Phase 4
N=45
A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A
Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT04565236 ↗Enrolled (actual)
45
Serious AEs
4.4%
Results posted
Apr 2025
Primary outcome: Primary: Annualized Bleeding Rate (ABR) of All Bleeding Episodes During Prophylaxis Treatment in Part A — 1.86; 3.38 Bleed per year
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 1 (Biological); Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 2 (Biological); Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 3 (Biological)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- Male
- Sponsor
- Bayer
- Primary completion
- Mar 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Annualized Bleeding Rate (ABR) of All Bleeding Episodes During Prophylaxis Treatment in Part A |
1.86; 3.38 | — |
| PRIMARY Annualized Bleeding Rate (ABR) of All Bleeding Episodes Within 48 Hours of Previous Prophylaxis Infusion in Part B |
0.00; 0.00 | — |
| SECONDARY Annualized Bleeding Rate (ABR) of All Bleeding Episodes Within 48 Hours of Previous Prophylaxis Infusion in Part A |
2.26; 1.36 | — |
| SECONDARY Annualized Bleeding Rate (ABR) of All Bleeding Episodes During Prophylaxis Treatment in Part B |
1.13; 2.05 | — |
| SECONDARY Number of Infusions Per Bleeding Episode |
1.78; 1.45; 14.00; 1.00 | — |
| SECONDARY Number of Surgeries Per Physician's Assessment of Adequacy of Hemostasis in Minor Surgery |
2; 2; 2; 2 | — |
| SECONDARY FVIII In-vivo Recovery in Part B |
1.90; 1.86; 0.91; 1.22; 1.55 | — |
| SECONDARY Factor VIII Inhibitor Development by the Nijmegen Bethesda Assay |
0; 0; 0; 0 | — |
| SECONDARY Number of Participants With Treatment-emergent Adverse Events |
13; 3; 1; 1; 10; 3 | — |
| SECONDARY FVIII In-vivo Recovery in Part A |
1.82; 2.03; 1.86; 2.12; 1.85; 2.06 | — |
| SECONDARY Maximum Observed Concentration of FVIII in Plasma (Cmax) in Part A |
95.20; 114.64; 115.95 | — |
| SECONDARY Area Under the Plasma Concentration of FVIII Versus Time Curve From Zero to Infinity (AUC) in Part A |
1292.97; 1519.38; 1559.29 | — |
| SECONDARY Half-life (t1/2) of FVIII in Plasma in Part A |
10.3655; 11.8605; 11.2630 | — |
Summary
The goal of this study is to gather more information on safety and efficacy of Kovaltry for the prevention and treatment of bleeds in Chinese children, adolescents/adults with severe hemophilia A. In addition, pharmacokinetic parameters of Kovaltry will be assessed in a subset of patients.
Eligibility Criteria
Inclusion Criteria
Part A (PTPs):
- Chinese participants with severe hemophilia A (defined as Factor VIII (FVIII): C 2.0 mg/dL) or active liver disease (alanine aminotransferase/aspartate aminotransferase [ALT/AST] > 5x ULN)
- Human immunodeficiency virus (HIV) positive with an absolute CD4 lymphocyte cell count 14 days) within the last 3 months.
- Requiring any pre-medication to tolerate FVIII infusions (e.g. antihistamines)
- Currently participating in another investigational drug study, or having previously participated in a clinical study involving an investigational drug within 30 days of signing informed consent or participated in completed interventional clinical studies with BAY81-8973 (Kovaltry)
- Planned major surgery, defined as surgery with respiratory assistance and/or general anesthesia
Part B (PUPs/MTPs):
- Any other bleeding disease that is different from hemophilia A (e.g. von Willebrand disease, hemophilia B)
- Platelet count 2× upper limit of normal [ULN]) or active liver disease (alanine aminotransferase [ALT] or aspartate aminotransferase [AST] >5× ULN) based on screening laboratory assessments
- MTPs with history of FVIII inhibitor formation
- Known hypersensitivity to the active substance, mouse or hamster protein
- First treatment with KOVALTRY for high risk bleeding situations (e.g., surgery, intracranial bleed) or requiring intensive or prolonged treatment
- Receiving chemotherapy, immune modulatory drugs other than anti-retroviral chemotherapy, or chronic use of oral or intravenous (IV) corticosteroids (> 14 days) within the last 3 months.
- Requiring any pre-medication to tolerate FVIII infusions (e.g. antihistamines)
- Currently participating in another investigational drug study, or having previously participated in a clinical study involving an investigational drug within 30 days of signing informed consent or participated in completed interventional clinical studies with BAY 81-8973 (Kovaltry)
- Planned major surgery, defined as surgery with respiratory assistance and/or general anesthesia
- Unable to tolerate volume of blood draws required for study participation
Data sourced from ClinicalTrials.gov (NCT04565236). 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.