Haemophilia A Haemophilia B MedDRA version: 20.0 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII) System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: LLT Classification code 10018939 Term: Haemophilia B (Factor IX) System Organ Class: 10010331 - Congenital, familial and genetic disorders
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. - Congenital severe haemophilia A (FVIII less than 1%) or B (FIX 2% or less) Are the trial subjects under 18? yes Number of subjects for this age range: 35 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 113 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: - Known or suspected hypersensitivity to any constituent of the trial product or related products - Known inherited or acquired coagulation disorder other than congenital haemophilia - Presence of confirmed inhibitors 0.6 BU or greater at screening - History of thromboembolic disease (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). 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 (thromboembolic risk factors could include, but are not limited to, hypercholesterolemia, diabetes mellitus, hypertension, obesity, smoking, family history of thromboembolic events, arteriosclerosis, other conditions associated with increased risk of thromboembolic events.)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 1. To compare effect of concizumab prophylaxis (PPX) to no prophylaxis (on-demand (OD) treatment with factor) in reducing the number of bleeding episodes in adult and adolescent patients with haemophilia A without inhibitors 2. To compare effect of concizumab prophylaxis to no prophylaxis (on-demand treatment with factor) in reducing the number of bleeding episodes in adult and adolescent patients with haemophilia B without inhibitors;Secondary Objective: 1. To compare the effect of concizumab prophylaxis to the patients’ previous prophylaxis treatment in reducing the number of bleeding episodes in adult and adolescent patients with haemophilia A without inhibitors 2. To compare the effect of concizumab prophylaxis to the patients’ previous prophylaxis treatment in reducing the number of bleeding episodes in adult and adolescent patients with haemophilia B without inhibitors 3. To investigate the safety of concizumab prophylaxis in adult and adolescent patients with haemophilia A or B without inhibitors 4. To investigate the PK and PD parameters of concizumab prophylaxis in adult and adolescent patients with haemophilia A or B without inhibitors;Primary end point(s): 1. For haemophilia A patients without inhibitors: The number of treated spontaneous and traumatic bleeding episodes 2. For haemophilia B patients without inhibitors: The number of treated spontaneous and traumatic bleeding episodes;Timepoint(s) of evaluation of this end point: 1-2. On demand (arm 1): From randomisation after the pause (week 0) up until start of concizumab treatment (week 24). Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the confirmatory analyses cut-off (at least 32 weeks) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. For haemophilia A patients without inhibitors: The number of treated spontaneous and traumatic bleeding episodes 2. For haemophilia B patients without inhibitors: The number of treated spontaneous and traumatic bleeding episodes 3. For haemophilia A patients without inhibitors: Number of treated spontaneous bleeding episodes 4. For haemophilia B patients without inhibitors: Number of treated spontaneous bleeding episodes 5. For haemophilia A patients without inhibitors: Number of treated spontaneous and traumatic joint bleeds 6. For haemophilia B patients without inhibitors: Number of treated spontaneous and traumatic joint bleeds 7. For haemophilia A patients without inhibitors: Number of treated spontaneous and traumatic target joint bleeds 8. For haemophilia B patients without inhibitors: Number of treated spontaneous and traumatic target joint bleeds 9. Number of thromboembolic events 10. Number of thromboembolic events 11. Number of hypersensitivity type reactions 12. Number of hypersensitivity type reactions 13. Number of injection site reactions 14. Number of injection site reactions 15. Number of patients with antibodies to concizumab 16. Number of patients with antibodies to concizumab 17. Pre-dose (trough) concizumab plasma concentration (Ctrough) 18. Pre-dose thrombin peak 19. Pre-dose free tissue factor pathway inhibitor (TFPI) concentration 20. Maximum concizumab plasma concentration (Cmax) 21. Area under the concizumab plasma concentration-time curve (AUC);Timepoint(s) of evaluation of this end point: 1-2:Arm 4:patients on stable PPX min.24wks in trial 4322: -Previous PPX (in 4322):PPX stable-end of trial(EOT) -Concizumab (Conci) PPX (in 4307):Maintenance dose is confirmed/increased/decreased–conf. analyses (CA) (min.24wks) 3-8:OD (arm 1):Randomisation post-pause(wk0)-dose start(wk24) -Conci (arm 2):New regimen start(wk0)–CA(min.32wks) 9,11,13: OD (arm 1 main part): -Randomisation–OD treatment (Trt.) to dose start 9,11,1 | — |
Countries
Algeria, Australia, Bosnia and Herzegovina, Bulgaria, Canada, Croatia, Denmark, Estonia, European Union, France, Germany, Hungary, India, Italy, Japan, Korea, Republic of, Lithuania, Malaysia, Mexico, Poland, Portugal, Russian Federation, Serbia, Slovakia, South Africa, Spain, Sweden, Switzerland, Thailand, Türkiye, Ukraine, United Kingdom, United States
Contacts
Public ContactClinical Transparency (2834)
Novo Nordisk A/S
Outcome results
None listed