Open-Label, Safety Dose Escalation and Pharmacokinetic - Full Text View

Sponsors and Collaborators:	Syntonix Pharmaceuticals, Inc. Biovitrum Biogen Idec
Information provided by:	Syntonix Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:	NCT00716716

Purpose

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.

Condition	Intervention	Phase
Hemophilia B	Drug: FIXFc	Phase I

Genetics Home Reference related topics: hemophilia L1 syndrome

Drug Information available for: Factor IX

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study
Official Title:	A Phase I/IIa Safety and Pharmacokinetic Study of Intravenous FIXFc in Previously Treated Hemophilia B

Further study details as provided by Syntonix Pharmaceuticals, Inc.:

Primary Outcome Measures:

• 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 ]

Secondary Outcome Measures:

• 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 ]

Estimated Enrollment:	14
Study Start Date:	April 2008
Estimated Study Completion Date:	February 2009
Estimated Primary Completion Date:	February 2009 (Final data collection date for primary outcome measure)

Intervention Details:

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.

Detailed Description:

A total of 14 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.

Eligibility

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

Criteria

Inclusion Criteria:

Are previously treated (PTPs) with severe (<1 IU/dL endogenous FIX level) hemophilia B and at least 150 prior documented exposure days to other FIX products.
Known gene defect. Documentation of genotype is mandatory for enrollment.
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.

Exclusion Criteria:

Presence of a major bleeding episode on Day 1 of study.
A FIX gene deletion that is either a large, partial or complete deletion or an Arg29 to stop nonsense genotype.
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 with an ALT or AST greater than 2 times upper limit of normal.
HIV-positive or known exposure to HIV.
Certain prior illnesses and other protocol-defined criteria.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00716716

Locations

United States, Indiana
Indiana Hemophilia & Thrombosis Center	Recruiting
Indianapolis, Indiana, United States, 46260
Contact: Angela Summers, LPN 317-871-0011 ext 218 asummers@IHTC.org
Principal Investigator: Amy Shapiro, MD
United States, North Carolina
University of North Carolina Medical School	Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Aime Grimsley, RN 919-966-4736 Aime_grimsley@med.unc.edu
Principal Investigator: Nigel Key, MD

Sponsors and Collaborators

Syntonix Pharmaceuticals, Inc.

Biovitrum

Biogen Idec

More Information

Responsible Party:	Syntonix Pharmaceuticals, Inc. ( Study Director )
Study ID Numbers:	SYN-FIXFc-07-001
Study First Received:	July 14, 2008
Last Updated:	July 23, 2008
ClinicalTrials.gov Identifier:	NCT00716716
Health Authority:	United States: Food and Drug Administration

Keywords provided by Syntonix Pharmaceuticals, Inc.:

Hemophilia B severe, previously treated patients

Study placed in the following topic categories:

Hemophilia B
Hemorrhagic Disorders
Genetic Diseases, Inborn
Hematologic Diseases

Blood Coagulation Disorders
Hemophilia A
Genetic Diseases, X-Linked
Hemostatic Disorders

Additional relevant MeSH terms:

Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders

ClinicalTrials.gov processed this record on January 16, 2009