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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Bayer
ClinicalTrials.gov Identifier:
NCT01775618
First received: January 23, 2013
Last updated: September 30, 2014
Last verified: September 2014
  Purpose

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. 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 Intervention Phase
Hemophilia A
Biological: BAY94-9027
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 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

Resource links provided by NLM:


Further study details as provided by Bayer:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 50
Study Start Date: May 2013
Estimated Study Completion Date: June 2018
Estimated Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: BAY94-9027 Biological: BAY94-9027
Prophylaxis treatment 25-60 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:

  • 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
  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: NCT01775618

  Show 39 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: NCT01775618     History of Changes
Other Study ID Numbers: 15912, 2012-004434-42
Study First Received: January 23, 2013
Last Updated: September 30, 2014
Health Authority: Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Belgium: The Federal Public Service (FPS) Health, Food Chain Safety and Environment
Bulgaria: Bulgarian Drug Agency
Canada: Health Canada
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Romania: National Medicines Agency
Israel: Ministry of Health
Italy: Ethics Committee
Lithuania: State Medicine Control Agency - Ministry of Health
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration

Keywords provided by Bayer:
Hemophilia A
Factor VIII
Prophylaxis
Pediatric

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Hematologic Diseases
Hemorrhagic Disorders
Factor VIII
Coagulants
Hematologic Agents
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on November 20, 2014