A Phase 3, Prospective, Open-label, Randomized Study to Evaluate Safety and Efficacy of Recombinant Activated FVII BI in the Treatment of Acute Bleeding Episodes per an On-demand Regimen in Patients with Hemophilia A or B with Inhibitors

Conditions: Hemophilia A or B with FVIII or FIX inhibitors
MedDRA version: 14.1Level: LLTClassification code 10053751Term: Hemophilia A with anti factor VIIISystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
MedDRA version: 14.1Level: LLTClassification code 10053752Term: Hemophilia B with anti factor IXSystem Organ Class: 100000004850

Registration Number: EUCTR2011-006294-26-PL

Lead Sponsor: Baxter Innovations GmbH

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: 50

Inclusion Criteria

• Subject is male with hemophilia A or B with inhibitors, with a high titer (=5 BU) or a historical high anamnestic response.
• Subject is 12 to 65 years old at the time of screening.
• Subject is currently using or has used bypassing agents for treatment of bleeding episodes.
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 40
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• Subject has a known hypersensitivity to rFVIIa, hamster or murine
proteins, or Tween 80.
• Subject has a prior history of thromboembolic event
• Subject is positive for a FVII inhibitor at screening

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the safety and efficacy of rFVIIa BI in the treatment of acute bleeding episodes per an on demand regimen in hemophilia A or B patients with inhibitors;Secondary Objective: To determine or evaluate:<br>• Treatment response based on a four-point scale <br>• Total percentage of subjects with sustained bleeding control for all acute bleeding episodes at 24 hours after infusion<br>• Safety and tolerability of treatment regimens<br>• Inhibitor development to FVII<br>• Treatment success rate of joint vs. non joint bleeding episodes<br>• Treatment success rates of severe vs. mild/moderate bleeds;Primary end point(s): The primary outcome measure is no additional hemostatic product required within 12 hours of the first dose of IP, other than the prescribed dosing regimen (up to 3 × 90 µg/kg doses or 1 × 270 µg/kg dose).;Timepoint(s) of evaluation of this end point: The endpoint is evaluated 12 hours after first dose.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): • Treatment response based on a four-point scale <br>• Total percentage of clinical responders (sustained bleeding control) for all acute bleeding episodes at 24 hours after infusion<br>• Safety and tolerability of treatment regimens by clinical assessment of related AEs<br>• Inhibitor development to FVII;Timepoint(s) of evaluation of this end point: Treatment response at the end of treatment and 24 hours<br>Safety endpoint continuously

Related Trials