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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02234323




Registration number
NCT02234323
Ethics application status
Date submitted
29/08/2014
Date registered
9/09/2014
Date last updated
7/10/2019

Titles & IDs
Public title
An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Untreated Males With Severe Hemophilia A
Scientific title
An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia A
Secondary ID [1] 0 0
2013-005512-10
Secondary ID [2] 0 0
997HA306
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 - rFVIIIFc

Experimental: rFVIIIFc - The dose for prophylaxis may be selected and adjusted based on the participant's response to dosing (i.e., available pharmacokinetics (PK) data, including FVIII activity levels, level of physical activity, and bleeding pattern), in the range of 25 to 65 international units per kilogram (IU/kg) at 3 to 5 day intervals. Dose may be increased up to 80 IU/kg if required.


Other interventions: rFVIIIFc
Administered as specified in the treatment arm

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with inhibitor development - Participants will be tested for development of inhibitors at time points throughout the study based on exposure days (ED). One ED is equivalent to a 24 hour period in which rFVIIIFc is dosed
Timepoint [1] 0 0
For the duration of study participation, approximately 3 years
Secondary outcome [1] 0 0
Annualized number of bleeding episodes per participant
Timepoint [1] 0 0
For the duration of study participation, approximately 3 years
Secondary outcome [2] 0 0
Annualized number of spontaneous joint bleeding episodes per participant
Timepoint [2] 0 0
For the duration of study participation, approximately 3 years
Secondary outcome [3] 0 0
Number of injections required to resolve a bleeding episode
Timepoint [3] 0 0
For the duration of study participation, approximately 3 years
Secondary outcome [4] 0 0
Dose per injection of rFVIIIFc required to resolve a bleeding episode
Timepoint [4] 0 0
For the duration of study participation, approximately 3 years
Secondary outcome [5] 0 0
Response to treatment with rFVIIIFc for bleeding episodes, using the 4-point bleeding response scale - Investigators will record assessments of each participant's response to their assigned rFVIIIFc regimen using the following 4-point scale: Excellent, Effective, Partially Effective, Ineffective
Timepoint [5] 0 0
For the duration of study participation, approximately 3 years
Secondary outcome [6] 0 0
Number of EDs per participant per year
Timepoint [6] 0 0
For the duration of study participation, approximately 3 years
Secondary outcome [7] 0 0
Annualized rFVIIIFc consumption per participant for the prevention and treatment of bleeding episodes
Timepoint [7] 0 0
For the duration of study participation, approximately 3 years
Secondary outcome [8] 0 0
rFVIIIFc incremental recovery (IR) as measured by the one-stage aPTT clotting assay and the two-stage chromogenic assay - Samples for incremental recovery taken when the participant is in a non-bleeding state.
Timepoint [8] 0 0
For the duration of study participation, approximately 3 years
Secondary outcome [9] 0 0
Response to immune tolerance induction (ITI) with rFVIIIFc (success, partial success, failure, early withdrawal)
Timepoint [9] 0 0
Until immune tolerance is achieved or up to 33 months

Eligibility
Key inclusion criteria
Key

- Ability of the participant's legally authorized representative (e.g. their parent or
legal guardian) to understand the purpose and risks of the study and provide signed
and dated informed consent and authorization to use confidential health information in
accordance with national and local subject privacy regulations.

- Weight >=3.5 kg at the time of screening.

- Severe hemophilia A defined as <1 IU/dL (<1%) endogenous FVIII documented in the
medical record or as tested during the Screening Period

Key
Minimum age
No limit
Maximum age
5 Years
Gender
Males
Can healthy volunteers participate?
No
Key exclusion criteria
- Any exposure to blood components, factor VIII replacement products, including
commercially available rFVIIIFc at any time prior to or during screening.

- Other coagulation disorder(s) in addition to hemophilia A.

- Any concurrent clinically significant major disease that, in the opinion of the
Investigator, would make the participant unsuitable for enrollment.

- Current systemic treatment with chemotherapy and/or other immunosuppressant drugs.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Research Site - Brisbane
Recruitment hospital [2] 0 0
Research Site - Parkville
Recruitment hospital [3] 0 0
Research Site - Perth
Recruitment hospital [4] 0 0
Research Site - Westmead
Recruitment postcode(s) [1] 0 0
4029 - Brisbane
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment postcode(s) [3] 0 0
6008 - Perth
Recruitment postcode(s) [4] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
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Arkansas
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California
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Indiana
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Kentucky
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Maine
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Michigan
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Minnesota
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Missouri
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New York
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Ohio
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Oregon
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Pennsylvania
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Texas
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Wisconsin
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Brazil
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Campinas
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Brazil
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Canoas
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Ribeirão Preto
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Brazil
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Rio de Janeiro
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Brazil
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São Paulo
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Canada
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Hamilton
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Canada
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London
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Canada
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Toronto
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France
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Caen cedex 9
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France
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Le Kremlin Bicêtre cedex
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France
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Lille Cedex
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France
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Nantes Cedex 1
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France
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Strasbourg
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France
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Toulouse cedex
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France
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Tours cedex 9
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Germany
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Berlin
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Germany
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Bonn
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Germany
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Duesseldorf
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Germany
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Frankfurt
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Germany
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Hannöver
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Ireland
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Dublin
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Italy
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VI
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Italy
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Bari
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Italy
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Florence
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Italy
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Genova
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Italy
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Milan
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Italy
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Napoli
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Italy
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Padova
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Italy
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Rome
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Netherlands
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Leiden
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Netherlands
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Utrecht
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New Zealand
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Auckland
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New Zealand
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Christchurch
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Poland
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Gdansk
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Kraków
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Lublin
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Barcelona
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Madrid
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Sweden
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Stockholm
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United Kingdom
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Cambridgeshire
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United Kingdom
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Hampshire
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London
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Cardiff
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United Kingdom
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Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Bioverativ Therapeutics Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Swedish Orphan Biovitrum
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The primary objective of the study is to evaluate the safety of rFVIIIFc (BIIB031) in
previously untreated participants with severe hemophilia A. The secondary objectives are to
evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in
previously untreated patients (PUPs), to evaluate rFVIIIFc consumption for the prevention and
treatment of bleeding episodes in PUPs, and to describe experience with the use of rFVIIIFc
for immune tolerance induction (ITI) in participants with inhibitors.
Trial website
https://clinicaltrials.gov/show/NCT02234323
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Bioverativ Therapeutics Inc.
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
Other publications