Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A
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.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||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|
- Annualized number of all bleeds [ Time Frame: Up to 11 months ] [ Designated as safety issue: No ]
- Pharmacokinetics profile of BAY94-9027 based on blood concentration over the defined time period [ Time Frame: 6 time points from pre-infusion to 72 hours post-infusion ] [ Designated as safety issue: No ]Pharmacokinetics profile includes maximum concentration (Cmax), incremental recovery, mean residence time (MRT), apparent volume of distribution at steady state (Vss), half-life, area under the curve (AUC), and clearance, which are calculated values based on time-weighed blood concentration of drug.
- Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent) [ Time Frame: Up to 11 months ] [ Designated as safety issue: No ]
- Characterization of a potential immune response [ Time Frame: Up to 12 weeks ] [ Designated as safety issue: Yes ]
- Inhibitor development after 10 to 15 EDs (exposure days) [ Time Frame: Up to 15 weeks ] [ Designated as safety issue: No ]
- Inhibitor development after 50 EDs [ Time Frame: Up to 11 months ] [ Designated as safety issue: No ]
- Number of participants with adverse events as a measure of safety and tolerability [ Time Frame: Up to 11 months ] [ Designated as safety issue: Yes ]
|Study Start Date:||May 2013|
|Estimated Study Completion Date:||December 2019|
|Primary Completion Date:||March 2015 (Final data collection date for primary outcome measure)|
|Experimental: Arm 1||
Prophylaxis treatment 25-60 IU/kg at least 1x/week.
|Experimental: Arm 2||
Prophylaxis treatment 25-60 IU/kg 2x/week for 12 weeks.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01775618
|Contact: Bayer Clinical Trials Contactfirstname.lastname@example.org|
|Contact: For trial location information (Phone Menu Options '3' or '4')||(+)1-888-84 22937|
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|Study Director:||Bayer Study Director||Bayer|