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Hemophilia APhase 2NCT00623727

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

A Phase 2 study testing rFVIII-FS/pegylated liposomes (BAY79-4980) and rFVIII-FS/WFI (BAY14-2222) in people with Hemophilia A, run by Bayer at 95 sites in 26 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 12 to 70 years, men only
Conditions the study is for
Hemophilia A
What is being tested?
rFVIII-FS/pegylated liposomes (BAY79-4980) and rFVIII-FS/WFI (BAY14-2222)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
143 participants
Where
95 sitesin 26 countries
Study dates
Jun 2008to Oct 2010
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 70 years
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

95 sites in 26 countries.

95 sites in 26 countries

United States20 sites

  • Study site

    Albuquerque, New Mexico, 87131-0001

  • Study site

    Aurora, Colorado, 80045

  • Study site

    Boston, Massachusetts, 02115

  • Study site

    Buffalo, New York, 14215

  • Study site

    Cincinnati, Ohio, 45229

  • Study site

    Detroit, Michigan, 48202

  • Study site

    East Lansing, Michigan, 48823

  • Study site

    Houston, Texas, 77030

  • Study site

    Kansas City, Missouri, 64108-9898

  • Study site

    Las Vegas, Nevada, 89109-2803

  • Study site

    Milwaukee, Wisconsin, 53226

  • Study site

    New Orleans, Louisiana, 70112-2699

  • Study site

    Orange, California, 92868

  • Study site

    Philadelphia, Pennsylvania, 19104-0506

  • Study site

    Pittsburgh, Pennsylvania, 15213

  • Study site

    Rochester, Minnesota, 55905

  • Study site

    Rochester, New York, 14621

  • Study site

    Sacramento, California, 95817

  • Study site

    San Francisco, California, 94143-0324

  • Study site

    Winston-Salem, North Carolina, 27157-1009

Other trials in United States

France9 sites

  • Study site

    Besançon, 25030

  • Study site

    Le Kremlin-Bicêtre, 94275

  • Study site

    Lyon, 69437

  • Study site

    Marseille, 13005

  • Study site

    Montpellier, 34059

  • Study site

    Paris, 75015

  • Study site

    Paris, 75674

  • Study site

    Rennes, 35033

  • Study site

    Strasbourg, 67091

Other trials in France

Canada7 sites

  • Study site

    Calgary, Alberta, T2N 2T9

  • Study site

    Edmonton, Alberta, T6G 2C8

  • Study site

    Halifax, Nova Scotia, B3H 2Y9

  • Study site

    Hamilton, Ontario, L8S 4J9

  • Study site

    Ottawa, Ontario, K1H 8L6

  • Study site

    Toronto, Ontario, M5B 1W8

  • Study site

    Toronto, Ontario, M5G 1X8

Other trials in Canada

Italy6 sites

  • Study site

    Catania, 95124

  • Study site

    Florence, 50134

  • Study site

    Milan, 20122

  • Study site

    Naples, 80131

  • Study site

    Naples, 80144

  • Study site

    Torino, 10126

Other trials in Italy

Spain6 sites

  • Study site

    Barcelona, Barcelona, 08035

  • Study site

    Madrid, Madrid, 28046

  • Study site

    Oviedo, Principality of Asturias, 33006

  • Study site

    Santa Cruz de Tenerife, Santa Cruz de Tenerife, 38009

  • Study site

    Seville, Andalusia, 41013

  • Study site

    Valencia, Valencia, 46026

Other trials in Spain

United Kingdom6 sites

  • Study site

    Birmingham, B15 2TH

  • Study site

    Cambridge, Cambridgeshire, CB2 0QQ

  • Study site

    Cardiff, South Glamorgan, CF14 4XW

  • Study site

    London, London, SE1 7EH

  • Study site

    Manchester, Manchester, M13 9WL

  • Study site

    Sheffield, South Yorkshire, S10 2JF

Other trials in United Kingdom

Poland5 sites

  • Study site

    Gdansk, 80-952

  • Study site

    Krakow, 31-501

  • Study site

    Lodz, 93-510

  • Study site

    Poznan, 61-833

  • Study site

    Warsaw, 02-776

Other trials in Poland

Argentina3 sites

  • Study site

    Buenos Aires, Ciudad Auton. de Buenos Aires, C1221ADC

  • Study site

    Buenos Aires, Ciudad Auton. de Buenos Aires, C1425AUM

  • Study site

    Rosario, Santa Fe Province, S2000CKF

Other trials in Argentina

Belgium3 sites

  • Study site

    Bruxelles - Brussel, 1020

  • Study site

    Bruxelles - Brussel, 1200

  • Study site

    Leuven, 3000

Other trials in Belgium

Germany3 sites

  • Study site

    Berlin, State of Berlin, 10249

  • Study site

    Bonn, North Rhine-Westphalia, 53105

  • Study site

    Münster, North Rhine-Westphalia, 48143

Other trials in Germany

Netherlands3 sites

  • Study site

    Groningen, 9713 GZ

  • Study site

    Nijmegen, 6525 GA

  • Study site

    Utrecht, 3508 GA

Other trials in Netherlands

Romania3 sites

  • Study site

    Brasov, 50035

  • Study site

    Bucharest, 022328

  • Study site

    Timișoara, Timiș County, 300011

Other trials in Romania

Taiwan3 sites

  • Study site

    Changhua, 500

  • Study site

    Taipei, 10002

  • Study site

    Taipei, 11217

Other trials in Taiwan

Turkey (Türkiye)3 sites

  • Study site

    Adana, 01-330

  • Study site

    Antalya, 07059

  • Study site

    Izmir, 35-100

Other trials in Turkey (Türkiye)

Austria2 sites

  • Study site

    Graz, 8036

  • Study site

    Vienna, State of Vienna, 1090

Other trials in Austria

Lithuania2 sites

  • Study site

    Klaipėda, 50809

  • Study site

    Vilnius, 08661

Other trials in Lithuania

New Zealand2 sites

  • Study site

    Auckland, 1023

  • Study site

    Christchurch, 8001

Other trials in New Zealand

Australia1 site

  • Study site

    Perth, Western Australia, 6000

Other trials in Australia

Chile1 site

  • Study site

    Santiago

Other trials in Chile

Croatia1 site

  • Study site

    Zagreb, 10000

Other trials in Croatia

Denmark1 site

  • Study site

    Århus N, 8200

Other trials in Denmark

Estonia1 site

  • Study site

    Tallinn, 13419

Other trials in Estonia

Israel1 site

  • Study site

    Tel Litwinsky, 52621

Other trials in Israel

Norway1 site

  • Study site

    Oslo, 0027

Other trials in Norway

South Africa1 site

  • Study site

    Johannesburg, Gauteng, 2132

Other trials in South Africa

Switzerland1 site

  • Study site

    Bern, 3010

Other trials in Switzerland

About this study

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

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.

Conditions
Hemophilia A
Being tested
rFVIII-FS/pegylated liposomes (BAY79-4980), rFVIII-FS/WFI (BAY14-2222)
Keywords
Hemophilia A, FVIII disease, rFVIII
Medical subject headings
Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • rFVIII-FS/pegylated liposomes (BAY79-4980)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)Biological

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

Study groups in NCT00623727
Study groupRoleWhat they receive
rFVIII-FS/pegylated liposomes (BAY79-4980)Experimental35 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))
Receives: Biological: rFVIII-FS/pegylated liposomes (BAY79-4980)
rFVIII-FS/WFI (BAY14-2222)Active comparator25 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)
Receives: Biological: rFVIII-FS/WFI (BAY14-2222)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Participants With Less Than 9 Total Bleeds Per Year

    Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event.

    Measured: up to one year

Secondary outcomes (2)

  1. Percentage of Participants With Less Than 5 Joint Bleeds Per Year

    Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event.

    Measured: up to one year

  2. Number of Joint Bleeds Per Participant Per Year in Responders

    Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. Responders were the subjects with less than 9 total bleeds per year

    Measured: up to one year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Feb 2008 (estimated)
  2. Study startJun 2008
  3. Primary completionApr 2010
  4. Study completionOct 2010

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
143 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00623727
Sponsor's protocol ID
12781
Other IDs
2007-003718-32
Status last verified
Jul 2013
First submitted
9 Jan 2008
Last updated
15 Jul 2013
Data monitoring committee
Yes

Publications

  • 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.

    PubMed 20059562

  • 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.

    PubMed 23014711

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing rFVIII-FS/pegylated liposomes (BAY79-4980) and rFVIII-FS/WFI (BAY14-2222) in people with Hemophilia A, run by Bayer at 95 sites in 26 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 15 Jul 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 95 sites in United States, France, Canada, Italy, Spain and 21 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 Aug 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants With Less Than 9 Total Bleeds Per Year (time frame: up to one year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00623727, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jul 2013.

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