Phase 2
N=26
A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors
Congenital Bleeding Disorder · Haemophilia A With Inhibitors · Haemophilia B With Inhibitors
Bottom Line
View on ClinicalTrials.gov: NCT03196284 ↗Enrolled (actual)
26
Serious AEs
11.8%
Results posted
Oct 2021
Primary outcome: Primary: The Number of Bleeding Episodes — 47; 77 episodes
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Concizumab (Drug); Eptacog alfa (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- Male
- Sponsor
- Novo Nordisk A/S
- Primary completion
- Sep 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Number of Bleeding Episodes |
256 | — |
| SECONDARY The Number of Bleeding Episodes |
256 | — |
| SECONDARY The Number of Spontaneous Bleeding Episodes |
36; 9; 7 | — |
| SECONDARY The Number of Spontaneous Bleeding Episodes |
36; 9; 7 | — |
| SECONDARY Number of Treatment-emergent Adverse Events (TEAEs) During at Least 24 Weeks From Treatment Onset |
39; 4; 18 | — |
| SECONDARY Number of Treatment-emergent Adverse Events (TEAEs) During at Least 76 Weeks From Treatment Onset |
104; 24; 3 | — |
| SECONDARY Number of Treatment-emergent Adverse Events (TEAEs) Within 24 Hours After Eptacog Alfa Administration |
— | — |
| SECONDARY Occurrence of Anti-concizumab Antibodies During at Least 24 Weeks From Treatment Onset |
3; 14 | — |
| SECONDARY Occurrence of Anti-concizumab Antibodies During at Least 76 Weeks From Treatment Onset |
6; 19 | — |
| SECONDARY Change in Fibrinogen |
-0.14; -0.56; -1.51 | — |
| SECONDARY Change in Fibrinogen |
-0.14; -0.56; -1.51 | — |
| SECONDARY Change in D-dimer |
308; 193; 340 | — |
| SECONDARY Change in D-dimer |
308; 193; 340 | — |
| SECONDARY Change in Prothrombin Fragment 1 + 2 (F1 + 2) |
159; 457; 349 | — |
| SECONDARY Change in Prothrombin Fragment 1 + 2 (F1 + 2) |
159; 457; 349 | — |
| SECONDARY Change in Prothrombin Time (PT) |
-0.4; 0.4; 0.3 | — |
| SECONDARY Change in Prothrombin Time (PT) |
-0.4; 0.4; 0.3 | — |
| SECONDARY Change in Activated Partial Thromboplastin Time (APTT) |
-6.6; 3.7; 20.9 | — |
| SECONDARY Change in Activated Partial Thromboplastin Time (APTT) |
-6.6; 3.7; 20.9 | — |
| SECONDARY Change in Anti-thrombin (AT) |
-3; -5; -1 | — |
| SECONDARY Change in Anti-thrombin (AT) |
-3; -5; -1 | — |
| SECONDARY Concentration of Concizumab |
205.5; 1354.7; 941.7 | — |
| SECONDARY Concentration of Concizumab |
205.5; 1354.7; 941.7 | — |
| SECONDARY Free Tissue Factor Pathway Inhibitor (TFPI) Concentration Value |
30.3; 8.3; 12.2 | — |
| SECONDARY Free Tissue Factor Pathway Inhibitor (TFPI) Concentration Value |
30.3; 8.3; 12.2 | — |
| SECONDARY Peak Thrombin Generation |
79.5; 98.7; 75.7 | — |
| SECONDARY Peak Thrombin Generation |
79.5; 98.7; 75.7 | — |
| SECONDARY Endogenous Thrombin Potential |
1056.5; 1356.9; 1178.0 | — |
| SECONDARY Endogenous Thrombin Potential |
1056.5; 1356.9; 1178.0 | — |
| SECONDARY Thrombin Generation Velocity Index |
10.0; 9.7; 6.7 | — |
| SECONDARY Thrombin Generation Velocity Index |
10.0; 9.7; 6.7 | — |
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.
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 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 Exclusion Criteria: - 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
Data sourced from ClinicalTrials.gov (NCT03196284). 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.