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NCT Number: NCT01625390

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

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.

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Key information

Conditions

Hemophilia A, Hemophilia B Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia A Hemophilia B Hemorrhagic Disorders

Age range

12 year–62 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Melbourne, Victoria, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

BAY86-6150

Drug

Four dose levels (6.5 µg/kg, 20 µg/kg, 50 µg/kg and 90 µg/kg) of BAY86-6150 will be studied.

eptacog alfa [activated]

Drug

comparative PK/PD (pharmacokinetics/pharmacodynamics) evaluation

Primary outcomes

  1. Successful treatments of bleeding episodes.

    Time frame: 10 hours after each bleed

    A bleed was defined as successfully treated, if no administration of rescue medication was required.

  2. Proportion of successful treatments of bleeding episodes on subject level.

    Time frame: 10 hours after each bleed

    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 outcomes

  1. Time to stop the bleed

    Time frame: 10 hours after each bleed

  2. Number of injections needed to stop the bleeding episode.

    Time frame: 10 hours after each bleed

  3. Effectiveness of treatment as rated by the subject's assessment (very effective, effective, partially effective, not effective).

    Time frame: 10 hours after each bleed

  4. Participant's reported outcome as assessed by Euro QoL (EQ-5D).

    Time frame: 14 days after last exposure to BAY86-6150

  5. Participant's reported outcome as assessed by Brief Pain Inventory.

    Time frame: 7 days after last exposure to BAY86-6150

  6. Participant's reported outcome as assessed by Work Productivity and Activity Impairment Questionaire.

    Time frame: 14 days after last exposure to BAY86-6150

Sponsors and collaborators

Lead sponsor

Bayer

Industry

Registry information

Official study 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

Important dates

Study start
2012
Primary completion
2014
Study completion
2014
First posted
Jun 21, 2012
Registry last updated
Jul 1, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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