A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A (PROTECT-VIII)

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
ClinicalTrials.gov Identifier:
First received: March 28, 2012
Last updated: December 16, 2015
Last verified: December 2015

Haemophilia A is an inherited disorder in which one of the proteins, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. In a person with haemophilia A, the clotting process is slowed and the person experiences bleeds that can result in serious problems and potential disability.

The current standard treatment for severe haemophilia A is regularly scheduled infusion of FVIII to keep levels high enough to prevent bleeding. 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 evaluated in subjects with severe Hemophilia A.

120-140 patients will receive open label treatment with long-acting rFVIII either on-demand to treat bleeds or prophylactically for 36 weeks in the main trial plus an optional extension to continue treatment for at least 100 total exposure days (ED). Patients on prophylactic treatment will receive study drug at dosing intervals between once and twice a week depending on their observed bleeding. Patients will attend the treatment centre for routine blood samples and be required to keep an electronic diary.

Male patients aged 12-65, with severe hemophilia A, previously treated with FVIII for at least 50 exposure days may be eligible for this study.

Condition Intervention Phase
Hemophilia A
Biological: BAY94-9027
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II/III, Multicenter, Partially Randomized, Open Label Trial Investigating Safety and Efficacy of On-demand and Prophylactic Treatment With BAY94-9027 in Severe Hemophilia A

Resource links provided by NLM:

Further study details as provided by Bayer:

Primary Outcome Measures:
  • Annualized number of total bleeds [ Time Frame: In the period of 36 weeks after Baseline ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Safety variables will be summarized using descriptive statistics based on adverse events collection [ Time Frame: Baseline visit up until end of treatment ] [ Designated as safety issue: Yes ]
  • Quantification of blood loss in major surgery [ Time Frame: Pre-surgery infusion up to 24 hours after surgery ] [ Designated as safety issue: No ]
  • Pharmacokinetic parameters will be measured by Tmax, Cmax, t1/2, AUC, and incremental recovery [ Time Frame: Baseline, 36 weeks ] [ Designated as safety issue: No ]

Enrollment: 141
Study Start Date: April 2012
Estimated Study Completion Date: February 2017
Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Arm 1
On-demand treatment of BAY94-9027 at individual dose and number of infusions based upon location and severity of bleeds
Biological: BAY94-9027
Intravenous infusion of BAY94-9027
Experimental: Arm 2
Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by 2 infusions per week over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED
Biological: BAY94-9027
Intravenous infusion of BAY94-9027
Experimental: Arm 3
Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by infusion every 5 days over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED
Biological: BAY94-9027
Intravenous infusion of BAY94-9027
Experimental: Arm 4
Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by infusion every 7 days over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED
Biological: BAY94-9027
Intravenous infusion of BAY94-9027

Detailed Description:

Subjects in prophylactic treatment arms will undergo clinical evaluation at 10 weeks. Those with adequate control of bleeding will undergo randomization to every 5 or 7 day infusion. Those with continued bleeding will remain in treatment arm and have an increase in dose.

Part B-major surgery - optional sub study included to collect information on efficacy of BAY94-9027 in major surgical setting. Due to rarity of surgery in this population, enrollment to this sub-study may be independent of participation in main study.


Ages Eligible for Study:   12 Years to 65 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male; 12-65 years of age
  • Subjects with severe hemophilia A
  • Previously treated with factor VIII for a minimum of 150 exposure days

Exclusion Criteria:

  • Inhibitors to FVIII (current evidence or history)
  • Any other inherited or acquired bleeding disorder in addition to Hemophilia A
  • Platelet count < 100,000/mm3
  • Creatinine > 2x upper limit of normal or AST/ALT (aspartate aminotransferase/alanine aminotransferase) > 5x 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: NCT01580293

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United States, Arizona
Tucson, Arizona, United States, 85724-5024
United States, Arkansas
Little Rock, Arkansas, United States, 72202
United States, California
Duarte, California, United States, 91010
Sacramento, California, United States, 95817
San Diego, California, United States, 92103-8651
United States, Florida
Jacksonville, Florida, United States, 32207
Miami, Florida, United States, 33136
Pensacola, Florida, United States, 32504
United States, Georgia
Atlanta, Georgia, United States, 30322
United States, Illinois
Chicago, Illinois, United States, 60612
United States, Kentucky
Lexington, Kentucky, United States, 40536
United States, Louisiana
New Orleans, Louisiana, United States, 70112
United States, Massachusetts
Worcester, Massachusetts, United States, 01655
United States, Michigan
Detroit, Michigan, United States, 48202
United States, Minnesota
Minneapolis, Minnesota, United States, 55455
United States, Missouri
Kansas City, Missouri, United States, 64108-9898
United States, New York
Rochester, New York, United States, 14621
Syracuse, New York, United States, 13210
United States, Ohio
Cincinnati, Ohio, United States, 45229-3039
Cleveland, Ohio, United States, 44106-6007
Columbus, Ohio, United States, 43205
United States, Pennsylvania
Hershey, Pennsylvania, United States, 17033
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
Chattanooga, Tennessee, United States, 37403
United States, Texas
Houston, Texas, United States, 77030
San Antonio, Texas, United States, 78207
United States, Virginia
Richmond, Virginia, United States, 23298
Bahia Blanca, Buenos Aires, Argentina, B8001HXM
Corrientes, Argentina, W3410AVV
Córdoba, Argentina, X5004CDT
San Luis, Argentina, D5702GOD
Wien, Austria, 1090
Brugge, Belgium, 8000
Leuven, Belgium, 3000
Canada, Alberta
Edmonton, Alberta, Canada, T6G 2E1
Canada, Ontario
London, Ontario, Canada, N6A 5W9
Ottawa, Ontario, Canada, K1H 8L6
Medellin, Antioquia, Colombia
Medellín, Antioquia, Colombia
Barranquilla, Atlántico, Colombia
Aarhus N, Denmark, 8200
BRON cedex, France, 69677
Marseille, France, 13005
Montpellier Cedex, France, 34295
Paris, France, 75015
Reims Cedex, France, 51092
Rennes Cedex, France, 35033
Heidelberg, Baden-Württemberg, Germany, 69004
Bonn, Nordrhein-Westfalen, Germany, 53127
Mainz, Rheinland-Pfalz, Germany, 55131
Berlin, Germany, 10249
Ramat Gan, Israel, 5262000
Milano, Italy, 20122
Napoli, Italy, 80131
Napoli, Italy, 80144
Parma, Italy, 43100
Roma, Italy, 00161
Torino, Italy, 10126
Nagoya, Aichi, Japan, 466-8560
Nishinomiya, Hyogo, Japan, 663-8501
Kashihara, Nara, Japan, 634-8522
Shinjuku-ku, Tokyo, Japan, 160-0023
Suginami, Tokyo, Japan, 167-0035
Hiroshima, Japan, 734-8551
Korea, Republic of
Busan, Busan Gwang''yeogsi, Korea, Republic of, 602-739
Daejeon, Daejeon Gwang''yeogsi, Korea, Republic of, 302-799
Seoul, Korea, Republic of, 120-752
Seoul, Korea, Republic of, 134-727
Amsterdam, Netherlands, 1105 AZ
Den Haag, Netherlands, 2545 CH
Eindhoven, Netherlands, 5600 PD
Groningen, Netherlands, 9713 GZ
Maastricht, Netherlands, 6229 HX
Oslo, Norway, 0027
Bialystok, Poland, 15-276
Lublin, Poland, 20-081
Warszawa, Poland, 02-776
Wroclaw, Poland, 50-367
Wroclaw, Poland, 50-368
Timisoara, Timis, Romania, 300011
Baia Mare, Romania, 430031
Bucharest, Romania, 011026
Bucharest, Romania, 022328
Singapore, Singapore, 119228
Singapore, Singapore, 169608
Singapore, Singapore, 229 899
Malmö, Sweden, 205 02
Stockholm, Sweden, 171 76
Changhua, Taiwan, 500
Taipei, Taiwan, 11217
Taipei, Taiwan, 100
Ankara, Turkey, 06100
Izmir, Turkey, 35100
United Kingdom
Basingstoke, Hampshire, United Kingdom, RG24 9NA
London, United Kingdom, SE1 7EH
Newcastle Upon Tyne, United Kingdom, NE1 4LP
Sheffield, United Kingdom, S10 2JF
Sponsors and Collaborators
Study Director: Bayer Study Director Bayer
  More Information

Additional Information:
Responsible Party: Bayer
ClinicalTrials.gov Identifier: NCT01580293     History of Changes
Other Study ID Numbers: 13024  2011-005210-11 
Study First Received: March 28, 2012
Last Updated: December 16, 2015
Health Authority: Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Austria: Federal Office for Safety in Health Care
Belgium: Federal Agency for Medicinal Products and Health Products
Canada: Health Canada
Colombia: INVIMA Instituto Nacional de Vigilancia de Medicamentos y Alimentos
Denmark: Danish Health and Medicines Authority
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
Japan: Pharmaceuticals and Medical Devices 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
Turkey: Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration
Taiwan: Department of Health
Sweden: Medical Products Agency
Singapore: Health Sciences Authority
Korea: Food and Drug Administration

Keywords provided by Bayer:
Hemophilia A, factor VIII, prophylaxis

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
Hematologic Agents
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on May 04, 2016