Phase 3
Completed N=240
Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A
Source: ClinicalTrials.gov NCT01454739 ↗Enrolled (actual)
240
Serious AEs
25.0%
Results posted
Nov 2018
Primary outcomePrimary: Number of Participants With Any Positive Inhibitor Development — 0; 0; 0 Participants
◆ Published Evidence
Established
74citations · ~12 / year
Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study.
Summary
The primary objective of the study is to evaluate the long-term safety of recombinant human Factor VIII Fc fusion protein (rFVIIIFc) in participants with hemophilia A. The secondary objective of the study is to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in participants with hemophilia A.
Linked Publications (3)
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Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study.
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Long-term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A.
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Long-term efmoroctocog alfa prophylaxis improves perceived pain, mental, and physical health in patients with hemophilia A: <i>post hoc</i> analysis of phase III trials using patient-reported outcomes.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Any Positive Inhibitor Development |
0; 0; 0 | — |
| SECONDARY Annualized Bleeding Rate (ABR) |
1.18; 1.59; 0.64; 1.90; 3.72; 1.01 | — |
| SECONDARY Annualized Spontaneous Joint Bleeding Episodes |
0.00; 0.00; 0.00; 0.58; 2.20; 0.00 | — |
| SECONDARY Total Number of Exposure Days (EDs) |
287.00; 373.00; 257.00; 203.50; 127.00; 107.0 | — |
| SECONDARY Annualized rFVIIIFc Consumption (International Units Per Kilogram [IU/kg]) |
5417.9; 4989.7; 4359.8; 3505.2; 5457.1; 4572.3 | — |
| SECONDARY Physicians' Global Assessment of Participant's Response to rFVIIIFc Regimen Using a 4-Point Scale |
403; 1061; 24; 186; 1; 5 | — |
| SECONDARY Participant's Assessment of Response (Excellent or Good Response) to rFVIIIFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale |
139; 155; 475; 260; 9; 1 | — |
Eligibility Criteria
Key Inclusion Criteria
- Subjects who have completed previous rFVIIIFc studies (NCT01181128, NCT02083965, NCT01458106 and NCT02502149)
- Ability to understand purposes and risks of the study and to provide signed and dated informed consent (or assent, as applicable).
Key Exclusion Criteria
- Confirmed positive high-titer inhibitor (≥5.00 BU/mL).
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT01454739) and the linked publication. 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.