Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia

Summary

This study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.

Timeline

Start

2007-12

Primary completion

2009-04

Completion

2009-04

Outcome

Missed primary endpoint

paper For both end points, the difference between the idebenone and placebo groups was not statistically different. PMID 20697044 ↗

paper Idebenone did not significantly alter neurological function in Friedreich ataxia during the 6-month study. PMID 20697044 ↗

Publications

• Results Meier T, Perlman SL, Rummey C, Coppard NJ, Lynch DR. Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia: data from a 6-month controlled study followed by a 12-month open-label extension study. J Neurol. 2012 Feb;259(2):284-91. doi: 10.1007/s00415-011-6174-y. Epub 2011 Jul 22.
• Lagedrost SJ, Sutton MS, Cohen MS, Satou GM, Kaufman BD, Perlman SL, Rummey C, Meier T, Lynch DR. Idebenone in Friedreich ataxia cardiomyopathy-results from a 6-month phase III study (IONIA). Am Heart J. 2011 Mar;161(3):639-645.e1. doi: 10.1016/j.ahj.2010.10.038. Epub 2011 Jan 31.
• Lynch DR, Perlman SL, Meier T. A phase 3, double-blind, placebo-controlled trial of idebenone in friedreich ataxia. Arch Neurol. 2010 Aug;67(8):941-7. doi: 10.1001/archneurol.2010.168.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Idebenone	Small molecule	450 mg	—
Subject	Idebenone	Small molecule	900 mg	—
Subject	Idebenone	Small molecule	1350 mg	—
Subject	Idebenone	Small molecule	2250 mg	—

Indications