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

July 8, 2013 updated by: Bayer

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

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

143

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
    • Ciudad Auton. de Buenos Aires
      • Buenos Aires, Ciudad Auton. de Buenos Aires, Argentina, C1221ADC
      • Buenos Aires, Ciudad Auton. de Buenos Aires, Argentina, C1425AUM
    • Santa Fe
      • Rosario, Santa Fe, Argentina, S2000CKF
  • Australia
    • Western Australia
      • Perth, Western Australia, Australia, 6000
  • Austria
      • Graz, Austria, 8036
      • Wien, Austria, 1090
  • Belgium
      • Bruxelles - Brussel, Belgium, 1200
      • Bruxelles - Brussel, Belgium, 1020
      • Leuven, Belgium, 3000
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2N 2T9
      • Edmonton, Alberta, Canada, T6G 2C8
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H 2Y9
    • Ontario
      • Hamilton, Ontario, Canada, L8S 4J9
      • Ottawa, Ontario, Canada, K1H 8L6
      • Toronto, Ontario, Canada, M5B 1W8
      • Toronto, Ontario, Canada, M5G 1X8
  • Chile
      • Santiago, Chile
      • Santiago de Chile, Chile
  • Croatia
      • Zagreb, Croatia, 10000
  • Denmark
      • Århus N, Denmark, 8200
  • Estonia
      • Tallin, Estonia, 13419
      • Tallinn, Estonia, 13419
  • France
      • Besancon, France, 25030
      • Le Kremlin-bicetre, France, 94275
      • Lyon Cedex, France, 69437
      • Marseille, France, 13005
      • Montpellier Cedex, France, 34059
      • Paris, France, 75674
      • Paris, France, 75015
      • Rennes Cedex, France, 35033
      • Strasbourg, France, 67091
  • Germany
      • Berlin, Germany, 10249
    • Nordrhein-Westfalen
      • Bonn, Nordrhein-Westfalen, Germany, 53105
      • Münster, Nordrhein-Westfalen, Germany, 48143
  • Israel
      • Tel Hashomer, Israel, 52621
  • Italy
      • Catania, Italy, 95124
      • Firenze, Italy, 50134
      • Milano, Italy, 20122
      • Napoli, Italy, 80131
      • Napoli, Italy, 80144
      • Torino, Italy, 10126
  • Lithuania
      • Klaipeda, Lithuania, 50809
      • Vilnius, Lithuania, 08661
  • Netherlands
      • Groningen, Netherlands, 9713 GZ
      • Nijmegen, Netherlands, 6525 GA
      • Utrecht, Netherlands, 3508 GA
  • New Zealand
      • Auckland, New Zealand, 1023
      • Christchurch, New Zealand, 8001
  • Norway
      • Oslo, Norway, 0027
  • Poland
      • Gdansk, Poland, 80-952
      • Krakow, Poland, 31-501
      • Lodz, Poland, 93-510
      • Poznan, Poland, 61-833
      • Warszawa, Poland, 02-776
  • Romania
      • Brasov, Romania, 50035
      • Bucharest, Romania, 022328
    • Timis
      • Timisoara, Timis, Romania, 300011
  • South Africa
    • Gauteng
      • Johannesburg, Gauteng, South Africa, 2132
  • Spain
      • Barcelona, Spain, 08035
      • Madrid, Spain, 28046
      • Valencia, Spain, 46026
    • Andalucía
      • Sevilla, Andalucía, Spain, 41013
    • Asturias
      • Oviedo, Asturias, Spain, 33006
    • Santa Cruz de Tenerife
      • Sta. Cruz de Tenerife, Santa Cruz de Tenerife, Spain, 38009
  • Switzerland
      • Bern, Switzerland, 3010
  • Taiwan
      • Changhua, Taiwan, 500
      • Taipei, Taiwan, 11217
      • Taipei, Taiwan, 10002
  • Turkey
      • Adana, Turkey, 01-330
      • Antalya, Turkey, 07059
      • Izmir, Turkey, 35-100
  • United Kingdom
      • Birmingham, United Kingdom, B15 2TH
      • London, United Kingdom, SE1 7EH
      • Manchester, United Kingdom, M13 9WL
    • Cambridgeshire
      • Cambridge, Cambridgeshire, United Kingdom, CB2 0QQ
    • South Glamorgan
      • Cardiff, South Glamorgan, United Kingdom, CF14 4XW
    • South Yorkshire
      • Sheffield, South Yorkshire, United Kingdom, S10 2JF
  • United States
    • California
      • Orange, California, United States, 92868
      • Sacramento, California, United States, 95817
      • San Francisco, California, United States, 94143-0324
    • Colorado
      • Aurora, Colorado, United States, 80045
    • Louisiana
      • New Orleans, Louisiana, United States, 70112-2699
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
    • Michigan
      • Detroit, Michigan, United States, 48202
      • East Lansing, Michigan, United States, 48823
    • Minnesota
      • Rochester, Minnesota, United States, 55905
    • Missouri
      • Kansas City, Missouri, United States, 64108-9898
    • Nevada
      • Las Vegas, Nevada, United States, 89109-2803
    • New Mexico
      • Albuquerque, New Mexico, United States, 87131-0001
    • New York
      • Buffalo, New York, United States, 14215
      • Rochester, New York, United States, 14621
    • North Carolina
      • Winston-Salem, North Carolina, United States, 27157-1009
    • Ohio
      • Cincinnati, Ohio, United States, 45229
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104-0506
      • Pittsburgh, Pennsylvania, United States, 15213
    • Texas
      • Houston, Texas, United States, 77030
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years to 70 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of Participants With Less Than 9 Total Bleeds Per Year
Time Frame: up to one year
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.
up to one year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of Participants With Less Than 5 Joint Bleeds Per Year
Time Frame: up to one year
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.
up to one year
Number of Joint Bleeds Per Participant Per Year in Responders
Time Frame: up to one year
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
up to one year

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Bleeds Per Year
Time Frame: up to one year
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
up to one year
Percentage of Bleeds Treated by Various Numbers of Injections
Time Frame: up to one year
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.
up to one year
Total rFVIII Consumption Per Year
Time Frame: up to one year
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
up to one year
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
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.
6 months after start of open label extension period

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Bayer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2008

Primary Completion (Actual)

April 1, 2010

Study Completion (Actual)

October 1, 2010

Study Registration Dates

First Submitted

January 9, 2008

First Submitted That Met QC Criteria

February 25, 2008

First Posted (Estimate)

February 26, 2008

Study Record Updates

Last Update Posted (Estimate)

July 15, 2013

Last Update Submitted That Met QC Criteria

July 8, 2013

Last Verified

July 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 12781
  • 2007-003718-32 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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