A Phase 2/ 3 Trial to Evaluate the Efficacy and Safety of BAY86-6150

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Bayer
ClinicalTrials.gov Identifier:
NCT01625390
First received: June 19, 2012
Last updated: July 6, 2014
Last verified: July 2014
  Purpose

Haemophilia is a disorder, usually genetic, affecting mostly male individuals, in which one of the proteins needed to form blood clots (FVIII) is missing or not present in sufficient levels. In a person with haemophilia, the clotting process is much slower and the person experiences bleeding episodes that can result in serious problems and potential disability.

The current haemophilia standard of care is to maintain FVIII activity level above 1%. Sometimes, patients can develop antibodies (so called "inhibitors") against FVIII and it is no longer effective at controlling bleeds. Bleeds in these patients are currently treated using other proteins involved in the clotting process.

The purpose of this study is to investigate how effectively BAY86-6150 may stop acute bleeds in "inhibitor" patients. This study consists of two parts, A and B. The purpose of part A is to find the most effective yet tolerable out of four doses of BAY86-6150 with regard to efficacy and safety (dose-finding part). Part A is expected to last 9 - 29 months. The purpose of part B is to confirm efficacy and safety of the dose found in part A in all participating patients (confirmatory part). Part B is expected to last 12-32 months.

Approximately 60 male subjects 12 to 62 years-of-age with moderate or severe haemophilia A or B, with inhibitors to FVIII or FIX, who have had 4 or more bleeding episodes in the last 6 months, will participate in this study.

Patient's bleeds will be treated with BAY86-6150 and with a rescue medication if no response is made to BAY86-6150. Patients will attend the treatment centre at regular intervals and be required to keep an electronic diary.


Condition Intervention Phase
Hemophilia A, Hemophilia B
Drug: BAY86-6150
Drug: eptacog alfa [activated]
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 2/3, Multicenter, Open-label Clinical Study to Assess the Safety and Efficacy of BAY86-6150 in Subjects With Hemophilia A or B With Inhibitors, Composed of 2 Parts (A & B). Part A: Sequential Cohorts of Four Dose Levels of the Modified rFVIIa BAY86-6150 Assessed in a Non-controlled Dose Response Design in Acutely Bleeding Subjects and for PK/ PD in an Intra-individual Crossover Design Compared With One Fixed Dose of Eptacog Alfa in Non-bleeding Subjects. Part B: Confirmatory Study to Further Investigate the Efficacy and Safety of BAY86-6150

Resource links provided by NLM:


Further study details as provided by Bayer:

Primary Outcome Measures:
  • Successful treatments of bleeding episodes. [ Time Frame: 10 hours after each bleed ] [ Designated as safety issue: No ]
    A bleed was defined as successfully treated, if no administration of rescue medication was required.

  • Proportion of successful treatments of bleeding episodes on subject level. [ Time Frame: 10 hours after each bleed ] [ Designated as safety issue: No ]
    Proportion of successful treatments of bleeding episodes was calculated as number of bleeding episodes treated successfully - without rescue medication - divided by the total number of bleeding episodes on a dose level.


Secondary Outcome Measures:
  • Time to stop the bleed [ Time Frame: 10 hours after each bleed ] [ Designated as safety issue: No ]
  • Number of injections needed to stop the bleeding episode. [ Time Frame: 10 hours after each bleed ] [ Designated as safety issue: No ]
  • Effectiveness of treatment as rated by the subject's assessment (very effective, effective, partially effective, not effective). [ Time Frame: 10 hours after each bleed ] [ Designated as safety issue: No ]
  • Participant's reported outcome as assessed by Euro QoL (EQ-5D). [ Time Frame: 14 days after last exposure to BAY86-6150 ] [ Designated as safety issue: No ]
  • Participant's reported outcome as assessed by Brief Pain Inventory. [ Time Frame: 7 days after last exposure to BAY86-6150 ] [ Designated as safety issue: No ]
  • Participant's reported outcome as assessed by Work Productivity and Activity Impairment Questionaire. [ Time Frame: 14 days after last exposure to BAY86-6150 ] [ Designated as safety issue: No ]

Enrollment: 5
Study Start Date: June 2012
Study Completion Date: March 2014
Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Arm 1 Drug: BAY86-6150
Four dose levels (6.5 µg/kg, 20 µg/kg, 50 µg/kg and 90 µg/kg) of BAY86-6150 will be studied.
Active Comparator: Arm 2 Drug: eptacog alfa [activated]
comparative PK/PD (pharmacokinetics/pharmacodynamics) evaluation
Experimental: Arm 3 Drug: BAY86-6150
Confirmation of recommended dose of BAY86-6150 to be evaluated further as determined in Part A.

  Eligibility

Ages Eligible for Study:   12 Years to 62 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male subjects
  • 12 to 62 years-of-age
  • History of moderate or severe congenital hemophilia A or B with inhibitors to FVIII or FIX
  • 4 or more bleeding episodes in the last 6 months before enrollment.

Exclusion Criteria:

  • Clinically relevant coagulation disorder other than congenital hemophilia A or B with inhibitors
  • History of coronary and/or peripheral atherosclerotic disease
  • Disseminated intravascular coagulopathy, or stage 2 hypertension
  • Angina pectoris
  • Myocardial infarction
  • Transient ischemic attack
  • Stroke
  • Congestive heart failure
  • Thromboembolic event
  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: NCT01625390

  Show 61 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: NCT01625390     History of Changes
Other Study ID Numbers: 15534, 2011-000323-33, U1111-1133-2156
Study First Received: June 19, 2012
Last Updated: July 6, 2014
Health Authority: Turkey: Ministry of Health Drug and Pharmaceutical Department
Italy: The Italian Medicines Agency
South Africa: Medicines Control Council
United Kingdom: Medicines and Healthcare Products Regulatory
United States: Food and Drug Administration
Japan: Ministry of Health, Labor and Welfare
Croatia: Ministry of Health and Social Care
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Israel: Ministry of Health
Poland: The Office for Reg. of Medicinal Products, Medical Devices and Biocidal Products - Central Register of Clinical Trials
Taiwan: Department of Health
Sweden: Medical Products Agency
Hungary: National Institute of Pharmacy
Serbia: Agency for Drugs and Medicinal Devices
Singapore: Health Sciences Authority
Netherlands: Dutch Health Care Inspectorate
Australia: Department of Health and Ageing Therapeutic Goods Administration
Bulgaria: Ministry of Health
Russia: Pharmacological Committee, Ministry of Health
Romania: State Institute for Drug Control
Denmark: The National Board of Health
Korea: Food and Drug Administration
Hong Kong: Department of Health
Colombia: Instituto Nacional de Vigilancia de Medicamentos y Alimentos
China: Food and Drug Administration
Ukraine: Ministry of Health
Peru: Ministry of Health
Indonesia: National Agency of Drug and Food Control
Brazil: National Health Surveillance Agency
Chile: Instituto de Salud Pública de Chile
India: Central Drugs Standard Control Organization
Mexico: Federal Commission for Protection Against Health Risks
New Zealand: Medsafe
Germany: Paul-Ehrlich-Institut

Keywords provided by Bayer:
hemophilia, haemophilia, inhibitor, FVIIa, Factor VII activated, bypassing

Additional relevant MeSH terms:
Hemophilia B
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on August 01, 2014