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

ABSTRACT: Objective: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. Methods: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). 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-?1b in FRDA. The modest stabilization compared to natural history data leaves open the possibility that longer studies may demonstrate benefit.

SUBMITTER: Lynch DR 

PROVIDER: S-EPMC6414489 | biostudies-literature | 2019 Mar

REPOSITORIES: biostudies-literature