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Phase I/IIa Study of FIXFc in Hemophilia B Patients

Primary Purpose

Hemophilia B

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

rFIXFc

About this trial

This is an interventional treatment trial for Hemophilia B focused on measuring Hemophilia B severe, previously treated patients

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

RUSH University Medical Center
Indiana Hemophilia & Thrombosis Center
Brigham & Women's Hospital
University of North Carolina Medical School
Hemophilia Center of Western PA
Puget Sound Blood Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

rFIXFc

Arm Description

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

Outcomes

Primary Outcome Measures

Number of Participants experiencing Adverse Events

Secondary Outcome Measures

Time to reach maximum concentration (Tmax)

Maximum concentration (Cmax)

Half-life (t½)

Clearance (CL)

Volume of distribution (Vd)

Area under the curve (AUC)

Mean residence time (MRT)

Incremental recovery (K)

Factor IX protein (FIX) activity

Recombinant (FIXFc) concentration over time curves

Full Information

NCT ID

NCT00716716

First Posted

July 14, 2008

Last Updated

December 16, 2020

Sponsor

Bioverativ Therapeutics Inc.

Collaborators

Syntonix Pharmaceuticals, Inc., Swedish Orphan Biovitrum

1. Study Identification

Unique Protocol Identification Number

NCT00716716

Brief Title

Phase I/IIa Study of FIXFc in Hemophilia B Patients

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2018

Overall Recruitment Status

Completed

Study Start Date

April 2008 (undefined)

Primary Completion Date

October 2009 (Actual)

Study Completion Date

October 2009 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Bioverativ Therapeutics Inc.

Collaborators

Syntonix Pharmaceuticals, Inc., Swedish Orphan Biovitrum

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hemophilia B

Keywords

Hemophilia B severe, previously treated patients

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

10 (Actual)

8. Arms, Groups, and Interventions

Arm Title

rFIXFc

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

rFIXFc

Other Intervention Name(s)

Alprolix, recombinant factor IX fusion protein, BIIB029, FIXFc

Intervention Description

As specified in the treatment arm

Primary Outcome Measure Information:

Title

Number of Participants experiencing Adverse Events

Time Frame

Up to 45 days

Secondary Outcome Measure Information:

Title

Time to reach maximum concentration (Tmax)

Time Frame

Up to 45 days

Title

Maximum concentration (Cmax)

Time Frame

Up to 45 days

Title

Half-life (t½)

Time Frame

Up to 45 days

Title

Clearance (CL)

Time Frame

Up to 45 days

Title

Volume of distribution (Vd)

Time Frame

Up to 45 days

Title

Area under the curve (AUC)

Time Frame

Up to 45 days

Title

Mean residence time (MRT)

Time Frame

Up to 45 days

Title

Incremental recovery (K)

Time Frame

Up to 45 days

Title

Factor IX protein (FIX) activity

Time Frame

Up to 45 days

Title

Recombinant (FIXFc) concentration over time curves

Time Frame

up to 45 days

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Medical Director

Organizational Affiliation

Bioverativ Therapeutics Inc.

Official's Role

Study Director

Facility Information:

Facility Name

RUSH University Medical Center

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60612

Country

United States

Facility Name

Indiana Hemophilia & Thrombosis Center

City

Indianapolis

State/Province

Indiana

ZIP/Postal Code

46260

Country

United States

Facility Name

Brigham & Women's Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

University of North Carolina Medical School

City

Chapel Hill

State/Province

North Carolina

ZIP/Postal Code

27599

Country

United States

Facility Name

Hemophilia Center of Western PA

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15213

Country

United States

Facility Name

Puget Sound Blood Center

City

Seattle

State/Province

Washington

ZIP/Postal Code

98104

Country

United States

12. IPD Sharing Statement

Citations:

PubMed Identifier

22110246

Citation

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, Hallen 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. doi: 10.1182/blood-2011-07-367003. Epub 2011 Nov 22.

Results Reference

derived

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Phase I/IIa Study of FIXFc in Hemophilia B Patients

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