Phase I/IIa Study of FIXFc in Hemophilia B Patients

NCT ID: NCT00716716

Last Updated: 2020-12-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-04-30

Study Completion Date

2009-10-31

Brief Summary

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

Detailed Description

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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.

Conditions

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Hemophilia B

Keywords

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Hemophilia B severe, previously treated patients

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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rFIXFc

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

Group Type EXPERIMENTAL

rFIXFc

Intervention Type DRUG

As specified in the treatment arm

Interventions

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rFIXFc

As specified in the treatment arm

Intervention Type DRUG

Other Intervention Names

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Alprolix recombinant factor IX fusion protein BIIB029 FIXFc

Eligibility Criteria

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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.

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.
Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Syntonix Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role collaborator

Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role collaborator

Bioverativ Therapeutics Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Bioverativ Therapeutics Inc.

Locations

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RUSH University Medical Center

Chicago, Illinois, United States

Site Status

Indiana Hemophilia & Thrombosis Center

Indianapolis, Indiana, United States

Site Status

Brigham & Women's Hospital

Boston, Massachusetts, United States

Site Status

University of North Carolina Medical School

Chapel Hill, North Carolina, United States

Site Status

Hemophilia Center of Western PA

Pittsburgh, Pennsylvania, United States

Site Status

Puget Sound Blood Center

Seattle, Washington, United States

Site Status

Countries

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United States

References

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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.

Reference Type DERIVED
PMID: 22110246 (View on PubMed)

Other Identifiers

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SYN-FIXFc-07-001

Identifier Type: -

Identifier Source: org_study_id