A Phase III Study on the Safety, Pharmacokinetics, and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Pediatric Patients from birth to <12 years old with Inhibitors to Factor VIII or IX: PerSept 2 - PerSept 2

FB USA, Inc.0 sites24 target enrollmentStarted: February 29, 2016Last updated: February 28, 2019

Trial Snapshot

No summary available.

•1. be male with a diagnosis of congenital hemophilia A or B of any severity
•2. have one of the following:
•a. a positive inhibitor test BU =5, OR
•b. a BU<5 but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the patient’s medical history, precluding the use of factor VIII or IX products to treat bleeding episodes, OR
•c. a BU<5 but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the patient’s medical history, precluding the use of factor VIII or IX products to treat bleeding episodes
•3. be aged from birth to <12 years of age
•4. have experienced at least 3 bleeding episodes of any severity in the past 6 months; if < 6 months old, have experienced at least 3 bleeding episodes since birth
•5. parents or legal guardians must be capable of understanding and willing to comply with the conditions of the protocol
•6. parents or legal guardians must have read, understood and provided written informed consent
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 24
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1. have any coagulation disorder other than hemophilia A or B
•2. be immunosuppressed (i.e., the patient should not be receiving systemic immunosuppressive medication, CD4 counts at screening should be >200/µl)
•3. have a known allergy or hypersensitivity to rabbits
•4. have platelet count <100,000/µL
•5. have had a major surgical procedure (e.g. orthopedic, abdominal) within 1 month prior to first administration of the study drug in this study
•6. 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
•7. have a clinically relevant hepatic (AST and/or ALT >3 times ULN) and/or renal impairment (creatinine >2 times ULN)
•8. have an active malignancy (those with non-melanoma skin cancer are allowed)
•9. have any life-threatening disease or other disease or condition which, in the investigator’s judgment, could imply a potential hazard to the patient, or interfere with the trial participation or trial outcome (e.g., a history of non-responsiveness to bypassing products or thromboembolic disease)
•10. known or suspected hypersensitivity to the active substance or to any of its excipients

Sponsor

FB USA, Inc.