Phase 3 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

Bottom Line

View on ClinicalTrials.gov: NCT02020369 ↗

Enrolled (actual)

Serious AEs

2.0%

Results posted

Jun 2017

Primary outcome: Primary: Proportion of Successfully Treated Mild/Moderate Bleeding Episodes — .849; .932 Proportion of Success of BEs

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Coagulation Factor VIIa (Recombinant) (Biological)
Age: Pediatric, Adult, Older Adult · 12+ yrs
Sex: Male
Sponsor: rEVO Biologics
Primary completion: Jul 2015

Outcome Measures

Outcome	Result	p-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	—

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

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). 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.