BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A

NCT ID: NCT00623727

Last Updated: 2013-07-15

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

143 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-06-30

Study Completion Date

2010-10-31

Brief Summary

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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.

Detailed Description

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Conditions

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Hemophilia A

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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rFVIII-FS/pegylated liposomes (BAY79-4980)

35 IU/kg body weight of BAY79-4980 1x/week plus 2 dummy injections/week (dummy = rFVIII (recombinant factor VIII)-FS (formulated with sucrose) excipient reconstituted in WFI (sterile water for injection))

Group Type EXPERIMENTAL

rFVIII-FS/pegylated liposomes (BAY79-4980)

Intervention Type BIOLOGICAL

35 IU/kg body weight intravenous 1x/week for 52 weeks This arm will be stopped by 30.04.10 the subjects will be offered to change to the active comparator arm

rFVIII-FS/WFI (BAY14-2222)

25 IU/kg body weight of rFVIII-FS 3x/week (employing 1 percent POPC (1-palmitoyl-2-oleoyl-sn-glycero-3-phosphocholine)-alone liposome (rFVIII-FS-POPC) as blinding agent used for first weekly injection and rFVIII-FS in WFI for 2nd and 3rd injection)

Group Type ACTIVE_COMPARATOR

rFVIII-FS/WFI (BAY14-2222)

Intervention Type BIOLOGICAL

25 IU/kg body weight intravenous 3x/week for 52 weeks

Interventions

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rFVIII-FS/pegylated liposomes (BAY79-4980)

35 IU/kg body weight intravenous 1x/week for 52 weeks This arm will be stopped by 30.04.10 the subjects will be offered to change to the active comparator arm

Intervention Type BIOLOGICAL

rFVIII-FS/WFI (BAY14-2222)

25 IU/kg body weight intravenous 3x/week for 52 weeks

Intervention Type BIOLOGICAL

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Maximum Eligible Age

70 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Bayer

INDUSTRY

Sponsor Role lead

Responsible Party

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Bayer HealthCare Pharmaceuticals Inc.

Principal Investigators

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Bayer Study Director

Role: STUDY_DIRECTOR

Bayer

Locations

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Birmingham, , United Kingdom

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Orange, California, United States

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Sacramento, California, United States

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San Francisco, California, United States

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Aurora, Colorado, United States

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New Orleans, Louisiana, United States

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Boston, Massachusetts, United States

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Detroit, Michigan, United States

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East Lansing, Michigan, United States

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Rochester, Minnesota, United States

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Kansas City, Missouri, United States

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Las Vegas, Nevada, United States

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Albuquerque, New Mexico, United States

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Buffalo, New York, United States

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Rochester, New York, United States

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Winston-Salem, North Carolina, United States

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Cincinnati, Ohio, United States

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Philadelphia, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Houston, Texas, United States

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Milwaukee, Wisconsin, United States

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Buenos Aires, Ciudad Auton. de Buenos Aires, Argentina

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Buenos Aires, Ciudad Auton. de Buenos Aires, Argentina

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Rosario, Santa Fe Province, Argentina

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Perth, Western Australia, Australia

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Vienna, Vienna, Austria

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Graz, , Austria

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Bruxelles - Brussel, , Belgium

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Bruxelles - Brussel, , Belgium

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Leuven, , Belgium

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Calgary, Alberta, Canada

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Edmonton, Alberta, Canada

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Halifax, Nova Scotia, Canada

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Hamilton, Ontario, Canada

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Ottawa, Ontario, Canada

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Toronto, Ontario, Canada

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Toronto, Ontario, Canada

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Santiago, , Chile

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Zagreb, , Croatia

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Århus N, , Denmark

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Tallinn, , Estonia

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Besançon, , France

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Le Kremlin-Bicêtre, , France

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Lyon, , France

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Marseille, , France

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Montpellier, , France

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Paris, , France

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Paris, , France

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Rennes, , France

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Strasbourg, , France

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Bonn, North Rhine-Westphalia, Germany

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Münster, North Rhine-Westphalia, Germany

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Berlin, State of Berlin, Germany

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Tel Litwinsky, , Israel

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Catania, , Italy

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Florence, , Italy

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Milan, , Italy

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Napoli, , Italy

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Napoli, , Italy

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Torino, , Italy

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Klaipėda, , Lithuania

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Vilnius, , Lithuania

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Groningen, , Netherlands

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Nijmegen, , Netherlands

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Utrecht, , Netherlands

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Auckland, , New Zealand

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Christchurch, , New Zealand

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Oslo, , Norway

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Gdansk, , Poland

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Krakow, , Poland

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Lodz, , Poland

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Poznan, , Poland

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Warsaw, , Poland

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Timișoara, Timiș County, Romania

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Brasov, , Romania

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Bucharest, , Romania

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Johannesburg, Gauteng, South Africa

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Seville, Andalusia, Spain

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Barcelona, Barcelona, Spain

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Madrid, Madrid, Spain

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Oviedo, Principality of Asturias, Spain

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Santa Cruz de Tenerife, Santa Cruz de Tenerife, Spain

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Valencia, Valencia, Spain

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Bern, , Switzerland

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Changhua, , Taiwan

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Taipei, , Taiwan

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Taipei, , Taiwan

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Adana, , Turkey (Türkiye)

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Antalya, , Turkey (Türkiye)

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Izmir, , Turkey (Türkiye)

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Cambridge, Cambridgeshire, United Kingdom

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London, London, United Kingdom

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Manchester, Manchester, United Kingdom

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Cardiff, South Glamorgan, United Kingdom

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Sheffield, South Yorkshire, United Kingdom

Site Status

Countries

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United States Argentina Australia Austria Belgium Canada Chile Croatia Denmark Estonia France Germany Israel Italy Lithuania Netherlands New Zealand Norway Poland Romania South Africa Spain Switzerland Taiwan Turkey (Türkiye) United Kingdom

References

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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.

Reference Type RESULT
PMID: 20059562 (View on PubMed)

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.

Reference Type RESULT
PMID: 23014711 (View on PubMed)

Other Identifiers

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2007-003718-32

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

12781

Identifier Type: -

Identifier Source: org_study_id

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