Haemophilia A without inhibitor Haemophilia A Concizumab
Conditions
Interventions
The trial is a multicentre single-
arm trial,
which aim to evaluate the efficacy and safety of
concizumab 0.15 mg/kg (with potential dose escalation to 0.20 and 0.25 mg/kg) administered daily
s.c. in patients with severe haemophilia A without inh
demand and prophylaxis patients will be eligible for the trial.
Experimental Drug
Sponsors
Novo Nordisk
Novo Nordisk
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. 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. 2. Male patients aged ≥ 18 years at the time of signing informed consent, diagnosed with severe haemophilia A (FVIII activity <1%), based on medical records or results at screening. 3. For patients being treated on-demand with FVIII replacement therapy, a minimum of six documented and treated bleeding episodes during the 24 weeks (or twelve bleeds during 52 weeks) prior to screening.
Exclusion criteria
Exclusion criteria: 1. Known or suspected hypersensitivity to trial product(s) or related products. 2. Previous participation in this trial. Participation is defined as signed informed consent. 3. Participation in any clinical trial of an approved or non-approved investigational medicinal product within the last 30 days or 5 half-lives (whichever is longer) from the last drug administration before screening. 4. Any disorder, which in the investigator’s opinion might jeopardise patient’s safety or compliance with the protocol. 5. Known inherited or acquired bleeding disorder other than haemophilia A. 6. Major surgery conducted within one month prior to the initiation of trial activities or major surgery planned to occur during the trial. 7. Previous history of thromboembolic disease. Current clinical signs of thromboembolic disease, or patients who in the judgement of the investigator are considered at high risk of thromboembolic events. 8. Mental incapacity, unwillingness to cooperate or language barrier precluding adequate understanding and cooperation. 9. Patients who, at screening, have a significant infection or known systemic inflammatory condition which require systemic treatment according to the investigator’s judgement. 10. Hepatic dysfunction defined as elevated liver transaminases (ALT) >3 times the upper limit of normal laboratory reference ranges at screening. 11. Renal impairment defined as estimated glomerular filtration rate (eGFR) ≤60 mL/min/1.73m2 based on serum creatinine measured at screening or evidence of renal damage. 12. Platelet count ≤ 100x109/L at screening. 13. Fibrinogen level < the lower limit of normal at screening 14. Presence of inhibitors (neutralising antibodies) to Factor VIII (≥ 0.6 Bethesda Units) at screening measured by the Nijmegen method. 15. History of inhibitors towards FVIII based on investigator’s knowledge or documentation in available medical records.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The number of bleeding episodes during at least 24 weeks from treatment during at least 24 weeks from treatment using negative binomial regression with log of exposure | — |
Secondary
| Measure | Time frame |
|---|---|
| Supportive secondary efficacy endpoints 76 weeks from treatment onset The number of bleeding episodes during 76 weeks from treatment onset,Supportive secondary efficacy endpoints 24 weeks from treatment onset The number of spontaneous bleeding episodes during at least 24 weeks from treatment onset,Supportive secondary efficacy endpoints 76 weeks from treatment onset The number of spontaneous bleeding episodes during 76 weeks from treatment onset,Supportive secondary safety endpoints 24 weeks from Number of treatment-emergent adverse events (TEAEs) during at least 24 weeks from,Supportive secondary safety endpoints 76 weeks from treatment onset Number of TEAEs during 76 weeks from treatment onset,Supportive secondary safety endpoints 24 weeks from treatment onset Occurrence of anti-concizumab antibodies during at least 24 weeks from treatment onset,Supportive secondary safety endpoints 76 weeks from treatment onset Occurrence of anti-concizumab antibodies during 76 weeks from treatment onset | — |
Countries
Thailand
Contacts
Public ContactProfessor Pantep Angchaisuksiri
Novo Nordisk
Outcome results
None listed