NCT01775618

Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A

A Multi-center, Phase III, Non-controlled, Open-label Trial to Evaluate the Pharmacokinetics, Safety, and Efficacy of BAY94-9027 for Prophylaxis and Treatment of Bleeding in Previously Treated Children (Age <12 Years) With Severe Hemophilia A

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 73 (actual)
• Sponsor: Bayer · Industry
• Sex: Male
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

Hemophilia A is an inherited blood disorder in which one protein, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. Hemophilia A causes the clotting process to be slowed and the person experiences bleeds causing serious problems that could lead to disability. The current standard treatment for severe hemophilia A is infusion of FVIII to stop bleeding, or regular scheduled treatment to prevent bleeds from occuring. Due to the short half-life of FVIII, prophylaxis may require treatment as often as every other day. In this trial safety and efficacy of a long-acting recombinant Factor VIII molecule is being evaluated in 50 male subjects, \< 12 years of age, with severe Hemophilia A. These subjects will receive open label treatment with long-acting rFVIII for approximately 6 months (or longer until 50 exposure days) on a regular schedule at least once every 7-days. Doses and dose intervals may be adapted to the subject's clinical need. A second group of patients will receive open label treatment with the same drug for 12 weeks on a regular schedule of 2x/week. Patients will attend the treatment center for routine blood samples and will be required to keep an electronic diary. Subjects will be offered participation in an optional extension study to collect observations for at least an additional 50 exposure days.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
BIOLOGICAL	BAY94-9027	Study drug dosing was adjusted to the clinical needs of each subject in the range of 25-60 IU/kg/administration, intravenous infusion, at least 50 EDs and a minimum of at least 6 months
BIOLOGICAL	BAY94-9027	Twice per week prophylaxis: 25-60 IU/kg, intravenous infusion, for 12 weeks
BIOLOGICAL	BAY94-9027	Study drug dosing was adjusted to the clinical needs of each subject in the range of 25-60 IU/kg/administration, intravenous infusion, at least 50 additional EDs to achieve at least 100 cumulative EDs, or until marketing authorization of the drug

Timeline

• Start date: 2013-05-29
• Primary completion: 2015-03-19
• Completion: 2020-02-19
• First posted: 2013-01-25
• Last updated: 2020-08-21

Locations

37 sites across 17 countries: United States, Argentina, Austria, Belgium, Bulgaria, Canada, Greece, Israel, Italy, Lithuania, Netherlands, New Zealand, Norway, Poland, Romania, Spain, United Kingdom

Regulatory

• FDA-regulated drug study