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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02234323
Ethics application status
Date submitted
29/08/2014
Date registered
9/09/2014

Titles & IDs
Public title
An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously 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
Treatment: Other - rFVIIIFc

Experimental: rFVIIIFc - Participants were to receive rFVIIIFc as follows- Prophylaxis regimen (PR): rFVIIIFc 25-80 international units per kilogram (IU/kg), at 3- to 5-day intervals until participant reached greater than or equal to (\>=) 50 exposure days (ED: 24-hour period in which \>=1 injection/dose of rFVIIIFc was given), or study withdrawal/end of study. Adjustments to dose/dosing interval was done as needed by investigator; Treatment with an optional ER (Episodic regimen) can be initiated before PR at investigators discretion; ITI: rFVIIIFc 200 IU/kg, daily for participants who, after exposure to rFVIIIFc, had positive high titer inhibitor (\>=5.00 Bethesda Units per milliliter \[BU/mL\]) or positive low titer inhibitor (\>=0.60 and \<5.00 BU/mL) and had poorly controlled bleeding despite increased rFVIIIFc doses, or required bypassing agent to treat bleeding.


Treatment: Other: rFVIIIFc
Administered as specified in arm description
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
Timepoint [1] 0 0
Up to 3 years
Secondary outcome [1] 0 0
Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR])
Timepoint [1] 0 0
Up to 3 years
Secondary outcome [2] 0 0
Annualized Number of Spontaneous Joint Bleeding Episodes
Timepoint [2] 0 0
Up to 3 years
Secondary outcome [3] 0 0
Number of rFVIIIFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale
Timepoint [3] 0 0
Up to 3 years
Secondary outcome [4] 0 0
Total Number of Exposure Days (EDs)
Timepoint [4] 0 0
Up to 3 years
Secondary outcome [5] 0 0
Total Annualized rFVIIIFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes
Timepoint [5] 0 0
Up to 3 years
Secondary outcome [6] 0 0
Number of Injections of rFVIIIFc Required to Resolve a Bleeding Episode
Timepoint [6] 0 0
Up to 3 years
Secondary outcome [7] 0 0
Average Dose Per Injection of rFVIIIFc Required to Resolve a Bleeding Episode
Timepoint [7] 0 0
Up to 3 years
Secondary outcome [8] 0 0
Change From Baseline in rFVIIIFc Incremental Recovery (IR)
Timepoint [8] 0 0
Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108 and 120
Secondary outcome [9] 0 0
Number of Participants With Response to Immune Tolerance Induction (ITI)
Timepoint [9] 0 0
Up to 3 years

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 participant privacy regulations.
* Weight >=3.5 kg at the time of screening.
* Severe hemophilia A defined as less than (<) 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
Sex
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.
* History of positive inhibitor testing. A prior history of inhibitors was defined based on a patient's historical positive inhibitor test using the local laboratory Bethesda value for a positive inhibitor test (ie, equal to or above lower level of detection).
* History of hypersensitivity reactions associated with any rFVIIIFc administration.
* 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
Not applicable
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
12/01/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
23/09/2019
Sample size
Target
Accrual to date
Final
108
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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United States of America
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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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Brazil
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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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Poland
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Kraków
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Poland
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Lublin
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Warszawa
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Spain
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Barcelona
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Spain
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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, a Sanofi company
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
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT02234323