Journal of Neurology

, 256:42 | Cite as

Clinical experience with high-dose idebenone in Friedreich ataxia

  • Jörg B. Schulz
  • Nicholas A. Di Prospero
  • Kenneth Fischbeck
Article

Abstract

Several reports in the literature describe the effects of low-dose (5 mg/kg/day) idebenone in significantly reducing cardiac hypertrophy in patients with Friedreich ataxia. However, the effects of idebenone on neurological function have not been reliably determined in these studies; when neurological parameters were reported, results were often inconclusive, usually because of subject heterogeneity and lack of adequate statistical power. In two of these studies, some patients showed beneficial effects of idebenone on their cardiomyopathy only when the dose was increased, prompting the systematic investigation of higher doses of idebenone. Following a phase 1 dose escalation study, a phase 2 tolerability and efficacy trial with low, intermediate, and high doses of idebenone was conducted. The results suggested that treatment with intermediate- and high-dose idebenone had beneficial effects on neurological symptoms. On the basis of these results, two phase 3 trials have been initiated, one in the United States with young ambulatory patients and one in Europe without limits on age and disease severity.

Key words

Friedreich ataxia idebenone rare disorders clinical trials cardiomyopathy neurologic ICARS FARS oxidative stress 

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Copyright information

© Steinkopff-Verlag 2009

Authors and Affiliations

  • Jörg B. Schulz
    • 1
  • Nicholas A. Di Prospero
    • 2
  • Kenneth Fischbeck
    • 3
  1. 1.Dept. of NeurologyUniversity Medical Centre, RWTH AachenAachenGermany
  2. 2.Johnson and Johnson Pharmaceutical Research and DevelopmentRaritanUSA
  3. 3.Neurogenetics BranchNational Institute of Neurological Disorders and Stroke, National Institute of HealthBethesdaUSA

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