Phase I/IIa Study of FIXFc in Hemophilia B Patients

This study has been completed.
Sponsor:
Collaborators:
Syntonix Pharmaceuticals, Inc.
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Biogen Idec
ClinicalTrials.gov Identifier:
NCT00716716
First received: July 14, 2008
Last updated: March 19, 2015
Last verified: March 2015
  Purpose

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


Condition Intervention Phase
Hemophilia B
Drug: rFIXFc
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase I/IIa Safety and Pharmacokinetic Study of Intravenous FIXFc in Previously Treated Hemophilia B Patients

Resource links provided by NLM:


Further study details as provided by Biogen Idec:

Primary Outcome Measures:
  • Number of Participants experiencing Adverse Events [ Time Frame: Up to 45 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Time to reach maximum concentration (Tmax) [ Time Frame: Up to 45 days ] [ Designated as safety issue: No ]
  • Maximum concentration (Cmax) [ Time Frame: Up to 45 days ] [ Designated as safety issue: No ]
  • Half-life (t½) [ Time Frame: Up to 45 days ] [ Designated as safety issue: No ]
  • Clearance (CL) [ Time Frame: Up to 45 days ] [ Designated as safety issue: No ]
  • Volume of distribution (Vd) [ Time Frame: Up to 45 days ] [ Designated as safety issue: No ]
  • Area under the curve (AUC) [ Time Frame: Up to 45 days ] [ Designated as safety issue: No ]
  • Mean residence time (MRT) [ Time Frame: Up to 45 days ] [ Designated as safety issue: No ]
  • Incremental recovery (K) [ Time Frame: Up to 45 days ] [ Designated as safety issue: No ]
  • Factor IX protein (FIX) activity [ Time Frame: Up to 45 days ] [ Designated as safety issue: No ]
  • Recombinant (FIXFc) concentration over time curves [ Time Frame: up to 45 days ] [ Designated as safety issue: No ]

Enrollment: 10
Study Start Date: April 2008
Study Completion Date: October 2009
Primary Completion Date: October 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

Detailed Description:

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

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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

  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: NCT00716716

Locations
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
Biogen Idec
Syntonix Pharmaceuticals, Inc.
Swedish Orphan Biovitrum
Investigators
Study Director: Medical Director Biogen Idec
  More Information

No publications provided by Biogen Idec

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Biogen Idec
ClinicalTrials.gov Identifier: NCT00716716     History of Changes
Other Study ID Numbers: SYN-FIXFc-07-001
Study First Received: July 14, 2008
Last Updated: March 19, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Biogen Idec:
Hemophilia B severe, previously treated patients

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 23, 2015