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

Completed · Phase 3

Conditions studied: Hemophilia A With Inhibitors, Hemophilia B With Inhibitors

In brief

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

Key facts

Study ID

NCT02020369

Run by

rEVO Biologics

People needed

27

Starts

2014-04-01

Expected to finish

2015-08-01

Last updated by the study team

2017-06-14

Who can join

Age: 12 and older, up to 75. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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<5 but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the subject's medical history, precluding the use of Factor VIII or IX products to treat bleedings, OR
• a BU<5 but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the subject's medical history, precluding the use of Factor VIII or IX products to treat bleedings
• be 12 years or older, up to and including 75 years of age (NOTE: different age restrictions may apply per local regulation and/or ethical considerations)
• have at least 3 bleeding episodes of any severity in the past 6 months be capable of understanding and willing to comply with the conditions of the protocol
• have read, understood and provided written informed consent (patient and/or parent(s)/legal guardian(s) if <18 years of age)

You may not qualify if…

• have any coagulation disorder other than hemophilia A or B
• be immuno-suppressed (i.e., the patient should not be receiving systemic immunosuppressive medication, cluster of differentiation 4 (CD4) counts at screening should be >200/µl)
• have a known allergy or hypersensitivity to rabbits
• have platelet count <100,000/mL
• have had within one month prior to first administration of the study drug in this study a major surgical procedure (e.g. orthopedic, abdominal)
• have received an investigational drug within 30 days of the first study drug administration, or is expected to receive such drug during participation in this study
• have a clinically relevant hepatic (AST and/or alanine aminotransferase (ALT) >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).

Where it is running

• Orthopaedic Hemophilia Treatment Center — Los Angeles, California, United States
• University of California Davis Comprehensive Cancer Center — Sacramento, California, United States
• University of Colorado Hemophilia and Thrombosis Center — Aurora, Colorado, United States
• Rush University Medical Center — Chicago, Illinois, United States
• University of Minnesota Medical Center Fairview — Minneapolis, Minnesota, United States
• Republican Research Center for Radiation Medicine and Human Ecology — Homyel, Belarus
• Specialized Hospital for Active Treatment of Hematological Diseases — Sofia, Bulgaria
• LTD HEMA — Tbilisi, Georgia
• Chaim Sheba Medical Center, Tel-hashomer hospital — Ramat Gan, Israel
• Institute of Hematology and Transfusion Medicine — Warsaw, Poland
• Sandor SRL — Bucharest, Romania
• Kirov Research Institute of Hematology and Blood Transfusion — Kirov, Russia
• Hematology Research Center — Moscow, Russia
• City Outpatient Clinic #37 — Saint Petersburg, Russia
• Kyiv City Clinical Hospital #9 — Kyiv, Ukraine
• Institute of Blood Pathology and Transfusion Medicine of Academy of Medical Sciences of Ukraine — Lviv, Ukraine
• Basingstoke and North Hampshire Hospital, Hemophilia, Hemostasis and Thrombosis Center — Basingstoke, United Kingdom

Full record on ClinicalTrials.gov

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