Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B (PUPs B-LONG)
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ClinicalTrials.gov Identifier: NCT02234310 |
Recruitment Status :
Completed
First Posted : September 9, 2014
Results First Posted : July 31, 2020
Last Update Posted : July 31, 2020
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Sponsor:
Bioverativ, a Sanofi company
Collaborator:
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Sanofi ( Bioverativ, a Sanofi company )
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Study Type | Interventional |
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Study Design | Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
Condition |
Hemophilia B |
Intervention |
Biological: rFIXFc |
Enrollment | 33 |
Participant Flow
Recruitment Details | The study was conducted at 28 active centers in 11 countries. Participants were screened between 13-Nov-2014 to 23-Jul-2018. |
Pre-assignment Details | A total of 38 participants were screened. A total of 33 participants were enrolled, 22 participants began study participation on episodic treatment regimen and 11 participants began study participation on prophylactic treatment regimen. |
Arm/Group Title | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) |
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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 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. |
Period Title: Overall Study | |
Started | 33 |
Episodic Treatment Regimen | 22 [1] |
Prophylactic Treatment Regimen | 28 [2] |
Completed | 27 |
Not Completed | 6 |
Reason Not Completed | |
Physician Decision | 1 |
Adverse Event | 1 |
Eligibility Criteria not fulfilled | 2 |
Withdrawal by Subject | 2 |
[1]
Participants were treated in more than one regimen and counted in all categories wherever applicable
[2]
17 participants among the 22 who started in episodic regimen, switched to the prophylactic regimen.
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Baseline Characteristics
Arm/Group Title | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | |
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Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which >=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 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. | |
Overall Number of Baseline Participants | 33 | |
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Analysis was performed on Safety Analysis Set which included all participants who received at least 1 dose of study rFIXFc.
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Age, Continuous
Median (Full Range) Unit of measure: Years |
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Number Analyzed | 33 participants | |
0.60
(0.08 to 2.00)
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 33 participants | |
Female |
0 0.0%
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Male |
33 100.0%
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Race/Ethnicity, Customized
Measure Type: Count of Participants Unit of measure: Participants |
Number Analyzed | 33 participants |
White |
22 66.7%
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Black or African-American |
1 3.0%
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Asian |
1 3.0%
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American Indian or Alaska Native |
0 0.0%
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Native Hawaiian or other Pacific Islander |
0 0.0%
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Unknown or Not reported |
5 15.2%
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Other |
4 12.1%
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Outcome Measures
Adverse Events
Limitations and Caveats
[Not Specified]
More Information
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
Results Point of Contact
Name/Title: | Trial Transparency Team |
Organization: | Bioverativ, a Sanofi company |
Phone: | 800-633-1610 ext 6 |
EMail: | Contact-US@sanofi.com |
Responsible Party: | Sanofi ( Bioverativ, a Sanofi company ) |
ClinicalTrials.gov Identifier: | NCT02234310 |
Other Study ID Numbers: |
998HB303 2013-003629-27 ( EudraCT Number ) |
First Submitted: | July 17, 2014 |
First Posted: | September 9, 2014 |
Results First Submitted: | July 15, 2020 |
Results First Posted: | July 31, 2020 |
Last Update Posted: | July 31, 2020 |