Phase 4
N=70
Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China
HEMOPHILIA B
Bottom Line
View on ClinicalTrials.gov: NCT02336178 ↗Enrolled (actual)
70
Serious AEs
2.9%
Results posted
May 2017
Primary outcome: Primary: Percentage of Participants Who Developed Factor IX Inhibitor — 3.3; 0.0; 0.0; 1.8 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Benefix (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Jul 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants Who Developed Factor IX Inhibitor |
3.3; 0.0; 0.0; 1.8; 0.0; 1.4 | — |
| PRIMARY Number of Participants With Allergic Reactions |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Number of Participants With Thrombotic Events |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
28; 26; 10; 46; 30; 62 | — |
| SECONDARY Annualized Bleeding Rates (ABRs) in Participants Receiving Prophylaxis Treatment With BeneFIX During Their Prophylaxis Period |
5.9; 7.5; 0.3; 6.5; 5.5; 6.5 | — |
| SECONDARY Number of Spontaneous/Non-traumatic Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX |
1.3; 2.6; 2.1; 2.6; 2.1 | — |
| SECONDARY Annualized Bleeding Rates (ABRs) in Participants Receiving On-demand Treatment With BeneFIX During Their On-demand Period |
14.8; 28.2; 6.2; 29.3; 26.3 | — |
| SECONDARY Number of Infusions Resulted in the Following Response to On-demand Treatment of Bleeds: Excellent, Good, Moderate, no Response |
77; 94; 9; 133; 122; 254 | — |
| SECONDARY Number of BeneFIX Infusions to Treat Each New Bleed |
1.7; 1.5; 1.1; 1.5; 1.5; 1.5 | — |
| SECONDARY Average Infusion Dose and Total Factor IX Consumption in On-demand Setting |
4277.8; 6550.0; 1770.0; 4418.8; 11672.9; 9826.1 | — |
| SECONDARY Average Infusion Dose and Total Factor IX Consumption in Prophylaxis Setting |
14709.3; 22102.7; 13846.4; 19224.1; 20628.4; 19224.1 | — |
| SECONDARY Average Infusion Dose and Total Factor IX Consumption in Recovery Setting |
1156.3; 1688.9; 250.0; 1378.8; 1467.3; 1419.8 | — |
| SECONDARY Percentage of Infusions With Less Than Expected Therapeutic Effects (LETEs) in On-demand Setting |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Percentage of Infusions With Less Than Expected Therapeutic Effects (LETEs) in Prophylaxis Setting |
0.1; 0.1; 0; 0.1; 0.2; 0.1 | — |
Summary
The purpose of this post-approval study is to evaluate the safety and efficacy of Benefix in subjects with hemophilia B in usual care settings in China.
Eligibility Criteria
Inclusion Criteria
- Male and/or female subjects with hemophilia B.
- Subjects/parents/legal representatives must be able to comply with study procedures (informed consent/assent process, clinical visits, reporting of infusion and bleed data, reporting of adverse events, etc)
Exclusion Criteria
- Presence of any other bleeding disorder in addition to hemophilia B. Subjects with a past history of, or current factor IX inhibitor. For laboratory-based assessments, any Bethesda inhibitor titer greater than the laboratory's normal range or ≥0.6 Bethesda Unit (BU)/mL.
- Subjects with known hypersensitivity to the active substance or to any of the excipients of BeneFIX.
- Subjects with a known hypersensitivity to Chinese Hamster Ovary cell proteins.
Data sourced from ClinicalTrials.gov (NCT02336178). 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.