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Study to Determine the Safety and Efficacy of rFIXFc in Untreated Males With Severe Hemophilia B (PUPs B-LONG)

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2017 by Bioverativ Therapeutics Inc.
Sponsor:
Collaborator:
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier:
NCT02234310
First received: July 17, 2014
Last updated: February 27, 2017
Last verified: February 2017
  Purpose
The primary objective of the study is to evaluate the safety of rFIXFc (recombinant coagulation factor IX Fc fusion protein, BIIB029) in previously untreated participants with severe hemophilia B. Secondary objectives are to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes in previously untreated participants (PUPs), and to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes in previously untreated participants.

Condition Intervention Phase
Hemophilia B
Biological: rFIXFc
Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Bioverativ Therapeutics Inc.:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Estimated Enrollment: 54
Study Start Date: November 2014
Estimated Study Completion Date: May 2022
Estimated Primary Completion Date: June 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: rFIXFc
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.
Biological: rFIXFc
Adjustments to the dose and interval of rFIXFc can be made in this study based on investigator discretion using available 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).
Other Names:
  • BIIB029
  • Alprolix
  • recombinant coagulation factor IX Fc fusion protein

  Eligibility

Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Weight >=3.5 kilogram at the time of informed consent
  • Severe hemophilia B defined as <=2 IU/dl (<=2%) endogenous factor IX documented in the medical record or as tested during the Screening Period.

Key Exclusion Criteria:

  • Prior history of inhibitor as defined by the reporting laboratory.
  • Measurable inhibitor activity at the Screening Visit, measured using the Nijmegen-modified Bethesda assay
  • History of hypersensitivity reactions associated with any intravenous immunoglobulin administration
  • History of hypersensitivity reactions associated with any recombinant coagulation factor IX Fc fusion protein (rFIXFc) administration
  • Other coagulation disorders in addition to hemophilia B
  • Injection with with a FIX concentrate other than rFIXFc
  • More than 3 injections of rFIXFc prior to confirmation of eligibility

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: NCT02234310

Contacts
Contact: Biogen clinicaltrials@biogen.com

  Show 61 Study Locations
Sponsors and Collaborators
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum
Investigators
Study Director: Medical Director Biogen
  More Information

Responsible Party: Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT02234310     History of Changes
Other Study ID Numbers: 998HB303
2013-003629-27 ( EudraCT Number )
Study First Received: July 17, 2014
Last Updated: February 27, 2017

Keywords provided by Bioverativ Therapeutics Inc.:
prophylaxis treatment
episodic treatment

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
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 March 24, 2017