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BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A

This study has been terminated.
Sponsor:
Information provided by:
Bayer
ClinicalTrials.gov Identifier:
NCT00623727
First received: January 9, 2008
Last updated: July 8, 2013
Last verified: July 2013
  Purpose

A study to assess treatment with a new formulation of recombinant factor VIII reconstituted with liposomes (BAY79-4980) to evaluate whether a once-a-week treatment is safe and can prevent bleeds in subjects with severe haemophilia A.


Condition Intervention Phase
Hemophilia A
Biological: rFVIII-FS/pegylated liposomes (BAY79-4980)
Biological: rFVIII-FS/WFI (BAY14-2222)
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Randomized, Active-controlled, Double-blind, Parallel Design Study to Evaluate the Efficacy and Safety of a Once-a-week Prophylaxis Treatment With BAY79-4980 Compared to Three Times-per-week Prophylaxis With rFVIII-FS in Previously Treated Patients With Severe Hemophilia A

Resource links provided by NLM:


Further study details as provided by Bayer:

Primary Outcome Measures:
  • Percentage of Participants With Less Than 9 Total Bleeds Per Year [ Time Frame: up to one year ] [ Designated as safety issue: No ]
    Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event.


Secondary Outcome Measures:
  • Percentage of Participants With Less Than 5 Joint Bleeds Per Year [ Time Frame: up to one year ] [ Designated as safety issue: No ]
    Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event.

  • Number of Joint Bleeds Per Participant Per Year in Responders [ Time Frame: up to one year ] [ Designated as safety issue: No ]
    Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. Responders were the subjects with less than 9 total bleeds per year


Other Outcome Measures:
  • Number of Bleeds Per Year [ Time Frame: up to one year ] [ Designated as safety issue: No ]
    Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. Number of bleeds 3 weeks after the first infusion per 12 months

  • Percentage of Bleeds Treated by Various Numbers of Injections [ Time Frame: up to one year ] [ Designated as safety issue: No ]
    Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event.

  • Total rFVIII Consumption Per Year [ Time Frame: up to one year ] [ Designated as safety issue: No ]
    Total number of units per kg of study medication (rFVIII) administered to participant for one year. rFVIII is recombinant factor VIII, factor VIII is functional coagulation factor

  • Percentage of Participants With Less Than 9 Total Bleeds Per Year in the Open Label Extension Period [ Time Frame: 6 months after start of open label extension period ] [ Designated as safety issue: No ]
    Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event.


Enrollment: 143
Study Start Date: June 2008
Study Completion Date: October 2010
Primary Completion Date: April 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: rFVIII-FS/pegylated liposomes (BAY79-4980)
35 IU/kg body weight of BAY79-4980 1x/week plus 2 dummy injections/week (dummy = rFVIII (recombinant factor VIII)-FS (formulated with sucrose) excipient reconstituted in WFI (sterile water for injection))
Biological: rFVIII-FS/pegylated liposomes (BAY79-4980)
35 IU/kg body weight intravenous 1x/week for 52 weeks This arm will be stopped by 30.04.10 the subjects will be offered to change to the active comparator arm
Active Comparator: rFVIII-FS/WFI (BAY14-2222)
25 IU/kg body weight of rFVIII-FS 3x/week (employing 1 percent POPC (1-palmitoyl-2-oleoyl-sn-glycero-3-phosphocholine)-alone liposome (rFVIII-FS-POPC) as blinding agent used for first weekly injection and rFVIII-FS in WFI for 2nd and 3rd injection)
Biological: rFVIII-FS/WFI (BAY14-2222)
25 IU/kg body weight intravenous 3x/week for 52 weeks

  Eligibility

Ages Eligible for Study:   12 Years to 70 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Males aged 12 to 70 years
  • Subjects with severe hemophilia A (< 1% factor VIII [FVIII]:C)
  • Subjects with equal or greater than 150 exposure days (EDs) with any FVIII in total
  • Subjects who have been on-demand treatment with a minimum of 1 relevant bleed per month or have been on secondary prophylaxis treatment with not more than a 3x/week schedule
  • Subjects with bleeding events and/or treatments during the last 6 months prior to study entry which are documented in the subjects medical records
  • Subjects with no measurable inhibitor activity
  • Subjects with no history of FVIII inhibitor antibody formation
  • Written informed consent by subject and parent / legal representative, if < 18 years

Exclusion Criteria:

  • Subjects who are receiving primary prophylaxis
  • Subjects on prophylaxis with documented requirements of > 75 IU/kg/week
  • Subjects with any other bleeding disease beside hemophilia A (i.e., von Willebrand disease)
  • Subjects with abnormal renal function
  • Subjects with elevated hepatic transaminases
  • Subjects on treatment with immunomodulatory agents within the last 3 months prior to study entry or during the study
  • Subjects with known hypersensitivity to the active substance, mouse or hamster protein, liposomes or polyethyleneglycol (PEG)
  • Subjects who require any pre-medication for FVIII injections
  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: NCT00623727

  Show 97 Study Locations
Sponsors and Collaborators
Bayer
Investigators
Study Director: Bayer Study Director Bayer
  More Information

Additional Information:
Publications:
Responsible Party: Therapeutic Area Head, Bayer HealthCare Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT00623727     History of Changes
Other Study ID Numbers: 12781, 2007-003718-32
Study First Received: January 9, 2008
Results First Received: June 10, 2011
Last Updated: July 8, 2013
Health Authority: Austria: Agency for Health and Food Safety
Belgium: Federal Agency for Medicinal Products and Health Products
Canada: Health Canada
Denmark: Danish Medicines Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Paul-Ehrlich-Institut
Israel: Ministry of Health
Italy: The Italian Medicines Agency
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Norway: Norwegian Medicines Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Spain: Spanish Agency of Medicines
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Croatia: Ministry of Health and Social Care
Turkey: Ministry of Health
United States: Food and Drug Administration

Keywords provided by Bayer:
Hemophilia A
FVIII disease
rFVIII

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 25, 2014