Phase I/IIa Study of FIXFc in Hemophilia B Patients

Study Overview
=================
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.

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

Conditions
-----------------
Hemophilia B

Intervention / Treatment
-----------------
* Drug: rFIXFc


Participation Criteria
=================
Eligibility Criteria
-----------------
Key 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. Key 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. NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
Male

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: rFIXFc<br>Six intravenous (IV) dose levels, 1, 5, 12.5, 25, 50, and 100 IU/kg | Drug: rFIXFc<br>* As specified in the treatment arm<br>* Other names: FIXFc;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants experiencing Adverse Events |  | Up to 45 days | 

Secondary Outcome Measures

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

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Hemophilia B severe, previously treated patients