Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B (PUPs B-LONG)
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02234310 |
Recruitment Status :
Completed
First Posted : September 9, 2014
Results First Posted : July 31, 2020
Last Update Posted : July 31, 2020
|
Sponsor:
Bioverativ, a Sanofi company
Collaborator:
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Sanofi ( Bioverativ, a Sanofi company )
- Study Details
- Tabular View
- Study Results
- Disclaimer
- How to Read a Study Record
Tracking Information | |||||
---|---|---|---|---|---|
First Submitted Date ICMJE | July 17, 2014 | ||||
First Posted Date ICMJE | September 9, 2014 | ||||
Results First Submitted Date ICMJE | July 15, 2020 | ||||
Results First Posted Date ICMJE | July 31, 2020 | ||||
Last Update Posted Date | July 31, 2020 | ||||
Actual Study Start Date ICMJE | November 13, 2014 | ||||
Actual Primary Completion Date | August 20, 2019 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay [ Time Frame: Up to 3 years ] Development of an inhibitor was defined as an inhibitor test result of >= 0.60 Bethesda units per milliliter (BU/mL) that was confirmed by a second test result of >=0.60 BU/mL from a separate sample, drawn 2 to 4 weeks after the date when the original sample was drawn, with both tests performed by the central laboratory using Nijmegen-modified Bethesda assay.
|
||||
Original Primary Outcome Measures ICMJE |
Number of Participants with Inhibitor Development [ Time Frame: For the duration of study participation, approximately 3 years ] Participants will be tested for development of inhibitors at timepoints throughout the study based on exposure days (ED). One ED is equivalent to a 24 hour period in which rFIXFc is dosed.
|
||||
Change History | |||||
Current Secondary Outcome Measures ICMJE |
|
||||
Original Secondary Outcome Measures ICMJE |
|
||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B | ||||
Official Title ICMJE | 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 | ||||
Brief Summary | The primary objective of the study was to evaluate the safety of recombinant coagulation factor IX Fc fusion protein (rFIXFc, BIIB029) in previously untreated patients (PUPs) with severe hemophilia B. Secondary objectives were to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes in PUPs, and to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes in PUPs. | ||||
Detailed Description | Not Provided | ||||
Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 3 | ||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
||||
Condition ICMJE | Hemophilia B | ||||
Intervention ICMJE | Biological: 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).
Other Names:
|
||||
Study Arms ICMJE | 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.
Intervention: Biological: rFIXFc
|
||||
Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
|||||
Recruitment Information | |||||
Recruitment Status ICMJE | Completed | ||||
Actual Enrollment ICMJE |
33 | ||||
Original Estimated Enrollment ICMJE |
60 | ||||
Actual Study Completion Date ICMJE | August 20, 2019 | ||||
Actual Primary Completion Date | August 20, 2019 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol-defined inclusion/exclusion criteria may apply. |
||||
Sex/Gender ICMJE |
|
||||
Ages ICMJE | up to 17 Years (Child) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | Australia, Denmark, France, Ireland, Italy, Netherlands, New Zealand, Poland, Sweden, United Kingdom, United States | ||||
Removed Location Countries | Belgium, Canada, Germany, Spain, Switzerland | ||||
Administrative Information | |||||
NCT Number ICMJE | NCT02234310 | ||||
Other Study ID Numbers ICMJE | 998HB303 2013-003629-27 ( EudraCT Number ) |
||||
Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product | Not Provided | ||||
IPD Sharing Statement ICMJE |
|
||||
Responsible Party | Sanofi ( Bioverativ, a Sanofi company ) | ||||
Study Sponsor ICMJE | Bioverativ, a Sanofi company | ||||
Collaborators ICMJE | Swedish Orphan Biovitrum | ||||
Investigators ICMJE | Not Provided | ||||
PRS Account | Sanofi | ||||
Verification Date | July 2020 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |