BAY81-8973 Pediatric Safety and Efficacy Trial

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by Bayer
Sponsor:
Information provided by (Responsible Party):
Bayer
ClinicalTrials.gov Identifier:
NCT01311648
First received: February 18, 2011
Last updated: August 4, 2014
Last verified: August 2014
  Purpose

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 Intervention Phase
Haemophilia A
Biological: Recombinant Factor VIII (BAY81-8973)
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Bayer:

Primary Outcome Measures:
  • Annualized number of bleeds within 48 hours (h) after a prophylaxis injection [ Time Frame: Up to 9 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Incidence of inhibitory antibody [ Time Frame: Up to 9 months ] [ Designated as safety issue: Yes ]
  • Total annualized consumption of FVIII per subject [ Time Frame: Up to 9 months ] [ Designated as safety issue: No ]
  • Number of infusions for the treatment of a bleed [ Time Frame: Up to 9 months ] [ Designated as safety issue: No ]
  • Recovery of FVIII after infusion [ Time Frame: Up to 9 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 75
Study Start Date: June 2011
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Arm 1 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

  Eligibility

Ages Eligible for Study:   up to 12 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
  • No current evidence of inhibitor 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01311648

Contacts
Contact: Bayer Clinical Trials Contact clinical-trials-contact@bayerhealthcare.com
Contact: For trial location information (Phone Menu Options '3' or '4') (+)1-888-84 22937

  Show 55 Study Locations
Sponsors and Collaborators
Bayer
Investigators
Study Director: Bayer Study Director Bayer
  More Information

Additional Information:
No publications provided

Responsible Party: Bayer
ClinicalTrials.gov Identifier: NCT01311648     History of Changes
Other Study ID Numbers: 13400, 2010-021781-29
Study First Received: February 18, 2011
Last Updated: August 4, 2014
Health Authority: Bulgaria: Bulgarian Drug Agency
Canada: Health Canada
Denmark: Danish Medicines Agency
Hungary: National Institute of Pharmacy
Ireland: Irish Medicines Board
Israel: Ministry of Health
Italy: The Italian Medicines Agency
Latvia: State Agency of Medicines
Lithuania: State Medicine Control Agency - Ministry of Health
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Romania: State Medicines Control Agency
Serbia: Medicines and Medical Devices Agency of Serbia
Sweden: Medical Products Agency
United States: Food and Drug Administration
Argentina: Ministry of Health
Austria: Agency for Health and Food Safety
Mexico: Federal Commission for Sanitary Risks Protection
Russia: Ministry of Health of the Russian Federation
Spain: Spanish Agency of Medicines
United Kingdom: Medicines and Healthcare Products Regulatory Agency

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
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 27, 2014