Clinical Trial: Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Patients With Leber's Hereditary Optic Neuropathy

Brief Summary: This study is meant to assess the effectiveness of idebenone on visual function measures in patients with Leber's Hereditary Optic Neuropathy over a 6 months period.

Detailed Summary: The study involves 6 clinic visits.
Sponsor: Santhera Pharmaceuticals

Current Primary Outcome: Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye [ Time Frame: 24 weeks ]

Original Primary Outcome: Best recovery of logMAR visual acuity [ Time Frame: 24 weeks ]

Current Secondary Outcome:

  • Change in the patient's best logMAR visual acuity between baseline and week 24 [ Time Frame: 24 weeks ]
  • Change in scotoma area in both eyes [ Time Frame: Day -1, Week 4, Week 12, Week 24 ]
  • Change in optic nerve fibre layer thickness in both eyes [ Time Frame: Day -1, Week 4, Week 12, Week 24 ]
  • Colour contrast sensitivity in both eyes (in a subset of patients) [ Time Frame: Day -1, Week 4, Week 12, Week 24 ]
  • logMAR visual acuity as a continuous variable in both eyes [ Time Frame: Screening, Day -1, Week 4, Week 12, Week 24, Week 28 ]
  • Clinical Global Impression of Change [ Time Frame: Week 4, Week 12 and Week 24 ]
  • Change in Health-Related Quality of Life (HRQOL) [ Time Frame: Day -1, Week 4, Week 12, Week 24 ]
  • Change in self-reported general energy levels [ Time Frame: Day -1, Week 4, Week 12, Week 24, Week 28 ]
  • Proportion of patients in which visual acuity in the initially least affected eye does not deteriorate to 1.0 log MAR or more ( in LHON patients with eye still less affected than 0.5 logMAR at trial entry) [ Time Frame: 24 weeks ]
  • Plasma levels of idebenone matched to measures of efficacy and safety [ Time Frame: 24 weeks ]
  • • Best visual acuity at Week 24 (best eye at Week 24) compared to best visual acuity at Baseline (best eye at Baseline) [ Time Frame: 24 weeks ]
  • • Count of eyes/ patients for which the visual acuity improves between baseline and week 24 [ Time Frame: 24 weeks ]


Original Secondary Outcome:

  • Change in best logMAR [ Time Frame: 24 weeks ]
  • Change in scotoma area [ Time Frame: Day -1, Week 4, Week 12, Week 24 ]
  • Change in optic nerve fibre layer thickness [ Time Frame: Day -1, Week 4, Week 12, Week 24 ]
  • Change in colour contrast sensitivity in both eyes (in a subset of patients) [ Time Frame: Day -1, Week 4, Week 12, Week 24 ]
  • logMAR visual acuity [ Time Frame: Screening, Day -1, Week 4, Week 12, Week 24, Week 28 ]
  • Clinical Global Impression of Change [ Time Frame: Week 4, Week 12 and Week 24 ]
  • Change in Health-Related Quality of Life (HRQOL) [ Time Frame: Day -1, Week 4, Week 12, Week 24 ]
  • Change in self-reported general energy levels [ Time Frame: Day -1, Week 4, Week 12, Week 24, Week 28 ]
  • Proportion of patients in which visual acuity in the initially least affected eye does not deteriorate to 1.0 log MAR or more ( in LHON patients with eye still less affected than 0.5 logMAR at trial entry) [ Time Frame: 24 weeks ]
  • Plasma levels of idebenone matched to measures of efficacy and safety [ Time Frame: 24 weeks ]


Information By: Santhera Pharmaceuticals

Dates:
Date Received: September 4, 2008
Date Started: November 2007
Date Completion:
Last Updated: May 24, 2013
Last Verified: May 2013