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Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Subjects With Hemophilia B

This study has been completed.
Sponsor:
Collaborator:
Swedish Orphan Biovitrum
Information provided by:
Biogen Idec
ClinicalTrials.gov Identifier:
NCT01027364
First received: December 4, 2009
Last updated: October 11, 2012
Last verified: October 2012
History of Changes
  Purpose

The study is to investigate the safety, pharmacokinetics (the determination of the concentration of the administered drug in blood over time), and efficacy (length of effect and duration of circulating) of recombinant Factor IX Fc fusion protein (rFIXFc) in previously-treated subjects with hemophilia B.


Condition Intervention Phase
Severe Hemophilia B
 Drug: rFIXFc
 Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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 Hemophilia B

Resource links provided by NLM:

Drug Information available for: Factor IX
U.S. FDA Resources

Further study details as provided by Biogen Idec:

Primary Outcome Measures:
  • Safety/tolerability include clinically notable changes from baseline in PE, vital signs, lab values, and incidence of AEs, including the incidence of inhibitor development [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ]
  • Number of breakthrough bleeding episodes with rFIXFc [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • To evaluate and assess the PK parameter estimates of rFIXFc and BeneFIX at baseline in the Sequential PK subgroup as well as rFIXFc at Week 26 [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ]
  • To evaluate the efficacy of rFIXFc used on-demand and surgical subgroup [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ]
  • To evaluate subjects' response to treatment [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ]
  • To evaluate rFIXFc consumption [ Time Frame: 156 weeks ] [ Designated as safety issue: Yes ]

Enrollment: 105
Study Start Date: December 2009
Study Completion Date: September 2012
Primary Completion Date: August 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: A:
Low dose prophylaxis
 Drug: rFIXFc
IV administration
Experimental: B
High dose prophylaxis
 Drug: rFIXFc
IV administration
Experimental: C
On-demand
 Drug: rFIXFc
IV administration
Experimental: D
Surgery
 Drug: rFIXFc
IV administration

Detailed Description:

The current hemophilia standard of care for the prevention of bleeds and arthropathy is to maintain FIX activity level above 1%. Due to the short half-life of the current FIX products, prophylaxis therapy will require injection of 2-3 times per week. Treatment usually involves intravenous access, an invasive procedure, especially difficult in children. Episodic treatment will involve 1-3 injections to treat bleeding episodes, depending on the severity of the hemorrhage. Severe hemophilia patients will treat prophylactically with the long-lasting recombinant factor IX Fc fusion protein (rFIXFc) in an interval to maintain FIX activity level for the prevention of bleeds. Any bleeding episodes will be reported. The response to treatment will also be recorded to access the effectiveness of the rFIXFc.

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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 FIX or IV immunoglobulin administration
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01027364

  Show 60 Study Locations
Sponsors and Collaborators
Biogen Idec
Swedish Orphan Biovitrum
  More Information

No publications provided

Responsible Party: Biogen-Idec Hemophilia Unit
ClinicalTrials.gov Identifier: NCT01027364     History of Changes
Other Study ID Numbers: 998HB102
Study First Received: December 4, 2009
Last Updated: October 11, 2012
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Hemophilia B
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on May 21, 2013