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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01625390




Registration number
NCT01625390
Ethics application status
Date submitted
19/06/2012
Date registered
21/06/2012
Date last updated
1/07/2015

Titles & IDs
Public title
A Phase 2/ 3 Trial to Evaluate the Efficacy and Safety of BAY86-6150
Scientific 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
Secondary ID [1] 0 0
2011-000323-33
Secondary ID [2] 0 0
15534
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A, Hemophilia B 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - BAY86-6150
Treatment: Drugs - eptacog alfa [activated]
Treatment: Drugs - BAY86-6150

Experimental: Arm 1 -

Active comparator: Arm 2 -

Experimental: Arm 3 -


Treatment: Drugs: 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.

Treatment: Drugs: eptacog alfa [activated]
comparative PK/PD (pharmacokinetics/pharmacodynamics) evaluation

Treatment: Drugs: BAY86-6150
Confirmation of recommended dose of BAY86-6150 to be evaluated further as determined in Part A.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Successful treatments of bleeding episodes.
Timepoint [1] 0 0
10 hours after each bleed
Primary outcome [2] 0 0
Proportion of successful treatments of bleeding episodes on subject level.
Timepoint [2] 0 0
10 hours after each bleed
Secondary outcome [1] 0 0
Time to stop the bleed
Timepoint [1] 0 0
10 hours after each bleed
Secondary outcome [2] 0 0
Number of injections needed to stop the bleeding episode.
Timepoint [2] 0 0
10 hours after each bleed
Secondary outcome [3] 0 0
Effectiveness of treatment as rated by the subject's assessment (very effective, effective, partially effective, not effective).
Timepoint [3] 0 0
10 hours after each bleed
Secondary outcome [4] 0 0
Participant's reported outcome as assessed by Euro QoL (EQ-5D).
Timepoint [4] 0 0
14 days after last exposure to BAY86-6150
Secondary outcome [5] 0 0
Participant's reported outcome as assessed by Brief Pain Inventory.
Timepoint [5] 0 0
7 days after last exposure to BAY86-6150
Secondary outcome [6] 0 0
Participant's reported outcome as assessed by Work Productivity and Activity Impairment Questionaire.
Timepoint [6] 0 0
14 days after last exposure to BAY86-6150

Eligibility
Key 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.
Minimum age
12 Years
Maximum age
62 Years
Sex
Males
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Crossover
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/2012
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/03/2014
Sample size
Target
Accrual to date
Final
10
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
- Melbourne
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Tennessee
Country [3] 0 0
Brazil
State/province [3] 0 0
Sao Paulo
Country [4] 0 0
Brazil
State/province [4] 0 0
Rio de Janeiro
Country [5] 0 0
Bulgaria
State/province [5] 0 0
Sofia
Country [6] 0 0
Chile
State/province [6] 0 0
Santiago
Country [7] 0 0
China
State/province [7] 0 0
Guangdong
Country [8] 0 0
China
State/province [8] 0 0
Beijing
Country [9] 0 0
China
State/province [9] 0 0
Tianjin
Country [10] 0 0
Colombia
State/province [10] 0 0
Atlántico
Country [11] 0 0
Colombia
State/province [11] 0 0
Bogotá
Country [12] 0 0
Denmark
State/province [12] 0 0
Aarhus N
Country [13] 0 0
France
State/province [13] 0 0
Lyon Cedex
Country [14] 0 0
France
State/province [14] 0 0
Tours
Country [15] 0 0
Germany
State/province [15] 0 0
Baden-Württemberg
Country [16] 0 0
Germany
State/province [16] 0 0
Rheinland-Pfalz
Country [17] 0 0
Hungary
State/province [17] 0 0
Budapest
Country [18] 0 0
Hungary
State/province [18] 0 0
Debrecen
Country [19] 0 0
India
State/province [19] 0 0
Andhra Pradesh
Country [20] 0 0
India
State/province [20] 0 0
Punjab
Country [21] 0 0
India
State/province [21] 0 0
Bangalore
Country [22] 0 0
India
State/province [22] 0 0
Pune
Country [23] 0 0
Israel
State/province [23] 0 0
Tel Hashomer
Country [24] 0 0
Italy
State/province [24] 0 0
Firenze
Country [25] 0 0
Italy
State/province [25] 0 0
Milano
Country [26] 0 0
Japan
State/province [26] 0 0
Nara
Country [27] 0 0
Japan
State/province [27] 0 0
Tokyo
Country [28] 0 0
Korea, Republic of
State/province [28] 0 0
Seoul
Country [29] 0 0
Mexico
State/province [29] 0 0
Jalisco
Country [30] 0 0
Mexico
State/province [30] 0 0
México D. F.
Country [31] 0 0
Mexico
State/province [31] 0 0
Oaxaca
Country [32] 0 0
Mexico
State/province [32] 0 0
San Luis Potosí
Country [33] 0 0
Netherlands
State/province [33] 0 0
Utrecht
Country [34] 0 0
New Zealand
State/province [34] 0 0
Christchurch
Country [35] 0 0
Poland
State/province [35] 0 0
Warszawa
Country [36] 0 0
Romania
State/province [36] 0 0
Timis
Country [37] 0 0
Romania
State/province [37] 0 0
Bucharest
Country [38] 0 0
Russian Federation
State/province [38] 0 0
Ekaterinburg
Country [39] 0 0
Russian Federation
State/province [39] 0 0
Khabarovsk
Country [40] 0 0
Russian Federation
State/province [40] 0 0
Samara
Country [41] 0 0
Russian Federation
State/province [41] 0 0
St. Petersburg
Country [42] 0 0
Singapore
State/province [42] 0 0
Singapore
Country [43] 0 0
South Africa
State/province [43] 0 0
Freestate
Country [44] 0 0
South Africa
State/province [44] 0 0
Gauteng
Country [45] 0 0
Sweden
State/province [45] 0 0
Göteborg
Country [46] 0 0
Taiwan
State/province [46] 0 0
Changhua
Country [47] 0 0
Taiwan
State/province [47] 0 0
Taipei
Country [48] 0 0
Turkey
State/province [48] 0 0
Istanbul
Country [49] 0 0
Turkey
State/province [49] 0 0
Izmir
Country [50] 0 0
Ukraine
State/province [50] 0 0
Donetsk
Country [51] 0 0
Ukraine
State/province [51] 0 0
Lviv
Country [52] 0 0
Ukraine
State/province [52] 0 0
Odessa
Country [53] 0 0
United Kingdom
State/province [53] 0 0
London
Country [54] 0 0
United Kingdom
State/province [54] 0 0
Truro

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Bayer
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/study/NCT01625390
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Bayer Study Director
Address 0 0
Bayer
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01625390