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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01775618
Recruitment Status : Active, not recruiting
First Posted : January 25, 2013
Last Update Posted : February 26, 2018
Information provided by (Responsible Party):

Brief 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.

Condition or disease Intervention/treatment Phase
Hemophilia A Biological: BAY94-9027 Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 73 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (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
Actual Study Start Date : May 29, 2013
Primary Completion Date : March 19, 2015
Estimated Study Completion Date : February 17, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia
U.S. FDA Resources

Arm Intervention/treatment
Experimental: BAY 94-9027
Patients will receive BAY 94-9027 intravenous prophylaxis injection in the main study.
Biological: BAY94-9027
Prophylaxis treatment 25-60 IU/kg at least 1x/week.
Experimental: Expansion group (Part 2)
Patients in expansion group will receive BAY 94-9027 intravenous prophylaxis injection in study part 2 .
Biological: BAY94-9027
Prophylaxis treatment 25-60 IU/kg 2x/week for 12 weeks.

Primary Outcome Measures :
  1. Annualized number of all bleeds [ Time Frame: Up to 11 months ]
  2. 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 ]
    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.

  3. Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent) [ Time Frame: Up to 11 months ]
  4. Characterization of a potential immune response [ Time Frame: Up to 12 weeks ]

Secondary Outcome Measures :
  1. Inhibitor development after 10 to 15 EDs (exposure days) [ Time Frame: Up to 15 weeks ]
  2. Inhibitor development after 50 EDs [ Time Frame: Up to 11 months ]
  3. Number of participants with adverse events as a measure of safety and tolerability [ Time Frame: Up to 11 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 12 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01775618

  Show 37 Study Locations
Sponsors and Collaborators
Study Director: Bayer Study Director Bayer

Additional Information:
Responsible Party: Bayer
ClinicalTrials.gov Identifier: NCT01775618     History of Changes
Other Study ID Numbers: 15912
2012-004434-42 ( EudraCT Number )
First Posted: January 25, 2013    Key Record Dates
Last Update Posted: February 26, 2018
Last Verified: February 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Bayer:
Hemophilia A
Factor VIII

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII