Haemophilia A Haemophilia A with inhibitors Haemophilia B Haemophilia B with inhibitors MedDRA version: 20.0 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII) System Organ Class: 100000004850 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 10018939 Term: Haemophilia B (Factor IX) System Organ Class: 100000004850 MedDRA version:
Conditions
Sponsors
Novo Nordisk A/S
Eligibility
Sex/Gender
Male
Inclusion criteria
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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 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
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Number of thromboembolic events 2. Number of hypersensitivity type reactions 3. Number of injection site reactions 4. Patient developed antibodies to concizumab – yes/no 5. Patient developed antibodies to concizumab – yes/no 6. Concizumab plasma concentrations prior to the last prophylaxis dose administration in main part 7. Peak thrombin generation prior to the last prophylaxis dose administration in main part 8. Free TFPI concentration value prior to the last prophylaxis dose administration in main part 9. Concizumab highest plasma concentration (Cmax) during the weekly PK profile 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) 5.: From start of treatment (week 0) to the end of trial (week 135) 6.-8..: Week 24 9.-10.:Assessed once between week 7 and week 24 | — |
Countries
Canada, European Union, India, Japan, Korea, Republic of, Netherlands, Russian Federation, Serbia, Sweden, Switzerland, Thailand, Ukraine, United States
Contacts
Public ContactClinical Disclosure (1452)
Novo Nordisk A/S
Outcome results
None listed