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Ann Clin Transl Neurol. 2019 Feb 27;6(3):546-553. doi: 10.1002/acn3.731. eCollection 2019 Mar.

Randomized, double-blind, placebo-controlled study of interferon-γ 1b in Friedreich Ataxia.

Author information

1
Division of Neurology Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Blvd Philadelphia Pennsylvania 19104-4318.
2
Division of Endocrinology & Diabetes Children's Hospital of Philadelphia Philadelphia 19104.
3
Department of Neurology University of California Los Angeles Box 956975, 1-167 RNRC Los Angeles California 90095.
4
Department of Neurology University of Florida Room L3-100, McKnight Brain Institute, 1149 Newell Drive Gainesville Florida 32611.
5
Department of Pediatrics and Neurology University of Iowa Carver College of Medicine Iowa City Iowa.
6
Department of Neurology University of Rochester Rochester New York 14620.
7
Horizon Pharma, Inc. Lake Forest Illinois 60045.
8
Friedreich's Ataxia Research Alliance 533 W Uwchlan Ave Downingtown Pennsylvania 19335.

Abstract

Objective:

In vitro, in vivo, and open-label studies suggest that interferon gamma (IFN-γ 1b) may improve clinical features in Friedreich Ataxia through an increase in frataxin levels. The present study evaluates the efficacy and safety of IFN-γ 1b in the treatment of Friedreich Ataxia through a double-blind, multicenter, placebo-controlled trial.

Methods:

Ninety-two subjects with FRDA between 10 and 25 years of age were enrolled. Subjects received either IFN-γ 1b or placebo for 6 months. The primary outcome measure was the modified Friedreich Ataxia Rating Scale (mFARS).

Results:

No difference was noted between the groups after 6 months of treatment in the mFARS or secondary outcome measures. No change was noted in buccal cell or whole blood frataxin levels. However, during an open-label extension period, subjects had a more stable course than expected based on natural history data.

Conclusions:

This study provides no direct evidence for a beneficial effect of IFN-γ1b in FRDA. The modest stabilization compared to natural history data leaves open the possibility that longer studies may demonstrate benefit.

Conflict of interest statement

Julie Ball, Renee Perdok, Amy Grahn, Tom Vescio, and Jeffrey W. Sherman are employees of Horizon Pharmaceutical. David Lynch also receives grants from the MDA, FDA, NIH, FARA, and Reata Pharmaceutical.

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