Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors
Phase 3
Active, not recruiting
NCT04082429
| Condition | Haemophilia A Without Inhibitors, Haemophilia B Without Inhibitors |
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| Phase | Phase 3 |
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| Sponsor | Novo Nordisk A/S |
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| Status | Active, not recruiting |
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| Enrollment | 156 participants |
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| Interventions | Concizumab |
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| Start date | Nov 13, 2019 |
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| Completion date | Jul 12, 2022 |
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| Results posted | Oct 31, 2025 |
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Summary
This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B without inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will be placed in one of two groups. In one group participants will get study medicine from the start of the study. In the other group participants will continue with their normal medicine and get study medicine after 6 months. Which treatment the participant gets is decided by chance. Participants who usually take medicine to prevent bleeds (prophylaxis treatment) or who are already being treated with concizumab (study medicine) will receive the study medicine from the start of the study. Participants will have to inject themselves with the study medicine 1 time every day under the skin. This can be done at home. The study doctor will hand out the medicine in the form of a pen-injector. The pen-injector will contain the study medicine. The study will last for up to 8 years. The length of time the participant will be in the study depends on when they agreed to take part and when the medicin
Haemophilia A Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Bleeding Episodes
On demand (arm 1): From week 0 up until start of concizumab treatment (week 24); Concizumab (arm 2): From week 0 up until the confirmatory analyses cut-off · Events per year
| Arm 1: Previous On Demand (OnD) Treatment: No Prophylaxis (PPX) - Participants With HA | 19.6 Events per year |
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| Arm 2: Previous OnD Treatment: Concizumab PPX - Participants With HA | 2.9 Events per year |
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| p-value | <0.001 |
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| ABR ratio | 0.14 (95% CI 0.07–0.29) |
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Haemophilia B Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Bleeding Episodes
On demand (arm 1): From week 0 up until start of concizumab treatment (week 24); Concizumab (arm 2): From week 0 up until the confirmatory analyses cut-off · Events per year
| Arm 1: Previous OnD Treatment: No PPX - Participants With HB | 14.9 Events per year |
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| Arm 2: Previous OnD Treatment: Concizumab PPX - Participants With HB | 1.6 Events per year |
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| p-value | <0.001 |
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| ABR ratio | 0.21 (95% CI 0.10–0.45) |
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Open in BENITIONS
View on ClinicalTrials.gov
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