Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B (Kids B-LONG)

This study has been completed.
Sponsor:
Collaborator:
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Biogen Idec
ClinicalTrials.gov Identifier:
NCT01440946
First received: September 16, 2011
Last updated: February 18, 2015
Last verified: February 2015
  Purpose

The primary objective of the study is to evaluate the safety of Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) in previously treated pediatric participants with hemophilia B. Secondary objectives of this study in this study population are as follows: To evaluate the efficacy of rFIXFc for prevention and treatment of bleeding episodes; To evaluate and assess the pharmacokinetics (PK) of rFIXFc; To evaluate rFIXFc consumption for prevention and treatment of bleeding episodes


Condition Intervention Phase
Hemophilia B
Drug: rFIXFc
Drug: FIX
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label, Multicenter Evaluation of Safety, Pharmacokinetics and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in the Prevention and Treatment of Bleeding Episodes in Pediatric Subjects With Hemophilia B

Resource links provided by NLM:


Further study details as provided by Biogen Idec:

Primary Outcome Measures:
  • Frequency of Inhibitor Development [ Time Frame: Up to 50 weeks +/- 7 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Number of annualized bleeding episodes [ Time Frame: Up to 50 weeks +/- 7 days ] [ Designated as safety issue: Yes ]
  • Assessments of response to treatment with rFIXFc for bleeding episodes [ Time Frame: Up to 50 weeks +/- 7 days ] [ Designated as safety issue: Yes ]
  • Total annualized rFIXFc consumption per participant for prevention of bleeding episodes [ Time Frame: Up to 50 weeks +/- 7 days ] [ Designated as safety issue: Yes ]
  • Total annualized rFIXFc consumption per participant for treatment of bleeding episodes [ Time Frame: Up to 50 weeks +/- 7 days ] [ Designated as safety issue: Yes ]
  • Time from last injection of rFIXFc to the bleeding episode [ Time Frame: Up to 50 weeks +/- 7 days ] [ Designated as safety issue: Yes ]
  • Number of injections and dose per injection of rFIXFc required to resolve a bleeding episode [ Time Frame: Up to 50 weeks +/- 7 days ] [ Designated as safety issue: Yes ]
  • Dose per injection of rFIXFc required to resolve a bleeding episode [ Time Frame: Up to 50 weeks +/- 7 days ] [ Designated as safety issue: Yes ]
  • Pharmacokinetics (PK) Parameter: Maximum Concentration (Cmax) of FIX and rFIXFc [ Time Frame: FIX Baseline Assessment (28 [±7] days prior to Day 1): pre-dose, up to 48 (±4) hours (2 days) after start of injection. rFIXFc Day 1 Assessment: pre-dose, up to 168 (±16) hours (7 days) after start of injection ] [ Designated as safety issue: No ]
  • PK Parameter: Half-life (t1/2) of FIX and rFIXFc [ Time Frame: FIX Baseline Assessment (28 [±7] days prior to Day 1): pre-dose, up to 48 (±4) hours (2 days) after start of injection. rFIXFc Day 1 Assessment: pre-dose, up to 168 (±16) hours (7 days) after start of injection ] [ Designated as safety issue: No ]
  • PK Parameter: Clearance (CL) of FIX and rFIXFc [ Time Frame: FIX Baseline Assessment (28 [±7] days prior to Day 1): pre-dose, up to 48 (±4) hours (2 days) after start of injection. rFIXFc Day 1 Assessment: pre-dose, up to 168 (±16) hours (7 days) after start of injection ] [ Designated as safety issue: No ]
  • PK Parameter: Volume of Distribution at Steady State (Vss) of FIX and rFIXFc [ Time Frame: FIX Baseline Assessment (28 [±7] days prior to Day 1): pre-dose, up to 48 (±4) hours (2 days) after start of injection. rFIXFc Day 1 Assessment: pre-dose, up to 168 (±16) hours (7 days) after start of injection ] [ Designated as safety issue: No ]
  • PK Parameter: Dose-normalized Area Under the Curve (DNAUC) of FIX and rFIXFc [ Time Frame: FIX Baseline Assessment (28 [±7] days prior to Day 1): pre-dose, up to 48 (±4) hours (2 days) after start of injection. rFIXFc Day 1 Assessment: pre-dose, up to 168 (±16) hours (7 days) after start of injection ] [ Designated as safety issue: No ]
  • PK Parameter: Mean Residence Time (MRT) of FIX and rFIXFc [ Time Frame: FIX Baseline Assessment (28 [±7] days prior to Day 1): pre-dose, up to 48 (±4) hours (2 days) after start of injection. rFIXFc Day 1 Assessment: pre-dose, up to 168 (±16) hours (7 days) after start of injection ] [ Designated as safety issue: No ]
  • PK Parameter: Incremental Recovery of FIX and rFIXFc [ Time Frame: FIX Baseline Assessment (28 [±7] days prior to Day 1): pre-dose, up to 48 (±4) hours (2 days) after start of injection. rFIXFc Day 1 Assessment: pre-dose, up to 168 (±16) hours (7 days) after start of injection ] [ Designated as safety issue: No ]

Enrollment: 30
Study Start Date: June 2012
Study Completion Date: November 2014
Primary Completion Date: November 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: rFIXFc Prophylaxis
At Baseline and at Day 1, participants will receive a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, lowest effective dosing regimen for prophylaxis the first prophylactic dose of approximately 50 to 60 IU/kg will be administered in clinic as an IV injection. Thereafter, treatment at the lowest effective dosing regimen for prophylaxis (adjusted in increments of 10 IU/kg, will initially be administered every 7 (±1) days for up to a total of 50 (±1) weeks to achieve at least 50 exposure days (EDs) to rFIXFc).
Drug: rFIXFc
IV administration
Other Names:
  • BIIB029
  • Recombinant Human Coagulation Factor IX Fusion Protein
  • Alprolix
Drug: FIX
IV administration

Detailed Description:

Previously treated pediatric participants will undergo an evaluation of the PK profile of pre-study factor IX (FIX) and rFIXFc, before starting prophylactic treatment with rFIXFc.

  Eligibility

Ages Eligible for Study:   up to 11 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Severe hemophilia B defined as ≤ 2IU/dl (≤2%)
  • Weight ≥ 13kg.
  • History of at least 50 documented prior exposure days to FIX.
  • No history of, or currently detectable, inhibitor.

Key Exclusion Criteria:

  • Other coagulation disorders in addition to Hemophilia B
  • History of anaphylaxis associated with any FIX or intravenous (IV) immunoglobulin administration

NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01440946

Locations
United States, Arizona
Research Site
Phoenix, Arizona, United States
United States, California
Research Site
Los Angeles, California, United States
Research Site
Sacramento, California, United States
United States, Georgia
Research Site
Atlanta, Georgia, United States
United States, Hawaii
Research Site
Honolulu, Hawaii, United States
United States, Indiana
Research Site
Indianapolis, Indiana, United States
United States, Michigan
Research Site
East Lansing, Michigan, United States
United States, Pennsylvania
Research Site
Pittsburgh, Pennsylvania, United States
Australia, Victoria
Research Site
Melbourne, Victoria, Australia
Australia, Western Australia
Research Site
West Perth, Western Australia, Australia
Hong Kong
Research Site
Hong Kong, Hong Kong
Ireland
Research Site
Dublin, Ireland
Netherlands
Research Site
Utrecht, Netherlands
South Africa
Research Site
Johannesburg, South Africa
United Kingdom
Research Site
Basingstoke, United Kingdom
Research Site
Cambridge, United Kingdom
Research Site
London, United Kingdom
Sponsors and Collaborators
Biogen Idec
Swedish Orphan Biovitrum
Investigators
Study Director: Medical Director Biogen Idec
  More Information

No publications provided

Responsible Party: Biogen Idec
ClinicalTrials.gov Identifier: NCT01440946     History of Changes
Other Study ID Numbers: 9HB02PED, 2011-003076-36
Study First Received: September 16, 2011
Last Updated: February 18, 2015
Health Authority: United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on April 16, 2015