Overview

This trial is active, not recruiting.

Condition hemophilia a
Treatments bay94-9027
Phase phase 3
Sponsor Bayer
Start date May 2013
End date March 2015
Trial size 77 participants
Trial identifier NCT01775618, 15912, 2012-004434-42

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
bay94-9027
Prophylaxis treatment 25-60 IU/kg at least 1x/week.
(Experimental)
bay94-9027
Prophylaxis treatment 25-60 IU/kg 2x/week for 12 weeks.

Primary Outcomes

Measure
Annualized number of all bleeds
time frame: Up to 11 months
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
Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent)
time frame: Up to 11 months
Characterization of a potential immune response
time frame: Up to 12 weeks

Secondary Outcomes

Measure
Inhibitor development after 10 to 15 EDs (exposure days)
time frame: Up to 15 weeks
Inhibitor development after 50 EDs
time frame: Up to 11 months
Number of participants with adverse events as a measure of safety and tolerability
time frame: Up to 11 months

Eligibility Criteria

Male participants up to 12 years old.

Inclusion Criteria: - Males < 12 years of age - Subjects with severe hemophilia A - Previously treated with FVIII for > 50 exposure days Exclusion Criteria: - Subjects with current evidence of or history of inhibitors to FVIII - Any other inherited or acquired bleeding disorder - Platelet counts < 100,000/mm3 - Creatinine > 2x the upper limit of normal - Aspartate aminotransferase (AST) / Alanine aminotransferase (ALT) > 5x the upper limit of normal

Additional Information

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
Trial information was received from ClinicalTrials.gov and was last updated in September 2016.
Information provided to ClinicalTrials.gov by Bayer.