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Phase I/IIa Study of FIXFc in Hemophilia B Patients

Sponsor
Bioverativ Therapeutics Inc. (Industry)
Overall Status
Completed
CT.gov ID
NCT00716716
Collaborator
Syntonix Pharmaceuticals, Inc. (Industry), Swedish Orphan Biovitrum (Industry)
10
Enrollment
6
Locations
1
Arm
18
Duration (Months)
1.7
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

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
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. In February, 2017, sponsorship of the trial was transferred to Bioverativ.

Study Design

Study Type:
Interventional
Actual Enrollment :
10 participants
Allocation:
N/A
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 :
Apr 1, 2008
Actual Primary Completion Date :
Oct 1, 2009
Actual Study Completion Date :
Oct 1, 2009

Arms and Interventions

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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Number of Participants experiencing Adverse Events [Up to 45 days]

    Secondary Outcome Measures

    1. Time to reach maximum concentration (Tmax) [Up to 45 days]

    2. Maximum concentration (Cmax) [Up to 45 days]

    3. Half-life (t½) [Up to 45 days]

    4. Clearance (CL) [Up to 45 days]

    5. Volume of distribution (Vd) [Up to 45 days]

    6. Area under the curve (AUC) [Up to 45 days]

    7. Mean residence time (MRT) [Up to 45 days]

    8. Incremental recovery (K) [Up to 45 days]

    9. Factor IX protein (FIX) activity [Up to 45 days]

    10. Recombinant (FIXFc) concentration over time curves [up to 45 days]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 RUSH University Medical Center Chicago Illinois United States 60612
    2 Indiana Hemophilia & Thrombosis Center Indianapolis Indiana United States 46260
    3 Brigham & Women's Hospital Boston Massachusetts United States 02115
    4 University of North Carolina Medical School Chapel Hill North Carolina United States 27599
    5 Hemophilia Center of Western PA Pittsburgh Pennsylvania United States 15213
    6 Puget Sound Blood Center Seattle Washington United States 98104

    Sponsors and Collaborators

    • Bioverativ Therapeutics Inc.
    • Syntonix Pharmaceuticals, Inc.
    • Swedish Orphan Biovitrum

    Investigators

    • Study Director: Medical Director, Bioverativ Therapeutics Inc.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Bioverativ Therapeutics Inc.
    ClinicalTrials.gov Identifier:
    NCT00716716
    Other Study ID Numbers:
    • SYN-FIXFc-07-001
    First Posted:
    Jul 16, 2008
    Last Update Posted:
    Dec 19, 2020
    Last Verified:
    Aug 1, 2018
    Keywords provided by Bioverativ Therapeutics Inc.
    Hemophilia B severe, previously treated patients
    Additional relevant MeSH terms:
    Hemophilia A
    Hemophilia B

    Study Results

    No Results Posted as of Dec 19, 2020