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.
The study involves 6 clinic visits.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
85
Unité de recherche clinique Ophtalmologie- Hopital Notre-Dame
Montreal, Quebec, Canada
Klinikum der Universität München - Grosshadern, Neurologische Klinik und Poliklinik
Munich, Germany
Clinical Research Facility, 4th Floor Leazes Wing, Royal Victoria Infirmary
Newcastle upon Tyne, United Kingdom
Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye
Time frame: 24 weeks
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
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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