A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa

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

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 or IX in 12 patients ( birth to <6 years old), and 12 patients (≥6 years old to <12 years old).

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

Inclusion Criteria:

• be male with a diagnosis of congenital hemophilia A or B of any severity
• have one of the following:
• a positive inhibitor test BU ≥5, OR
• a Bethesda Unit (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
• 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
• be aged from birth to <12 years old
• have experienced at least 3 bleeding episodes of any severity in the past 6 months
• parents or legal guardians must be capable of understanding and be willing to comply with the conditions of the protocol
• parents or legal guardians must have read, understood, and provided written informed consent

Exclusion Criteria:

• have any coagulation disorder other than hemophilia A or B
• be immunosuppressed (i.e., the patient may not be receiving systemic immunosuppressive medication; cluster of differentiation 4 (CD4) counts at screening must be >200/µL)
• have a known allergy or hypersensitivity to rabbits
• have platelet count <100,000/mL
• have had a major surgical procedure (e.g. orthopedic, abdominal) within 1 month prior to first administration of study drug
• have received an investigational drug within 30 days of first study drug administration, or be expected to receive such drug during participation in this study