A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors
- Conditions
- Haemophilia B With InhibitorsCongenital Bleeding DisorderHaemophilia A With Inhibitors
- Interventions
- Registration Number
- NCT03196284
- Lead Sponsor
- Novo Nordisk A/S
- Brief Summary
This trial is conducted in Africa, Asia, Europe and North America. The aim of the trial is to assess the efficacy of concizumab administered s.c. (subcutaneously, under the skin) once daily in preventing bleeding episodes in haemophilia A and B patients with inhibitors.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 26
- 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 the suitability for the trial - Male haemophilia A or B patients with inhibitors aged 18 years or older at the time of signing informed consent - Patients currently in need of treatment with bypassing agents
- Known or suspected hypersensitivity to trial product(s) or related products - Known inherited or acquired bleeding disorder other than haemophilia - Ongoing or planned immune tolerance induction therapy or prophylaxis with FVIII or FIX
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Eptacog alfa and concizumab Eptacog alfa Eptacog alfa administered on-demand during bleeding episodes as the only intervention during the main phase. Concizumab given in the extension phase Concizumab Concizumab Concizumab administered in both the main phase and extension phase, with eptacog alfa administered on-demand during bleeding episodes Concizumab Eptacog alfa Concizumab administered in both the main phase and extension phase, with eptacog alfa administered on-demand during bleeding episodes Eptacog alfa and concizumab Concizumab Eptacog alfa administered on-demand during bleeding episodes as the only intervention during the main phase. Concizumab given in the extension phase
- Primary Outcome Measures
Name Time Method The Number of Bleeding Episodes During at least 24 weeks from treatment onset (week 0) The number of bleeding episodes that were treated during at least 24 weeks from treatment onset (week 0) are presented.
- Secondary Outcome Measures
Name Time Method The Number of Bleeding Episodes During at least 76 weeks from treatment onset (week 0) The number of bleeding episodes that were treated during at least 76 weeks from treatment onset (week 0) are presented. This outcome measure is applicable for only 'Concizumab' treatment arm.
The Number of Spontaneous Bleeding Episodes During at least 76 weeks from treatment onset (week 0) Bleeds that were not linked to a specific, known action or event are called spontaneous bleeding episodes. The number of spontaneous bleeding episodes that were treated during at least 76 weeks from treatment onset (week 0) are presented. The data is presented per the last dose level which the participants have reached at the time of assessment.
Number of Treatment-emergent Adverse Events (TEAEs) During at Least 24 Weeks From Treatment Onset During at least 24 weeks from treatment onset (week 0) An adverse event (AE) was any untoward medical occurrence in a participant administered a medicinal product, and which does not necessarily had a causal relationship with this treatment. A TEAE was defined as an event that had onset from the first exposure to treatment until the last visit in the trial. Number of TEAEs that occurred during at least 24 weeks from treatment onset (week 0) are presented. The data is presented per the dose level which the participants have reached at the time of event.
Number of Treatment-emergent Adverse Events (TEAEs) Within 24 Hours After Eptacog Alfa Administration Within 24 hours after eptacog alfa administration An adverse event (AE) was any untoward medical occurrence in a participant administered a medicinal product, and which does not necessarily had a causal relationship with this treatment. A TEAE was defined as an event that had onset from the first exposure to treatment until the last visit in the trial. Number of TEAEs that occurred within 24 hours after eptacog alfa administration are presented. This outcome measure is applicable only for 'Eptacog alfa' treatment arm.
Change in D-dimer During at least 76 weeks from treatment onset (week 0) Change in D-dimer during at least 76 weeks from treatment onset (week 0) is presented. The data is presented per the last dose level which the participants have reached at the time of assessment.
Change in Prothrombin Time (PT) During at least 76 weeks from treatment onset (week 0) Change in PT during at least 76 weeks from treatment onset (week 0) is presented. The data is presented per the last dose level which the participants have reached at the time of assessment.
Change in Anti-thrombin (AT) After at least 76 weeks from treatment onset (week 0) Change in AT after at least 76 weeks from treatment onset (week 0) is presented. The data is presented per the last dose level which the participants have reached at the time of assessment.
Free Tissue Factor Pathway Inhibitor (TFPI) Concentration Value Prior to the last dose administration after atleast 76 weeks Free TFPI concentration value prior to the last dose administration after atleast 76 weeks is presented. The data is presented per the last dose level which the participants have reached at the time of assessment.
Number of Treatment-emergent Adverse Events (TEAEs) During at Least 76 Weeks From Treatment Onset During at least 76 weeks from treatment onset (week 0) An adverse event (AE) was any untoward medical occurrence in a participant administered a medicinal product, and which does not necessarily had a causal relationship with this treatment. A TEAE was defined as an event that had onset from the first exposure to treatment until the last visit in the trial. Number of TEAEs that occurred during at least 76 weeks from treatment onset (week 0) are presented. The data is presented per the dose level which the participants have reached at the time of event.
Peak Thrombin Generation Prior to the last dose administration after atleast 76 weeks Peak thrombin generation is the maximal concentration of thrombin formed at a given point in time. Peak thrombin generation prior to the last dose administration after atleast 76 weeks is presented. The data is presented per the last dose level which the participants have reached at the time of assessment.
Endogenous Thrombin Potential Prior to the last dose administration after atleast 76 weeks Endogenous thrombin potential prior to the last dose administration after at least 76 weeks is presented. The data is presented per the last dose level which the participants have reached at the time of assessment.
Change in Fibrinogen During at least 76 weeks from treatment onset (week 0) Change in fibrinogen during at least 76 weeks from treatment onset (week 0) is presented. The data is presented per the last dose level which the participants have reached at the time of assessment.
Change in Prothrombin Fragment 1 + 2 (F1 + 2) During at least 76 weeks from treatment onset (week 0) Change in F1 + 2 during at least 76 weeks from treatment onset (week 0) is presented. The data is presented per the last dose level which the participants have reached at the time of assessment.
Change in Activated Partial Thromboplastin Time (APTT) During at least 76 weeks from treatment onset (week 0) Change in APTT during at least 76 weeks from treatment onset (week 0) is presented. The data is presented per the last dose level which the participants have reached at the time of assessment.
Concentration of Concizumab Prior to the last dose administration after atleast 76 weeks Concentration of concizumab prior to the last dose administration after atleast 76 weeks is presented. The data is presented per the last dose level which the participants have reached at the time of assessment.
Occurrence of Anti-concizumab Antibodies During at Least 24 Weeks From Treatment Onset During at least 24 weeks from treatment onset (week 0) Occurrence of anti-concizumab antibodies during at least 24 weeks from treatment onset (week 0) is presented. This outcome measure is applicable for only 'Concizumab' treatment arm.
Occurrence of Anti-concizumab Antibodies During at Least 76 Weeks From Treatment Onset During at least 76 weeks from treatment onset (week 0) Occurrence of anti-concizumab antibodies during at least 76 weeks from treatment onset (week 0) is presented. This outcome measure is applicable for only 'Concizumab' treatment arm.
Thrombin Generation Velocity Index Prior to the last dose administration after atleast 76 weeks Thrombin generation velocity index prior to the last dose administration after at least 76 weeks is presented. The data is presented per the last dose level which the participants have reached at the time of assessment.
Trial Locations
- Locations (1)
Novo Nordisk Investigational Site
🇬🇧Sheffield, United Kingdom