A multi-center, phase III, non-controlled, open-label trial to evaluate the pharmacokinetics, safety, and efficacy of BAY 94-9027 for prophylaxis and treatment of bleeding in previously treated children (age < 12 years) with severe hemophilia A.

Phase 3

Completed

Conditions: Hemophilia A
10064477

Registration Number: NL-OMON45210

Lead Sponsor: Bayer

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 5

Inclusion Criteria

- Males < 12 years of age
- Subjects with severe hemophilia A
- Previously treated with FVIII for > 50 exposure days
- Subjects in the expansion group < 6 years of age

Exclusion Criteria

- Subjects with current evidence of or history of inhibitors to FVIII
- Any other inherited or acquired bleeding disorder
- Platelet counts < 100,000/mm^3
- Creatinine > 2x the upper limit of normal
- Aspartate aminotransferase (AST) / Alanine aminotransferase (ALT) > 5x the upper limit of normal

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Number of bleeding events during prophylactic treatment<br /><br><br /><br>Assessment of PK, including Cmax, incremental recovery, Mean Residence Time<br /><br>(MRT), Apparent volume of distribution at steady state (Vss), half-life, area<br /><br>under the curve (AUC), and clearance, in at least 12 subjects in each age<br /><br>subgroup, with at least 4 pre-selected time points between pre-dose and 72 h<br /><br>post-infusion.<br /><br><br /><br>Response of acute bleeding events to treatment will be rated using a 4-point<br /><br>scale (poor, moderate, good, or excellent) by the subject/parent or by the<br /><br>treating physician if the subject is hospitalized.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Inhibitor development after 10-15 and 50 ED<br /><br>Assess incremental recovery in all subjects<br /><br>Safety and tolerability assessments</p><br>