Phase 2
N=36
A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors
Haemostasis · Haemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT03196297 ↗Enrolled (actual)
36
Serious AEs
4.9%
Results posted
May 2021
Primary outcome: Primary: The Number of Bleeding Episodes During at Least 24 Weeks From Treatment Onset — 43; 13; 14 Episodes
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Concizumab (Drug); Turoctocog alfa (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- Male
- Sponsor
- Novo Nordisk A/S
- Primary completion
- Jun 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Number of Bleeding Episodes During at Least 24 Weeks From Treatment Onset |
43; 13; 14 | — |
| SECONDARY The Number of Bleeding Episodes During at Least 76 Weeks From Treatment Onset |
67; 42; 123 | — |
| SECONDARY The Number of Spontaneous Bleeding Episodes During at Least 24 Weeks From Treatment Onset |
16; 8; 2 | — |
| SECONDARY The Number of Spontaneous Bleeding Episodes During at Least 76 Weeks From Treatment Onset |
39; 15; 29 | — |
| SECONDARY Number of Treatment-emergent Adverse Events (TEAEs) During at Least 24 Weeks From Treatment Onset |
105; 16; 9 | — |
| SECONDARY Number of Treatment-emergent Adverse Events (TEAEs) During at Least 76 Weeks From Treatment Onset |
201; 53; 44 | — |
| SECONDARY Occurrence of Anti-concizumab Antibodies During at Least 24 Weeks From Treatment Onset |
3; 33 | — |
| SECONDARY Occurrence of Anti-concizumab Antibodies During at Least 76 Weeks From Treatment Onset |
9; 27 | — |
| SECONDARY Change in Fibrinogen During 24 Weeks From Treatment Onset |
-0.08; -0.19; -0.27 | — |
| SECONDARY Change in Fibrinogen During at Least 76 Weeks From Treatment Onset |
-0.05; -0.35; -0.23 | — |
| SECONDARY Change in D-dimer During 24 Weeks From Treatment Onset |
184.5; 272.9; 703.8 | — |
| SECONDARY Change in D-dimer During at Least 76 Weeks From Treatment Onset |
265.4; 506.7; 1109.3 | — |
| SECONDARY Change in Prothrombin Fragment 1 + 2 (F1 + F2) During 24 Weeks From Treatment Onset |
134; 257; 580 | — |
| SECONDARY Change in Prothrombin Fragment 1 + 2 (F1 + F2) During at Least 76 Weeks From Treatment Onset |
128; 211; 889 | — |
| SECONDARY Change in Prothrombin Time (PT) During 24 Weeks From Treatment Onset |
-0.0; -0.3; 0.3 | — |
| SECONDARY Change in Prothrombin Time (PT) During at Least 76 Weeks From Treatment Onset |
0.0; 0.8; 0.4 | — |
| SECONDARY Change in Activated Partial Thromboplastin Time (APTT) During 24 Weeks From Treatment Onset |
1.5; 3.1; 6.5 | — |
| SECONDARY Change in Activated Partial Thromboplastin Time (APTT) During at Least 76 Weeks From Treatment Onset |
3.1; 9.2; 2.1 | — |
| SECONDARY Change in Anti-thrombin (AT) During 24 Weeks From Treatment Onset |
7; 17; 7 | — |
| SECONDARY Change in Anti-thrombin (AT) After at Least 76 Weeks From Treatment |
0; 11; 15 | — |
| SECONDARY Concentration of Concizumab Prior to the Last Dose Administration at 24 Weeks |
195.2; 374.4; 2640.8 | — |
| SECONDARY Concentration of Concizumab Prior to the Last Dose Administration After at Least 76 Weeks |
195.1; 392.3; 4015.1 | — |
| SECONDARY Free Tissue Factor Pathway Inhibitor (TFPI) Concentration Value Prior to the Last Dose Administration at 24 Weeks |
30.1; 64.4; 12.4 | — |
| SECONDARY Free Tissue Factor Pathway Inhibitor (TFPI) Concentration Value Prior to the Last Dose Administration After at Least 76 Weeks |
26.9; 36.1; 10.1 | — |
| SECONDARY Peak Thrombin Generation Prior to the Last Dose Administration at 24 Weeks |
88.6; 67.5; 83.4 | — |
| SECONDARY Peak Thrombin Generation Prior to the Last Dose Administration After at Least 76 Weeks |
90.8; 99.1; 111.6 | — |
| SECONDARY Endogenous Thrombin Potential Prior to the Last Dose Administration at 24 Weeks |
1229.1; 965.3; 1176.0 | — |
| SECONDARY Endogenous Thrombin Potential Prior to the Last Dose Administration After at Least 76 Weeks |
1253.0; 1352.6; 1233.4 | — |
| SECONDARY Thrombin Generation Velocity Index Prior to the Last Dose Administration at 24 Weeks |
9.3; 7.0; 8.2 | — |
| SECONDARY Thrombin Generation Velocity Index Prior to the Last Dose Administration After at Least 76 Weeks |
10.3; 10.5; 16.0 | — |
Summary
This trial is conducted in Asia, Europe and the United States of America (USA). 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 patients with severe haemophilia A without inhibitors.
Eligibility 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 the suitability for the trial - Male patients aged 18 years or older at the time of signing informed consent, diagnosed with severe haemophilia A (FVIII activity below 1%), based on medical records or results at screening Exclusion Criteria: - Known or suspected hypersensitivity to trial product(s) or related products - Known inherited or acquired bleeding disorder other than haemophilia A - Presence of inhibitors (neutralising antibodies) to Factor VIII (equal to or above 0.6 Bethesda Units) at screening measured by the Nijmegen method
Data sourced from ClinicalTrials.gov (NCT03196297). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.