Phase 3
Completed N=120
Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B
Severe Hemophilia B
Source: ClinicalTrials.gov NCT01425723 ↗
Enrolled (actual)
120
Serious AEs
28.5%
Results posted
Nov 2018
Primary outcomePrimary: Number of Participants With Any Positive Inhibitor Development — 0; 0; 0 Participants
◆ Published Evidence
Emerging
9citations · ~1 / year
Extending recombinant factor IX Fc fusion protein dosing interval to 14 or more days in patients with hemophilia B.
Summary
The primary objective of the study is to evaluate the long-term safety of rFIXFc in participants with hemophilia B.
The secondary objective of this study is to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes.
Linked Publications (3)
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Extending recombinant factor IX Fc fusion protein dosing interval to 14 or more days in patients with hemophilia B.
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Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B.
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Recombinant factor IX Fc prophylaxis reduces pain and increases levels of physical activity, with sustained, long-term improvements in patients with hemophilia B: <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) |
1.04; 1.14; 2.26; 3.69; 1.85; 0.54 | — |
| SECONDARY Annualized Spontaneous Joint Bleeding Episodes |
0.00; 0.00; 0.38; 0.00; 0.38; 0.00 | — |
| SECONDARY Total Number of Exposure Days (EDs) |
55.00; 165.00; 169.00; 54.00; 110.00; 157.00 | — |
| SECONDARY Annualized rFIXFc Consumption (International Units Per Kilogram [IU/kg]) |
3382.5; 3212.0; 2598.0; 3700.7; 2894.8; 3331.7 | — |
| SECONDARY Physicians' Global Assessment of Participant's Response to rFIXFc Regimen Using a 4-Point Scale |
131; 622; 19; 184; 1; 9 | — |
| SECONDARY Participant's Assessment of Response (Excellent or Good Response) to rFIXFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale |
60; 47; 342; 25; 336; 0 | — |
Eligibility Criteria
Key Inclusion Criteria
- Subjects who have completed studies 998HB102 (NCT01027364) or 9HB02PED (NCT01440946) or other studies with rFIXFc
- Ability to understand the purposes & risks of the study and provide signed and dated informed consent.
Key Exclusion Criteria
- High-titer inhibitor (>/=5.00 BU/mL)
NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT01425723) 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.