Brief Title
Phase I/IIa Study of FIXFc in Hemophilia B Patients
Official Title
A Phase I/IIa Safety and Pharmacokinetic Study of Intravenous FIXFc in Previously Treated Hemophilia B Patients
Brief Summary
The primary objective of the study is to assess safety of FIXFc at doses ranging from 1 to
100 IU/kg.
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 Phase
Phase 1
Study Type
Interventional
Primary Outcome
Number of Participants experiencing Adverse Events
Secondary Outcome
Time to reach maximum concentration (Tmax)
Condition
Hemophilia B
Intervention
rFIXFc
Study Arms / Comparison Groups
rFIXFc
Description: Six intravenous (IV) dose levels, 1, 5, 12.5, 25, 50, and 100 IU/kg
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
10
Start Date
April 2008
Completion Date
October 2009
Primary Completion Date
October 2009
Eligibility 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
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Medical Director, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT00716716
Organization ID
SYN-FIXFc-07-001
Responsible Party
Sponsor
Study Sponsor
Bioverativ Therapeutics Inc.
Collaborators
Syntonix Pharmaceuticals, Inc.
Study Sponsor
Medical Director, Study Director, Bioverativ Therapeutics Inc.
Verification Date
August 2018