Haemophilia A with or without inhibitors Haemophilia B with our without inhibitors MedDRA version: 20.0 Level: LLT Classification code 10053752 Term: Hemophilia B with anti factor IX 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 10053753 Term: Hemophilia A without inhibitors System Organ Class: 100000004850 MedDRA versi
Conditions
Interventions
Product Name: Concizumab C 10 mg/mL PDS290
Pharmaceutical Form: Solution for injection in pre-filled pen
INN or Proposed INN: Concizumab
Other descriptive name: Concizumab
Concentration unit: mg/ml mi
Sponsors
Novo Nordisk A/S
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: - Informed consent/assent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study. - Diagnosis of congenital severe haemophilia A (FVIII less than 1%) or moderate/severe congenital haemophilia B (FIX equal to or less than 2%), or congenital haemophilia with inhibitors. - For arm 1 only: Male aged less than 12 years of age at the time of signing informed consent. - For arm 1 only: Patients with medical records of a total of at least 26 weeks of treatment within the last 52 weeks prior to enrolment(a) * Patients with HAwI with medical records of a total of at least 26 weeks of on demand treatment within the last 52 weeks prior to enrolment. * Patients with HBwI with medical records of a total of at least 26 weeks of on demand treatment within the last 52 weeks prior to enrolment * Patients with HBwI regardless of the regimen and duration of previous haemophilia treatment * Patients without inhibitors with medical records of a total of at least 26 weeks of PPX treatment within the last 52 weeks prior to enrolment - For arm 2 only: Male patients (regardless of age) previously treated with concizumab via compassionate use. (a) For patients that have been diagnosed with haemophilia less than 1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available Are the trial subjects under 18? yes Number of subjects for this age range: 95 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 1 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Known or suspected hypersensitivity to study intervention or related products. - Known inherited or acquired coagulation disorder other than congenital haemophilia. - Ongoing or planned Immune Tolerance Induction treatment. - History of thromboembolic disease(a). 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(b) (a) 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. (b) 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: To establish superiority of concizumab prophylaxis in concizumab-naïve children less than 12 years with haemophilia A or B with inhibitors compared to their previous on demand treatment on the number of treated spontaneous and traumatic bleeding episodes.;Secondary Objective: - To establish non-inferiority of concizumab prophylaxis in concizumab-naïve children less than 12 years with haemophilia A or B without inhibitors compared to their previous PPX treatment on the number of treated spontaneous and traumatic bleeding episodes - To evaluate the safety of concizumab administered once-daily in concizumab-naïve children less than 12 years with haemophilia A or B with or without inhibitors - To evaluate the safety of concizumab administered once-daily in patients previously treated with concizumab via compassionate use - To evaluate pharmacokinetics and pharmacodynamics of concizumab administered once-daily in concizumab-naïve children less than 12 years with haemophilia A or B with or without inhibitors;Primary end point(s): For inhibitor patients with at least 26 weeks on demand treatment during the last year before enrolment: The number of treated spontaneous and traumatic bleeding episodes;Timepoint(s) of evaluation of this end point: From start of treatment (week 0) up until the analysis cut-off (a) (a) The analysis cut-off is defined as the point in time when all inhibitor patients in arm 1 have completed the week 32 visit (or withdrawn) and all non-inhibitor patients in arm 1 have completed the week 40 visit (or withdrawn). The individual patient’s cut-off will be the last completed visit before the cut-off date. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. For inhibitor patients with at least 26 weeks on demand treatment during the last year before enrolment: The number of all bleeding episodes (spontaneous and traumatic) 2. For inhibitor patients with at least 26 weeks on demand treatment during the last year before enrolment: Number of treated spontaneous bleeding episodes 3. For inhibitor patients with at least 26 weeks on demand treatment during the last year before enrolment: Number of treated joint bleeding episodes 4. For inhibitor patients with at least 26 weeks on demand treatment during the last year before enrolment: Number of treated bleeding episodes in baseline target joints 5. For non-inhibitor patients with at least 26 weeks PPX treatment during the last year before enrolment: Number of treated spontaneous and traumatic bleeding episodes 6. For non-inhibitor patients with at least 26 weeks PPX treatment during the last year before enrolment: The number of all bleeding episodes (spontaneous and traumatic) 7. For non-inhibitor patients with at least 26 weeks PPX treatment during the last year before enrolment: Number of treated spontaneous bleeding episodes 8. For non-inhibitor patients with at least 26 weeks PPX treatment during the last year before enrolment: Number of treated joint bleeding episodes 9. For non-inhibitor patients with at least 26 weeks PPX treatment during the last year before enrolment: Number of treated bleeding episodes in baseline target joints 10. Number of treatment emergent adverse events 11. Number of thromboembolic events 12. Number of hypersensitivity type reactions 13. Number of injection site reactions 14. Number of patients who develop antibodies to concizumab – yes/no 15. Number of treatment emergent adverse events 16. Concizumab plasma concentrations prior to dosing 17. Peak thrombin generation prior to dosing 18. Free TFPI concentration prior to dosing 19. Pre-dose (trough) concizumab plasma concentration (Ctrough) 20. Maximum con | — |
Countries
Bosnia and Herzegovina, Bulgaria, Canada, Estonia, European Union, France, Greece, India, Italy, Japan, Lithuania, North Macedonia, Norway, Poland, Russian Federation, Sweden, Thailand, Turkey, Ukraine, United Kingdom, United States
Contacts
Public ContactClinical Transparency (1452)
Novo Nordisk A/S
Outcome results
None listed