Journal article
Randomized, double-blind, placebo-controlled study of interferon- γ 1b in Friedreich Ataxia
Annals of clinical and translational neurology, Vol.6(3), pp.546-553
03/2019
DOI: 10.1002/acn3.731
PMCID: PMC6414489
PMID: 30911578
Abstract
In vitro, in vivo, and open-label studies suggest that interferon gamma (IFN-
1b) may improve clinical features in Friedreich Ataxia through an increase in frataxin levels. The present study evaluates the efficacy and safety of IFN-
1b in the treatment of Friedreich Ataxia through a double-blind, multicenter, placebo-controlled trial.
Ninety-two subjects with FRDA between 10 and 25 years of age were enrolled. Subjects received either IFN-
1b or placebo for 6 months. The primary outcome measure was the modified Friedreich Ataxia Rating Scale (mFARS).
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.
This study provides no direct evidence for a beneficial effect of IFN-
1b in FRDA. The modest stabilization compared to natural history data leaves open the possibility that longer studies may demonstrate benefit.
Details
- Title: Subtitle
- Randomized, double-blind, placebo-controlled study of interferon- γ 1b in Friedreich Ataxia
- Creators
- David R Lynch - Division of Neurology Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Blvd Philadelphia Pennsylvania 19104-4318Lauren Hauser - Division of Neurology Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Blvd Philadelphia Pennsylvania 19104-4318Ashley McCormick - Division of Neurology Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Blvd Philadelphia Pennsylvania 19104-4318McKenzie Wells - Division of Neurology Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Blvd Philadelphia Pennsylvania 19104-4318Yi Na Dong - Division of Neurology Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Blvd Philadelphia Pennsylvania 19104-4318Shana McCormack - Division of Endocrinology & Diabetes Children's Hospital of Philadelphia Philadelphia 19104Kim Schadt - Division of Neurology Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Blvd Philadelphia Pennsylvania 19104-4318Susan Perlman - Department of Neurology University of California Los Angeles Box 956975, 1-167 RNRC Los Angeles California 90095Sub H Subramony - Department of Neurology University of Florida Room L3-100, McKnight Brain Institute, 1149 Newell Drive Gainesville Florida 32611Katherine D Mathews - Department of Pediatrics and Neurology University of Iowa Carver College of Medicine Iowa City IowaAlicia Brocht - Department of Neurology University of Rochester Rochester New York 14620Julie Ball - Horizon Pharma, Inc. Lake Forest Illinois 60045Renee Perdok - Horizon Pharma, Inc. Lake Forest Illinois 60045Amy Grahn - Horizon Pharma, Inc. Lake Forest Illinois 60045Tom Vescio - Horizon Pharma, Inc. Lake Forest Illinois 60045Jeffrey W Sherman - Horizon Pharma, Inc. Lake Forest Illinois 60045Jennifer M Farmer - Friedreich's Ataxia Research Alliance 533 W Uwchlan Ave Downingtown Pennsylvania 19335
- Resource Type
- Journal article
- Publication Details
- Annals of clinical and translational neurology, Vol.6(3), pp.546-553
- DOI
- 10.1002/acn3.731
- PMID
- 30911578
- PMCID
- PMC6414489
- NLM abbreviation
- Ann Clin Transl Neurol
- ISSN
- 2328-9503
- eISSN
- 2328-9503
- Publisher
- United States
- Grant note
- U54 NS053672 / NINDS NIH HHS
- Language
- English
- Date published
- 03/2019
- Academic Unit
- Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
- Record Identifier
- 9984070680602771
Metrics
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