BAY81-8973 Pediatric Safety and Efficacy Trial

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01311648
Recruitment Status : Recruiting
First Posted : March 9, 2011
Last Update Posted : December 21, 2018
Information provided by (Responsible Party):

Brief Summary:
The objective of the study is to demonstrate the safety and efficacy of treatment with BAY81-8973 for prophylaxis and breakthrough bleeds in children with severe haemophilia A.

Condition or disease Intervention/treatment Phase
Haemophilia A Biological: Recombinant Factor VIII (BAY81-8973) Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 101 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy of BAY81-8973 in Children With Severe Hemophilia A Under Prophylaxis Therapy
Actual Study Start Date : June 9, 2011
Estimated Primary Completion Date : December 31, 2022
Estimated Study Completion Date : December 31, 2022

Arm Intervention/treatment
Experimental: Arm 1
PTPs prophylaxis treatment 25-50 IU/kg at least 2x/week. PUPs 15 -50 IU/kg at least 1x/week.
Biological: Recombinant Factor VIII (BAY81-8973)
PTPs prophylaxis treatment 25-50 IU/kg at least 2x/week. PUPs 15 -50 IU/kg at least 1x/week.

Primary Outcome Measures :
  1. Annualized number of bleeds within 48 hours (h) after a prophylaxis injection [ Time Frame: Up to 9 months ]

Secondary Outcome Measures :
  1. Incidence of inhibitory antibody [ Time Frame: Up to 9 months ]
  2. Total annualized consumption of FVIII per subject [ Time Frame: Up to 9 months ]
  3. Number of infusions for the treatment of a bleed [ Time Frame: Up to 9 months ]
  4. Recovery of FVIII after infusion [ Time Frame: Up to 9 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:

  • Male, aged </= 12 years
  • Severe hemophilia A defined as < 1% FVIII:C
  • PTPs (previously treated patients) > 50 Exposure days (ED) with any high purity FVIII, or
  • PUPs (previously untreated patients) no prior exposure to any high purity FVIII
  • MTPs (minimally treated patients) not previously having received more than 3 injections with any FVIII concentrate
  • No current evidence or history of inhibitory antibody

Exclusion Criteria:

  • History of FVIII inhibitor formation
  • Diagnosed with other bleeding disorder
  • Platelet count < 100000 cells/μL
  • Kidney or liver dysfunction
  • Known hypersensitivity to any FVIII

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 identifier (NCT number): NCT01311648

Contact: Bayer Clinical Trials Contact
Contact: For trial location information (Phone Menu Options '3' or '4') (+)1-888-84 22937

  Show 71 Study Locations
Sponsors and Collaborators
Study Director: Bayer Study Director Bayer

Additional Information:
Publications of Results:
Responsible Party: Bayer Identifier: NCT01311648     History of Changes
Other Study ID Numbers: 13400
2010-021781-29 ( EudraCT Number )
First Posted: March 9, 2011    Key Record Dates
Last Update Posted: December 21, 2018
Last Verified: December 2018

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

Keywords provided by Bayer:
recombinant factor VIII
pediatric use

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