Study to Determine the Safety and Efficacy of rFIXFc in Untreated Males With Severe Hemophilia B (PUPs B-LONG)
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|ClinicalTrials.gov Identifier: NCT02234310|
Recruitment Status : Recruiting
First Posted : September 9, 2014
Last Update Posted : February 5, 2018
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia B||Biological: rFIXFc||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||54 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official 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|
|Study Start Date :||November 2014|
|Estimated Primary Completion Date :||June 2019|
|Estimated Study Completion Date :||March 2022|
rFIXFc is administered by intravenous injection. A prophylaxis regimen will start prior to or immediately following the third occurrence of hemarthrosis (joint bleed). The recommended starting prophylactic dose of rFIXFc is 50 IU/kg weekly. Treatment will continue until the participant has reached at least 100 exposure days to rFIXFc. Optional episodic treatment doses are determined by the investigator and range from 25% Factor IX level for minor bleeding episodes to 100% for major episodes or prior to surgery. Single injections of rFIXFc up to 200 IU/kg are allowed.
Adjustments to the dose and interval of rFIXFc can be 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 is an option to start study dosing as episodic treatment (on-demand).
- 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.
- Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) per Participant [ Time Frame: For the duration of study participation, approximately 3 years ]
- Annualized Number of Spontaneous Joint Bleeding Episodes per Participant [ Time Frame: For the duration of study participation, approximately 3 years ]
- Response to Treatment for Bleeding Episodes [ Time Frame: For the duration of study participation, approximately 3 years ]Response to treatment for bleeding episodes uses a 4-point bleeding response scale that the investigator completes for bleeding episodes treated in the clinic, and the parent or caregiver completes for all other bleeding episodes.
- Total Number of Exposure Days per Participant per Year [ Time Frame: For the duration of study participation, approximately 3 years ]
- Total Annualized rFIXFc Consumption per Participant for the Prevention and Treatment of Bleeding Episodes [ Time Frame: For the duration of study participation, approximately 3 years ]
- Number of Injections of rFIXFc Required to Resolve a Bleeding Episode [ Time Frame: For the duration of study participation, approximately 3 years ]
- Dose per Injection of rFIXFc Required to Resolve a Bleeding Episode [ Time Frame: For the duration of study participation, approximately 3 years ]
- rFIXFc Incremental Recovery [ Time Frame: Predose; within 30 minutes of the injection and at 10 (±5) minutes post dose ]Blood samples will be taken at trough for assessment of incremental recovery, measured by the one-stage clotting assay.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02234310
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|Study Director:||Medical Director||Bioverativ Therapeutics Inc.|