Phase I/IIa Study of FIXFc in Hemophilia B Patients

This study has been completed.

Sponsor:

Collaborators:

Swedish Orphan Biovitrum

Biogen Idec

Information provided by:

Syntonix Pharmaceuticals, Inc.

ClinicalTrials.gov Identifier:

NCT00716716

First received: July 14, 2008

Last updated: November 1, 2009

Last verified: November 2009

History of Changes

Tracking Information
First Received Date ^ICMJE	July 14, 2008
Last Updated Date	November 1, 2009
Start Date ^ICMJE	April 2008
Primary Completion Date	October 2009 (final data collection date for primary outcome measure)
Current Primary Outcome Measures ^ICMJE (submitted: July 14, 2008)	• Safety of a single-dose of intravenous FIXFc. Safety will be evaluated by adverse events and laboratory changes over time. [ Time Frame: 45 days ] [ Designated as safety issue: No ]
Original Primary Outcome Measures ^ICMJE	Same as current
Change History	Complete list of historical versions of study NCT00716716 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ^ICMJE (submitted: July 14, 2008)	• The study will estimate the pharmacokinetic parameters of a long-acting FIX following a single intravenous dose. [ Time Frame: 45 days ] [ Designated as safety issue: No ]
Original Secondary Outcome Measures ^ICMJE	Same as current
Current Other Outcome Measures ^ICMJE	Not Provided
Original Other Outcome Measures ^ICMJE	Not Provided

Descriptive Information
Brief Title ^ICMJE	Phase I/IIa Study of FIXFc in Hemophilia B Patients
Official Title ^ICMJE	A Phase I/IIa Safety and Pharmacokinetic Study of Intravenous FIXFc in Previously Treated Hemophilia B
Brief Summary	Primary Outcome Measures: • Safety of a single-dose of intravenous FIXFc. Safety will be evaluated by adverse events and laboratory changes over time. Secondary Outcome Measures: • The study will estimate the pharmacokinetic parameters of a long-acting FIX following a single intravenous dose.
Detailed Description	A total of 10 severe hemophilia B previously treated patients will receive a single intravenous dose of FIXFc followed by a 30-day evaluation period. Patients will be enrolled sequentially based on safety at dose levels of 1, 5, 12.5, 25, 50, and 100 IU/kg.
Study Type ^ICMJE	Interventional
Study Phase	Phase 1
Study Design ^ICMJE	Allocation: Non-Randomized Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label
Condition ^ICMJE	Hemophilia B
Intervention ^ICMJE	Drug: FIXFc Single dose of FIXFc administered IV. Six (6) dose levels, 1, 5, 12.5, 25, 50, and 100 IU/kg, are planned for evaluation in the study. Other Name: No other names or serial numbers apply.
Study Arm (s)	Not Provided
Publications *	Shapiro AD, Ragni MV, Valentino LA, Key NS, Josephson NC, Powell JS, Cheng G, Thompson AR, Goyal J, Tubridy KL, Peters RT, Dumont JA, Euwart D, Li L, Hallén B, Gozzi P, Bitonti AJ, Jiang H, Luk A, Pierce GF. Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients. Blood. 2012 Jan 19;119(3):666-72. Epub 2011 Nov 22.
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information
Recruitment Status ^ICMJE	Completed
Estimated Enrollment ^ICMJE	10
Completion Date	October 2009
Primary Completion Date	October 2009 (final data collection date for primary outcome measure)
Eligibility Criteria ^ICMJE	Inclusion Criteria: 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. 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. No prior history of an allergic reaction or anaphylaxis associated with any FIX or IVIG administration. No concurrent autoimmune disease. At least 7 days since their last dose of FIX (wash-out period). Certain laboratory testing criteria and other protocol-defined criteria may apply. 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. Exclusion Criteria: Presence of a major bleeding episode on Day 1 of study. Any coagulation disorder in addition to hemophilia B. 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. A positive d-dimer at screening. Documented history of liver cirrhosis. 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. Certain prior illnesses and other protocol-defined criteria.
Gender	Male
Ages	18 Years and older
Accepts Healthy Volunteers	No
Contacts ^ICMJE	Contact information is only displayed when the study is recruiting subjects
Location Countries ^ICMJE	United States

Administrative Information
NCT Number ^ICMJE	NCT00716716
Other Study ID Numbers ^ICMJE	SYN-FIXFc-07-001
Has Data Monitoring Committee	Yes
Responsible Party	Study Director, Syntonix Pharmaceuticals, Inc.
Study Sponsor ^ICMJE	Syntonix Pharmaceuticals, Inc.
Collaborators ^ICMJE	Swedish Orphan Biovitrum Biogen Idec
Investigators ^ICMJE	Not Provided
Information Provided By	Syntonix Pharmaceuticals, Inc.
Verification Date	November 2009
^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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