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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/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
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
Secondary ID [1] 0 0
2007-003718-32
Secondary ID [2] 0 0
12781
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - rFVIII-FS/pegylated liposomes (BAY79-4980)
Other interventions - 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)


Other interventions: 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

Other interventions: rFVIII-FS/WFI (BAY14-2222)
25 IU/kg body weight intravenous 3x/week for 52 weeks
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants With Less Than 9 Total Bleeds Per Year
Timepoint [1] 0 0
up to one year
Secondary outcome [1] 0 0
Percentage of Participants With Less Than 5 Joint Bleeds Per Year
Timepoint [1] 0 0
up to one year
Secondary outcome [2] 0 0
Number of Joint Bleeds Per Participant Per Year in Responders
Timepoint [2] 0 0
up to one year

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
Minimum age
12 Years
Maximum age
70 Years
Sex
Males
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/2008
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/10/2010
Sample size
Target
Accrual to date
Final
143
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 0 0
- Perth
Recruitment postcode(s) [1] 0 0
6000 - Perth
Recruitment outside Australia
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United States of America
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California
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Colorado
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Michigan
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Wisconsin
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Argentina
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Ciudad Auton. de Buenos Aires
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Santa Fe
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Graz
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Leuven
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Catania
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Adana
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Antalya
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Izmir
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United Kingdom
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South Yorkshire
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Birmingham
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London
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Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Bayer
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00623727
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Bayer Study Director
Address 0 0
Bayer
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT00623727