Randomized, double‐blind, placebo‐controlled study of interferon‐γ 1b in Friedreich Ataxia. Issue 3 (27th February 2019)

• Record Type: Journal Article
• Authors: Lynch, David R.; Hauser, Lauren; McCormick, Ashley; Wells, McKenzie; Dong, Yi Na; McCormack, Shana; Schadt, Kim; Perlman, Susan; Subramony, Sub H.; Mathews, Katherine D.; Brocht, Alicia; Ball, Julie; Perdok, Renee; Grahn, Amy; Vescio, Tom; Sherman, Jeffrey W.; Farmer, Jennifer M.
• Is Part Of: Annals of clinical and translational neurology. Volume 6:Issue 3(2019)
• Page Start: 546
• Page End: 553
• Publication Date: 2019-02-27
• Subjects: Nervous system -- Diseases -- Periodicals; Neurology -- Periodicals; 616.8005
• DOI: 10.1002/acn3.731
• Languages: English
• ISSNs: 2328-9503

Abstract

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‐ γ 1b in FRDA. The modest stabilization compared to natural history data leaves open the possibility that longer studies may demonstrate benefit.