Clinical trial NCT01625390: A Phase 2/ 3 Trial to Evaluate the Efficacy and Safety of BAY86-6150
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Hemophilia A, Hemophilia B
Keywords: hemophilia, haemophilia, inhibitor, FVIIa, Factor VII activated, bypassing
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2012-06-21
Enrollment: 10
Sex: MALE
Minimum age (years): 12
Maximum age (years): 62
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Successful treatments of bleeding episodes.
timeFrame: 10 hours after each bleed
measure: Proportion of successful treatments of bleeding episodes on subject level.
timeFrame: 10 hours after each bleed
Secondary outcomes:
measure: Time to stop the bleed
timeFrame: 10 hours after each bleed
measure: Number of injections needed to stop the bleeding episode.
timeFrame: 10 hours after each bleed
measure: Effectiveness of treatment as rated by the subject's assessment (very effective, effective, partially effective, not effective).
timeFrame: 10 hours after each bleed
measure: Participant's reported outcome as assessed by Euro QoL (EQ-5D).
timeFrame: 14 days after last exposure to BAY86-6150
measure: Participant's reported outcome as assessed by Brief Pain Inventory.
timeFrame: 7 days after last exposure to BAY86-6150
measure: Participant's reported outcome as assessed by Work Productivity and Activity Impairment Questionaire.
timeFrame: 14 days after last exposure to BAY86-6150
Other outcomes:
Locations:
city: Sacramento
country: United States
state: California
city: Chattanooga
country: United States
state: Tennessee
city: Melbourne
country: Australia
state: Victoria
city: São Paulo
country: Brazil
state: Sao Paulo
city: Rio de Janeiro
country: Brazil
city: Sao Paulo
country: Brazil
city: Sofia
country: Bulgaria
city: Santiago
country: Chile
city: Guangzhou
country: China
state: Guangdong
city: Beijing
country: China
city: Tianjin
country: China
city: Barranquilla
country: Colombia
state: Atlántico
city: Bogotá
country: Colombia
city: Aarhus N
country: Denmark
city: Lyon Cedex
country: France
city: Tours
country: France
city: Villingen-Schwenningen
country: Germany
state: Baden-Württemberg
city: Mainz
country: Germany
state: Rheinland-Pfalz
city: Budapest
country: Hungary
city: Debrecen
country: Hungary
city: Hyderabad
country: India
state: Andhra Pradesh
city: Ludhiana
country: India
state: Punjab
city: Bangalore
country: India
city: Pune
country: India
city: Tel Hashomer
country: Israel
city: Firenze
country: Italy
city: Milano
country: Italy
city: Kashihara
country: Japan
state: Nara
city: Shinjuku-ku
country: Japan
state: Tokyo
city: Suginami
country: Japan
state: Tokyo
city: Seoul
country: Korea, Republic of
city: Guadalajara
country: Mexico
state: Jalisco
city: México D. F.
country: Mexico
city: Oaxaca
country: Mexico
city: San Luis Potosí
country: Mexico
city: Utrecht
country: Netherlands
city: Christchurch
country: New Zealand
city: Warszawa
country: Poland
city: Timisoara
country: Romania
state: Timis
city: Bucharest
country: Romania
city: Bucharest
country: Romania
city: Ekaterinburg
country: Russian Federation
city: Khabarovsk
country: Russian Federation
city: Samara
country: Russian Federation
city: St. Petersburg
country: Russian Federation
city: Singapore
country: Singapore
city: Singapore
country: Singapore
city: Bloemfontein
country: South Africa
state: Freestate
city: Johannesburg
country: South Africa
state: Gauteng
city: Pretoria
country: South Africa
state: Gauteng
city: Göteborg
country: Sweden
city: Changhua
country: Taiwan
city: Taipei
country: Taiwan
city: Taipei
country: Taiwan
city: Istanbul
country: Turkey
city: Izmir
country: Turkey
city: Donetsk
country: Ukraine
city: Lviv
country: Ukraine
city: Odessa
country: Ukraine
city: London
country: United Kingdom
city: Truro
country: United Kingdom
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References: