Phase I/IIa Study of FIXFc in Hemophilia B Patients

Phase 1

Completed

• Conditions: Hemophilia B
• Interventions: Drug: rFIXFc

• Registration Number: NCT00716716
• Lead Sponsor: Bioverativ Therapeutics Inc.

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 Timeline

• Study Start: 2008-04
• Study First Submitted: 2008-07-14
• Study First Submitted QC: 2008-07-14
• Study First Posted: 2008-07-16
• Primary Completion: 2009-10
• Study Completion: 2009-10
• Status Verified: 2018-08
• Last Update Submitted: 2020-12-16
• Last Update Posted: 2020-12-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 10

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
rFIXFc	rFIXFc	Six intravenous (IV) dose levels, 1, 5, 12.5, 25, 50, and 100 IU/kg

Primary Outcome Measures

Name	Time	Method
Number of Participants experiencing Adverse Events	Up to 45 days

Secondary Outcome Measures

Name	Time	Method
Half-life (t½)	Up to 45 days
Clearance (CL)	Up to 45 days
Mean residence time (MRT)	Up to 45 days
Incremental recovery (K)	Up to 45 days
Time to reach maximum concentration (Tmax)	Up to 45 days
Maximum concentration (Cmax)	Up to 45 days
Area under the curve (AUC)	Up to 45 days
Volume of distribution (Vd)	Up to 45 days
Factor IX protein (FIX) activity	Up to 45 days
Recombinant (FIXFc) concentration over time curves	up to 45 days

Trial Locations

Locations (6)

Puget Sound Blood Center; 🇺🇸 Seattle, Washington, United States

Brigham & Women's Hospital; 🇺🇸 Boston, Massachusetts, United States

Hemophilia Center of Western PA; 🇺🇸 Pittsburgh, Pennsylvania, United States

RUSH University Medical Center; 🇺🇸 Chicago, Illinois, United States

Indiana Hemophilia & Thrombosis Center; 🇺🇸 Indianapolis, Indiana, United States

University of North Carolina Medical School; 🇺🇸 Chapel Hill, North Carolina, United States