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Phase 3 Completed N=27 Randomized Treatment

Phase III Study of Coagulation FVIIa (Recombinant) in Congenital Hemophilia A or B Patients With Inhibitors

Hemophilia A With Inhibitors · Hemophilia B With Inhibitors
Source: ClinicalTrials.gov NCT02020369 ↗
Enrolled (actual)
27
Serious AEs
2.0%
Results posted
Jun 2017
Primary outcomePrimary: Proportion of Successfully Treated Mild/Moderate Bleeding Episodes — .849; .932 Proportion of Success of BEs
◆ Published Evidence
Not yet cited
0citations
Treatment of severe bleeds with eptacog beta in hemophilia A or B with inhibitors: a post hoc analysis of the PERSEPT 1 and 2 trials.
Blood vessels, thrombosis & hemostasis · 2025 · Open access · Likely link

Summary

The purpose of the study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII/IX

Linked Publications (2)

  • Treatment of severe bleeds with eptacog beta in hemophilia A or B with inhibitors: a post hoc analysis of the PERSEPT 1 and 2 trials.
    Blood vessels, thrombosis & hemostasis · 2025 · 0 citations · Open access · Likely link
  • Safety and Use of Eptacog Beta 225 µg/kg in Patients With Haemophilia A or B With Inhibitors.
    Haemophilia : the official journal of the World Federation of Hemophilia · 2025 · 0 citations · Open access · Likely link

Outcome Measures

OutcomeResultp-value
PRIMARY
Proportion of Successfully Treated Mild/Moderate Bleeding Episodes
.849; .932
SECONDARY
Proportion of Mild/Moderate Bleeding Episodes With Patient (Pt)-Reported "Good" or "Excellent" Responses at 12 Hours
0.857; 0.937
SECONDARY
Time to Assessment of a "Good" or "Excellent" Response of Mild/Moderate Bleeding Episodes by the Patient
5.98; 3.00
SECONDARY
Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode
2.5; 1.4
SECONDARY
Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode
187.868; 252.963

Eligibility Criteria

Inclusion Criteria

  • be male with a diagnosis of congenital hemophilia A and/or B of any severity
  • have one of the following:
  • a positive inhibitor test Bethesda Unit (BU) ≥ 5 (as confirmed at screening by the institutional lab), OR
  • a BU 200/µl)
  • have a known allergy or hypersensitivity to rabbits
  • have platelet count 3 times the upper limit of normal) and/or renal impairment (creatinine >2 times the upper limit of normal)
  • have a history of arterial and/or venous thromboembolic events (such as myocardial infarction, ischemic strokes, transient ischemic attacks, deep venous thrombosis or pulmonary embolism) within 2 years prior to first dose of study drug, or current New York Heart Association (NYHA) functional classification score of stage II -IV
  • have an active malignancy (those with non-melanoma skin cancer are allowed)
  • have any life-threatening disease or other disease or condition which, according to the investigator's judgment, could imply a potential hazard to the patient, interfere with the trial participation or trial outcome (e.g., a history of non-responsiveness to bypassing products).
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02020369) 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.

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