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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02234310




Registration number
NCT02234310
Ethics application status
Date submitted
17/07/2014
Date registered
9/09/2014

Titles & IDs
Public title
Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
Scientific title
An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc; BIIB029) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia B
Secondary ID [1] 0 0
2013-003629-27
Secondary ID [2] 0 0
998HB303
Universal Trial Number (UTN)
Trial acronym
PUPs B-LONG
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia B 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 - rFIXFc

Experimental: Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) - Participants received rFIXFc intravenous (IV) injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 International Units per kilogram (IU/kg) weekly until a participant reached at least 50 exposure days (ED=24-hour period in which greater than or equal to (\>=1) injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX (FIX) levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.


Treatment: Other: rFIXFc
Adjustments to the dose and interval of rFIXFc was made in this study based on investigator discretion using available pharmacokinetic (PK) data, subsequent FIX trough and peak levels, level of physical activity, and bleeding pattern, in accordance with local standards of care for a prophylactic regimen. There was an option to start study dosing as episodic treatment (on-demand).
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 rFIXFc 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 rFIXFc 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 rFIXFc Required to Resolve a Bleeding Episode
Timepoint [6] 0 0
Up to 3 years
Secondary outcome [7] 0 0
Average Dose Per Injection of rFIXFc Required to Resolve a Bleeding Episode
Timepoint [7] 0 0
Up to 3 years
Secondary outcome [8] 0 0
Change From Baseline in rFIXFc Incremental Recovery (IR)
Timepoint [8] 0 0
Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144

Eligibility
Key inclusion criteria
Key

* Weight >=3.5 kilogram at the time of informed consent.
* Severe hemophilia B was defined as less than or equal to (<=)2 International Units per deciliter (IU/dL) (<=2 percent [%]) endogenous FIX documented in the medical record or as tested during the Screening Period.

Key
Minimum age
No limit
Maximum age
17 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* History of positive inhibitor testing. A prior history of inhibitors was defined based on a participant's historical positive inhibitor test using the local laboratory Bethesda value for a positive inhibitor test (that is equal to or above lower limit of detection).
* History of hypersensitivity reactions associated with any rFIXFc administration.
* Exposure to blood components or injection with a coagulation factor IX (FIX) concentrate (including plasma derived) other than rFIXFc.
* Injection with commercially available rFIXFc more than 28 days prior to Screening.
* More than 3 injections of commercially available rFIXFc prior to confirmation of eligibility.
* Other coagulation disorders in addition to hemophilia B.
* Any concurrent clinically significant major disease that, in the opinion of the Investigator, would have made the participant unsuitable for enrollment (example HIV infection with cluster of differentiation 4 (CD4) lymphocyte count less than (<)200 cells/microliter (mcL) or a viral load greater than (>)200 particles/mcL, or any other known congenital or acquired immunodeficiency).
* Current systemic treatment with chemotherapy and/or other immunosuppressant drugs. Use of steroids for treatment of asthma or management of acute allergic episodes or otherwise life-threatening episodes was allowed. Treatment in these circumstances should not have exceeded a 14-day duration.
* Participation within the past 30 days in any other clinical study involving investigational treatment.
* Current enrollment in any other clinical study involving investigational treatment.
* Inability to comply with study requirements.
* Other unspecified reasons that, in the opinion of the Investigator or Bioverativ, would have made the participant unsuitable for enrollment.

NOTE: Other protocol-defined inclusion/exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
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
13/11/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
20/08/2019
Sample size
Target
Accrual to date
Final
33
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Research Site - Westmead
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Kentucky
Country [6] 0 0
United States of America
State/province [6] 0 0
Louisiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Oregon
Country [10] 0 0
United States of America
State/province [10] 0 0
Pennsylvania
Country [11] 0 0
Denmark
State/province [11] 0 0
Aarhus
Country [12] 0 0
France
State/province [12] 0 0
Nord
Country [13] 0 0
France
State/province [13] 0 0
Rhone
Country [14] 0 0
Ireland
State/province [14] 0 0
Dublin
Country [15] 0 0
Italy
State/province [15] 0 0
Milano
Country [16] 0 0
Italy
State/province [16] 0 0
Napoli
Country [17] 0 0
Italy
State/province [17] 0 0
Parma
Country [18] 0 0
Italy
State/province [18] 0 0
Roma
Country [19] 0 0
Netherlands
State/province [19] 0 0
Utrecht
Country [20] 0 0
New Zealand
State/province [20] 0 0
Auckland
Country [21] 0 0
Poland
State/province [21] 0 0
Warszawa
Country [22] 0 0
Sweden
State/province [22] 0 0
Malmo
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Cambridgeshire
Country [24] 0 0
United Kingdom
State/province [24] 0 0
London

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/NCT02234310