<br>6. Concizumab plasma concentrations prior to the last prophylaxis dose administration in main part<br>7. Peak thrombin generation prior to the last prophylaxis dose administration in main part<br>8. Free TFPI concentration value prior to the last prophylaxis dose administration in main part<br>9. Concizumab highest plasma concentration (Cmax) during the weekly PK profile<br>10. Concizumab plasma concentration AUC during the weekly PK profile;Timepoint(s) of evaluation of this end point: 1.- 4.: From start of treatment (week 0) to the end of the main part (24 weeks)<br>5.: From start of treatment (week 0) to the end of trial (week 135)<br>6.-8..: Week 24<br>9.-10.:Assessed once between week 7 and week 24

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