Phase I/IIa Study of FIXFc in Hemophilia B Patients

Hemophilia B Clinical Trial

Official title:

A Phase I/IIa Safety and Pharmacokinetic Study of Intravenous FIXFc in Previously Treated Hemophilia B Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00716716
• Other study ID #: SYN-FIXFc-07-001
• Status: Completed
• Phase: Phase 1
• Start date: April 2008
• Est. completion date: October 2009

Study information

• Verified date: August 2018
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: October 2009
• Est. primary completion date: October 2009
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Drug:
• rFIXFc
As specified in the treatment arm

Locations

Country	Name	City	State
United States	Brigham & Women's Hospital	Boston	Massachusetts
United States	University of North Carolina Medical School	Chapel Hill	North Carolina
United States	RUSH University Medical Center	Chicago	Illinois
United States	Indiana Hemophilia & Thrombosis Center	Indianapolis	Indiana
United States	Hemophilia Center of Western PA	Pittsburgh	Pennsylvania
United States	Puget Sound Blood Center	Seattle	Washington

Sponsors (3)

Lead Sponsor	Collaborator
Bioverativ Therapeutics Inc.	Swedish Orphan Biovitrum, Syntonix Pharmaceuticals, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants experiencing Adverse Events		Up to 45 days
Secondary	Time to reach maximum concentration (Tmax)		Up to 45 days
Secondary	Maximum concentration (Cmax)		Up to 45 days
Secondary	Half-life (t½)		Up to 45 days
Secondary	Clearance (CL)		Up to 45 days
Secondary	Volume of distribution (Vd)		Up to 45 days
Secondary	Area under the curve (AUC)		Up to 45 days
Secondary	Mean residence time (MRT)		Up to 45 days
Secondary	Incremental recovery (K)		Up to 45 days
Secondary	Factor IX protein (FIX) activity		Up to 45 days
Secondary	Recombinant (FIXFc) concentration over time curves		up to 45 days