A Phase 2/ 3 Trial to Evaluate the Efficacy and Safety of BAY86-6150
This study is ongoing, but not recruiting participants.
Sponsor:
Bayer
Information provided by:
Bayer
ClinicalTrials.gov Identifier:
NCT01625390
First received: June 19, 2012
Last updated: May 8, 2013
Last verified: May 2013
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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:
Genetics Home Reference related topics:
hemophilia
MedlinePlus related topics:
Hemophilia
U.S. FDA Resources
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: | 10 |
| Study Start Date: | June 2012 |
| Estimated Study Completion Date: | June 2013 |
| Estimated Primary Completion Date: | June 2013 (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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01625390
Show 61 Study Locations
Show 61 Study LocationsSponsors and Collaborators
Bayer
Investigators
| Study Director: | Bayer Study Director | Bayer |
More Information
Additional Information:
No publications provided
| Responsible Party: | Therapeutic Area Head, Bayer Healthcare AG |
| 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: | May 8, 2013 |
| 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 Publica 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 May 19, 2013