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.
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
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
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Las Vegas, Nevada, 89109-2803
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Milwaukee, Wisconsin, 53226
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New Orleans, Louisiana, 70112-2699
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Orange, California, 92868
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Philadelphia, Pennsylvania, 19104-0506
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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
France9 sites
Study site
Besançon, 25030
Study site
Le Kremlin-Bicêtre, 94275
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Lyon, 69437
Study site
Marseille, 13005
Study site
Montpellier, 34059
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Paris, 75015
Study site
Paris, 75674
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Rennes, 35033
Study site
Strasbourg, 67091
Canada7 sites
Study site
Calgary, Alberta, T2N 2T9
Study site
Edmonton, Alberta, T6G 2C8
Study site
Halifax, Nova Scotia, B3H 2Y9
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Hamilton, Ontario, L8S 4J9
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Ottawa, Ontario, K1H 8L6
Study site
Toronto, Ontario, M5B 1W8
Study site
Toronto, Ontario, M5G 1X8
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
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
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
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Manchester, Manchester, M13 9WL
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Sheffield, South Yorkshire, S10 2JF
Poland5 sites
Study site
Gdansk, 80-952
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Krakow, 31-501
Study site
Lodz, 93-510
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Poznan, 61-833
Study site
Warsaw, 02-776
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
Belgium3 sites
Study site
Bruxelles - Brussel, 1020
Study site
Bruxelles - Brussel, 1200
Study site
Leuven, 3000
Germany3 sites
Study site
Berlin, State of Berlin, 10249
Study site
Bonn, North Rhine-Westphalia, 53105
Study site
Münster, North Rhine-Westphalia, 48143
Netherlands3 sites
Study site
Groningen, 9713 GZ
Study site
Nijmegen, 6525 GA
Study site
Utrecht, 3508 GA
Romania3 sites
Study site
Brasov, 50035
Study site
Bucharest, 022328
Study site
Timișoara, Timiș County, 300011
Taiwan3 sites
Study site
Changhua, 500
Study site
Taipei, 10002
Study site
Taipei, 11217
Turkey (Türkiye)3 sites
Study site
Adana, 01-330
Study site
Antalya, 07059
Study site
Izmir, 35-100
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 group | Role | What they receive |
|---|---|---|
| rFVIII-FS/pegylated liposomes (BAY79-4980) | Experimental | 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)) Receives: Biological: rFVIII-FS/pegylated liposomes (BAY79-4980) |
| rFVIII-FS/WFI (BAY14-2222) | Active comparator | 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) Receives: Biological: rFVIII-FS/WFI (BAY14-2222) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
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
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.
- First posted26 Feb 2008 (estimated)
- Study startJun 2008
- Primary completionApr 2010
- Study completionOct 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by BayerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Bayer Study Director, Study director, Bayer
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
- Results posted
- Yes, on ClinicalTrials.gov
- 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 20059562Powell 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