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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT00623727
Registration number
NCT00623727
Ethics application status
Date submitted
9/01/2008
Date registered
26/02/2008
Date last updated
15/07/2013
Titles & IDs
Public title
BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A
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Scientific 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
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Secondary ID [1]
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2007-003718-32
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Secondary ID [2]
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12781
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hemophilia A
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Condition category
Condition code
Blood
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Clotting disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Other - rFVIII-FS/pegylated liposomes (BAY79-4980)
Treatment: Other - rFVIII-FS/WFI (BAY14-2222)
Experimental: 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))
Active comparator: 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)
Treatment: Other: 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
Treatment: Other: rFVIII-FS/WFI (BAY14-2222)
25 IU/kg body weight intravenous 3x/week for 52 weeks
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Intervention code [1]
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Treatment: Other
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percentage of Participants With Less Than 9 Total Bleeds Per Year
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Assessment method [1]
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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.
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Timepoint [1]
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up to one year
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Secondary outcome [1]
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Percentage of Participants With Less Than 5 Joint Bleeds Per Year
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Assessment method [1]
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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.
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Timepoint [1]
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up to one year
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Secondary outcome [2]
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Number of Joint Bleeds Per Participant Per Year in Responders
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Assessment method [2]
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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
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Timepoint [2]
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up to one year
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Eligibility
Key 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
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Minimum age
12
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Maximum age
70
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Sex
Males
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/06/2008
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
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Actual
1/10/2010
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Sample size
Target
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Accrual to date
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Final
143
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Recruitment in Australia
Recruitment state(s)
WA
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Recruitment hospital [1]
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- Perth
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Recruitment postcode(s) [1]
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6000 - Perth
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Manchester
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Bayer
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/study/NCT00623727
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Trial related presentations / 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. 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.
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Public notes
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Contacts
Principal investigator
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Bayer Study Director
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Bayer
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
Type
Citations or Other Details
Journal
Di Minno G, Cerbone AM, Coppola A, Cimino E, Di Ca...
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Journal
Powell J, Martinowitz U, Windyga J, Di Minno G, He...
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Results are available at
https://clinicaltrials.gov/study/NCT00623727
Download to PDF