Phase 3
Completed N=83
A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A
Source: ClinicalTrials.gov NCT01233258 ↗Enrolled (actual)
83
Serious AEs
2.5%
Results posted
Feb 2014
Primary outcomePrimary: Annualized Number of All Bleeds — 57.7; 4.9 Bleeds per year per participant — p=0.0001
Summary
The objective of the trial is to demonstrate that 2-3 times per week prophylaxis therapy with BAY81-8973 is superior to on-demand therapy with BAY81-8973 in patients with severe Hemophilia A. The hypothesis is that prophylaxis will result in fewer bleeds than on-demand treatment.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Annualized Number of All Bleeds |
57.7; 4.9 | 0.0001 sig |
| SECONDARY Annualized Number of All Bleeds During CS/EP Period |
57.6; 5.1 | 0.0001 sig |
| SECONDARY Annualized Number of All Bleeds During CS/ADJ Period |
59.7; 4.8 | 0.0001 sig |
| SECONDARY Percentage of Bleeds Per Participant Controlled With ≤ 2 Injections in Participants Treated on Demand With rFVIII (BAY81-8973) |
96.8; 100.0 | 0.0001 sig |
Eligibility Criteria
Inclusion Criteria
- Male, aged 12 to 65 years
- Severe hemophilia A
- History of more than 150 exposure days (ED) with clotting factor concentrates
- Currently receiving episodic treatment with FVIII; no regular prophylaxis for more than 6 consecutive months in the past 5 years
- No current Factor VIII inhibitor or history of inhibitor
- Willing to use electronic patient diary
Exclusion Criteria
- Presence of another bleeding disease that is different from hemophilia A
- Thrombocytopenia
- Abnormal renal function
- Presence of active liver disease
- Known hypersensitivity to FVIII
Data sourced from ClinicalTrials.gov (NCT01233258). 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. Informational only — not medical advice.