Clinical trial NCT00623727: BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A
Official title: Randomized, Active-controlled, Double-blind, Parallel Design Study to Evaluate the Efficacy and Safety of a Once-a-week Prophylaxis Treatment With BAY79-4980 Compared to Three Times-per-week Prophylaxis With rFVIII-FS in Previously Treated Patients With Severe Hemophilia A
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Hemophilia A
Keywords: Hemophilia A
FVIII disease
rFVIII
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2008-02-26
Enrollment: 143
Sex: MALE
Minimum age (years): 12
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: A study to assess treatment with a new formulation of recombinant factor VIII reconstituted with liposomes (BAY79-4980) to evaluate whether a once-a-week treatment is safe and can prevent bleeds in subjects with severe haemophilia A.


Eligibility criteria: Inclusion Criteria:

* Males aged 12 to 70 years
* Subjects with severe hemophilia A (\< 1% factor VIII \[FVIII\]:C)
* Subjects with equal or greater than 150 exposure days (EDs) with any FVIII in total
* Subjects who have been on-demand treatment with a minimum of 1 relevant bleed per month or have been on secondary prophylaxis treatment with not more than a 3x/week schedule
* Subjects with bleeding events and/or treatments during the last 6 months prior to study entry which are documented in the subjects medical records
* Subjects with no measurable inhibitor activity
* Subjects with no history of FVIII inhibitor antibody formation
* Written informed consent by subject and parent / legal representative, if \< 18 years

Exclusion Criteria:

* Subjects who are receiving primary prophylaxis
* Subjects on prophylaxis with documented requirements of \> 75 IU/kg/week
* Subjects with any other bleeding disease beside hemophilia A (i.e., von Willebrand disease)
* Subjects with abnormal renal function
* Subjects with elevated hepatic transaminases
* Subjects on treatment with immunomodulatory agents within the last 3 months prior to study entry or during the study
* Subjects with known hypersensitivity to the active substance, mouse or hamster protein, liposomes or polyethyleneglycol (PEG)
* Subjects who require any pre-medication for FVIII injections

Primary outcomes:
measure: Percentage of Participants With Less Than 9 Total Bleeds Per Year
timeFrame: up to one year
Secondary outcomes:
measure: Percentage of Participants With Less Than 5 Joint Bleeds Per Year
timeFrame: up to one year
measure: Number of Joint Bleeds Per Participant Per Year in Responders
timeFrame: up to one year
Other outcomes:
measure: Number of Bleeds Per Year
timeFrame: up to one year
measure: Percentage of Bleeds Treated by Various Numbers of Injections
timeFrame: up to one year
measure: Total rFVIII Consumption Per Year
timeFrame: up to one year
measure: Percentage of Participants With Less Than 9 Total Bleeds Per Year in the Open Label Extension Period
timeFrame: 6 months after start of open label extension period
Locations:
city: Orange
country: United States
state: California
city: Sacramento
country: United States
state: California
city: San Francisco
country: United States
state: California
city: Aurora
country: United States
state: Colorado
city: New Orleans
country: United States
state: Louisiana
city: Boston
country: United States
state: Massachusetts
city: Detroit
country: United States
state: Michigan
city: East Lansing
country: United States
state: Michigan
city: Rochester
country: United States
state: Minnesota
city: Kansas City
country: United States
state: Missouri
city: Las Vegas
country: United States
state: Nevada
city: Albuquerque
country: United States
state: New Mexico
city: Buffalo
country: United States
state: New York
city: Rochester
country: United States
state: New York
city: Winston-Salem
country: United States
state: North Carolina
city: Cincinnati
country: United States
state: Ohio
city: Philadelphia
country: United States
state: Pennsylvania
city: Pittsburgh
country: United States
state: Pennsylvania
city: Houston
country: United States
state: Texas
city: Milwaukee
country: United States
state: Wisconsin
city: Buenos Aires
country: Argentina
state: Ciudad Auton. de Buenos Aires
city: Buenos Aires
country: Argentina
state: Ciudad Auton. de Buenos Aires
city: Rosario
country: Argentina
state: Santa Fe
city: Perth
country: Australia
state: Western Australia
city: Graz
country: Austria
city: Wien
country: Austria
city: Bruxelles - Brussel
country: Belgium
city: Bruxelles - Brussel
country: Belgium
city: Leuven
country: Belgium
city: Calgary
country: Canada
state: Alberta
city: Edmonton
country: Canada
state: Alberta
city: Halifax
country: Canada
state: Nova Scotia
city: Hamilton
country: Canada
state: Ontario
city: Ottawa
country: Canada
state: Ontario
city: Toronto
country: Canada
state: Ontario
city: Toronto
country: Canada
state: Ontario
city: Santiago de Chile
country: Chile
city: Santiago
country: Chile
city: Zagreb
country: Croatia
city: Århus N
country: Denmark
city: Tallinn
country: Estonia
city: Tallin
country: Estonia
city: Besancon
country: France
city: Le Kremlin-bicetre
country: France
city: Lyon Cedex
country: France
city: Marseille
country: France
city: Montpellier Cedex
country: France
city: Paris
country: France
city: Paris
country: France
city: Rennes Cedex
country: France
city: Strasbourg
country: France
city: Bonn
country: Germany
state: Nordrhein-Westfalen
city: Münster
country: Germany
state: Nordrhein-Westfalen
city: Berlin
country: Germany
city: Tel Hashomer
country: Israel
city: Catania
country: Italy
city: Firenze
country: Italy
city: Milano
country: Italy
city: Napoli
country: Italy
city: Napoli
country: Italy
city: Torino
country: Italy
city: Klaipeda
country: Lithuania
city: Vilnius
country: Lithuania
city: Groningen
country: Netherlands
city: Nijmegen
country: Netherlands
city: Utrecht
country: Netherlands
city: Auckland
country: New Zealand
city: Christchurch
country: New Zealand
city: Oslo
country: Norway
city: Gdansk
country: Poland
city: Krakow
country: Poland
city: Lodz
country: Poland
city: Poznan
country: Poland
city: Warszawa
country: Poland
city: Timisoara
country: Romania
state: Timis
city: Brasov
country: Romania
city: Bucharest
country: Romania
city: Johannesburg
country: South Africa
state: Gauteng
city: Sevilla
country: Spain
state: Andalucía
city: Oviedo
country: Spain
state: Asturias
city: Sta. Cruz de Tenerife
country: Spain
state: Santa Cruz de Tenerife
city: Barcelona
country: Spain
city: Madrid
country: Spain
city: Valencia
country: Spain
city: Bern
country: Switzerland
city: Changhua
country: Taiwan
city: Taipei
country: Taiwan
city: Taipei
country: Taiwan
city: Adana
country: Turkey
city: Antalya
country: Turkey
city: Izmir
country: Turkey
city: Cambridge
country: United Kingdom
state: Cambridgeshire
city: Cardiff
country: United Kingdom
state: South Glamorgan
city: Sheffield
country: United Kingdom
state: South Yorkshire
city: Birmingham
country: United Kingdom
city: London
country: United Kingdom
city: Manchester
country: United Kingdom
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References:
citation: Di Minno G, Cerbone AM, Coppola A, Cimino E, Di Capua M, Pamparana F, Tufano A, Di Minno MN. Longer-acting factor VIII to overcome limitations in haemophilia management: the PEGylated liposomes formulation issue. Haemophilia. 2010 Jan;16 Suppl 1:2-6. doi: 10.1111/j.1365-2516.2009.02155.x.
citation: Powell J, Martinowitz U, Windyga J, Di Minno G, Hellmann A, Pabinger I, Maas Enriquez M, Schwartz L, Ingerslev J; LipLong Study Investigators. Efficacy and safety of prophylaxis with once-weekly BAY 79-4980 compared with thrice-weekly rFVIII-FS in haemophilia A patients. A randomised, active-controlled, double-blind study. Thromb Haemost. 2012 Nov;108(5):913-22. doi: 10.1160/TH12-03-0188. Epub 2012 Sep 26.