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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.
Sponsor:
Information provided by (Responsible Party):
Bayer
ClinicalTrials.gov Identifier:
NCT01580293
First received: March 28, 2012
Last updated: November 5, 2014
Last verified: November 2014
  Purpose

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 ]

Estimated Enrollment: 130
Study Start Date: April 2012
Estimated Study Completion Date: January 2015
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.

  Eligibility

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

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

  Show 97 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: NCT01580293     History of Changes
Other Study ID Numbers: 13024, 2011-005210-11
Study First Received: March 28, 2012
Last Updated: November 5, 2014
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
Coagulants
Hematologic Agents
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on November 20, 2014