Brief Title
Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B
Official Title
B-LONG: An Open-Label, Multicenter Evaluation of the Safety, Pharmacokinetics, and Efficacy of Recombinant, Long-acting Coagulation Factor IX Fc Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding in Previously Treated Subjects With Severe Hemophilia B
Brief Summary
The primary objectives of the study were: to evaluate the safety and tolerability of rFIXFc;
to evaluate the efficacy of rFIXFc in all treatment arms; to evaluate the effectiveness of
prophylaxis over on-demand (episodic) therapy by comparing the annualized number of bleeding
episodes between participants receiving rFIXFc on each prevention (prophylaxis) regimen and
participants receiving rFIXFc on an episodic regimen. The secondary objectives of the study
were: to evaluate and assess the pharmacokinetic (PK) parameter estimates of rFIXFc and rFIX
(BeneFIX®) at baseline in the Sequential PK subgroup as well as rFIXFc at Week 26 (±1 week);
to evaluate participants' response to treatment; to evaluate rFIXFc consumption.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities
Secondary Outcome
Participant Assessment of Response to Injections to Treat a Bleeding Episode
Condition
Severe Hemophilia B
Intervention
Factor IX (rFIXFc)
Study Arms / Comparison Groups
Fixed Weekly Interval
Description: 50 IU/kg rFIXFc via intravenous (IV) injection once every 7 days initially, then at a dose indicated by the participant's baseline pharmacokinetic (PK) assessment that ensured a target trough of 1% to 3% above baseline or higher, as clinically indicated. Adjustments to the initial weekly dose of rFIXFc (50 IU/kg) were to be made based on baseline PK assessments, occurrence of spontaneous bleeding episodes, and the trough levels, which were to be monitored at Weeks 4, 16, 26, and 39. Prior to the first dose of rFIXFc, participants in the Sequential PK subgroup were to receive a single dose of 50 IU/kg BeneFIX administered IV in the clinic, followed by PK sampling. A single dose of 50 IU/kg rFIXFc was administered following a 120-hour washout from BeneFIX, followed by PK sampling for a baseline PK profiling. At Week 26 (±1 week) subjects were to receive a single dose of 50 IU/kg rFIXFc for repeat PK profiling.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
123
Start Date
December 2009
Completion Date
July 2012
Primary Completion Date
July 2012
Eligibility Criteria
Inclusion Criteria:
- Male and 12 years of age and older and weigh at least 40 kg
- Diagnosed with hemophilia B (baseline Factor IX level less than or equal to 2%)
- History of at least 100 exposure days to any Factor IX product
- Platelet count ≥100,000 cells/μL
Exclusion Criteria:
- History of Factor IX inhibitors
- Kidney or liver dysfunction
- Diagnosed with another coagulation defect other than hemophilia B
- Prior history of anaphylaxis associated with any Factor IX or intravenous (IV)
immunoglobulin administration
Gender
Male
Ages
12 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Medical Director, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT01027364
Organization ID
998HB102
Secondary IDs
2009-014295-21
Responsible Party
Sponsor
Study Sponsor
Bioverativ Therapeutics Inc.
Collaborators
Swedish Orphan Biovitrum
Study Sponsor
Medical Director, Study Director, Bioverativ Therapeutics Inc.
Verification Date
August 2018