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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.
Verified October 2017 by Bioverativ Therapeutics Inc.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02234310
First Posted: September 9, 2014
Last Update Posted: October 16, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Bioverativ Therapeutics Inc.
  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: None (Open Label)
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 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).
Other Names:
  • BIIB029
  • Alprolix
  • recombinant coagulation factor IX Fc fusion protein

  Eligibility

Information from the National Library of Medicine

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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:

  • History of positive inhibitor testing. A prior history of inhibitors is defined based on a patient's historical positive inhibitor test using the local laboratory Bethesda value for a positive inhibitor test (that is equal to or above lower limit of detection)
  • History of hypersensitivity reactions associated with any Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) administration
  • Exposure to blood components or injection with a coagulation factor IX (FIX) concentrate (including plasma derived) other than rFIXFc
  • Injection with commercially available rFIXFc more than 28 days prior to Screening
  • More than 3 injections of commercially available rFIXFc prior to confirmation of eligibility
  • Other coagulation disorders in addition to hemophilia B
  • Any concurrent clinically significant major disease that, in the opinion of the Investigator, would make the subject unsuitable for enrollment (example HIV infection with CD4 lymphocyte count less than (<)200 cells/microliter (mcL) or a viral load greater than (>)200 particles/mcL, or any other known congenital or acquired immunodeficiency)
  • Current systemic treatment with chemotherapy and/or other immunosuppressant drugs. Use of steroids for treatment of asthma or management of acute allergic episodes or otherwise life-threatening episodes is allowed. Treatment in these circumstances should not exceed a 14-day duration
  • Participation within the past 30 days in any other clinical study involving investigational treatment
  • Current enrollment in any other clinical study involving investigational treatment
  • Inability to comply with study requirements
  • Other unspecified reasons that, in the opinion of the Investigator or Bioverativ, make the subject unsuitable for enrollment

NOTE: Other protocol-defined inclusion/exclusion criteria may apply.

  Contacts and Locations
Information from the National Library of Medicine

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


Contacts
Contact: Bioverativ clinicaltrials@bioverativ.com

  Show 60 Study Locations
Sponsors and Collaborators
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum
Investigators
Study Director: Medical Director Bioverativ Therapeutics Inc.
  More Information

Responsible Party: Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT02234310     History of Changes
Other Study ID Numbers: 998HB303
2013-003629-27 ( EudraCT Number )
First Submitted: July 17, 2014
First Posted: September 9, 2014
Last Update Posted: October 16, 2017
Last Verified: October 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