Phase I/IIa Study of FIXFc in Hemophilia B Patients

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. Read our disclaimer for details. Identifier: NCT00716716
Recruitment Status : Completed
First Posted : July 16, 2008
Last Update Posted : February 28, 2017
Syntonix Pharmaceuticals, Inc.
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Bioverativ Therapeutics Inc.

Brief Summary:
The primary objective of the study is to assess safety of FIXFc at doses ranging from 1 to 100 IU/kg.

Condition or disease Intervention/treatment Phase
Hemophilia B Drug: rFIXFc Phase 1

Detailed Description:
This study was previously posted by Syntonix Pharmaceuticals, Inc. In January, 2007, sponsorship of the trial was transferred to Biogen Idec.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I/IIa Safety and Pharmacokinetic Study of Intravenous FIXFc in Previously Treated Hemophilia B Patients
Study Start Date : April 2008
Actual Primary Completion Date : October 2009
Actual Study Completion Date : October 2009

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: rFIXFc
Six intravenous (IV) dose levels, 1, 5, 12.5, 25, 50, and 100 IU/kg
Drug: rFIXFc
As specified in the treatment arm
Other Names:
  • Alprolix
  • recombinant factor IX fusion protein
  • BIIB029
  • FIXFc

Primary Outcome Measures :
  1. Number of Participants experiencing Adverse Events [ Time Frame: Up to 45 days ]

Secondary Outcome Measures :
  1. Time to reach maximum concentration (Tmax) [ Time Frame: Up to 45 days ]
  2. Maximum concentration (Cmax) [ Time Frame: Up to 45 days ]
  3. Half-life (t½) [ Time Frame: Up to 45 days ]
  4. Clearance (CL) [ Time Frame: Up to 45 days ]
  5. Volume of distribution (Vd) [ Time Frame: Up to 45 days ]
  6. Area under the curve (AUC) [ Time Frame: Up to 45 days ]
  7. Mean residence time (MRT) [ Time Frame: Up to 45 days ]
  8. Incremental recovery (K) [ Time Frame: Up to 45 days ]
  9. Factor IX protein (FIX) activity [ Time Frame: Up to 45 days ]
  10. Recombinant (FIXFc) concentration over time curves [ Time Frame: up to 45 days ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Key Inclusion Criteria:

  1. Are previously treated (PTPs) with severe (<2 IU/dL endogenous FIX level) hemophilia B and at least 150 prior documented exposure days to other FIX products.
  2. Have no prior history of or currently detectable inhibitor defined as > 0.6 Bethesda units by the local lab. A family history of inhibitors will not exclude the patient.
  3. No prior history of an allergic reaction or anaphylaxis associated with any FIX or IVIG administration.
  4. No concurrent autoimmune disease.
  5. At least 7 days since their last dose of FIX (wash-out period).
  6. Certain laboratory testing criteria and other protocol-defined criteria may apply.
  7. HIV negative or if HIV positive with a CD4 count ≥ 200 cells/mm3. HIV patients are allowed to receive protease inhibitors per the discretion of the Investigator.

Key Exclusion Criteria:

  1. Presence of a major bleeding episode on Day 1 of study.
  2. Any coagulation disorder in addition to hemophilia B.
  3. A patient currently on a dose and regimen of FIX that would preclude participation in the study due to possible increased risk of bleeding because of the requirement to withhold treatment during the study period.
  4. A positive d-dimer at screening.
  5. Documented history of liver cirrhosis.
  6. Positive for HBsAg and/or positive for hepatitis C antibody, and/or HIV positive with an ALT or AST greater than 5 times upper limit of normal.
  7. Certain prior illnesses and other protocol-defined criteria.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

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 identifier (NCT number): NCT00716716

United States, Illinois
RUSH University Medical Center
Chicago, Illinois, United States, 60612
United States, Indiana
Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, United States, 46260
United States, Massachusetts
Brigham & Women's Hospital
Boston, Massachusetts, United States, 02115
United States, North Carolina
University of North Carolina Medical School
Chapel Hill, North Carolina, United States, 27599
United States, Pennsylvania
Hemophilia Center of Western PA
Pittsburgh, Pennsylvania, United States, 15213
United States, Washington
Puget Sound Blood Center
Seattle, Washington, United States, 98104
Sponsors and Collaborators
Bioverativ Therapeutics Inc.
Syntonix Pharmaceuticals, Inc.
Swedish Orphan Biovitrum
Study Director: Medical Director Biogen

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Bioverativ Therapeutics Inc. Identifier: NCT00716716     History of Changes
Other Study ID Numbers: SYN-FIXFc-07-001
First Posted: July 16, 2008    Key Record Dates
Last Update Posted: February 28, 2017
Last Verified: February 2017

Keywords provided by Bioverativ Therapeutics Inc.:
Hemophilia B severe, previously treated patients

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