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

Study Overview
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Brief Summary
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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 Description
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The study involves 6 clinic visits.

Official Title
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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

Conditions
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Leber's Hereditary Optic Neuropathy

Intervention / Treatment
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* Drug: Idebenone
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age > or = 14 years and < 65 years Impaired visual acuity in at least one eye due to LHON Onset of visual loss due to LHON lies five years or less prior to Baseline Confirmation of either G11778A, T14484C or G3460A LHON mtDNA mutations at >60% in blood No explanation for the visual failure besides LHON Body weight ≥ 45 kg Negative urine pregnancy test at Screening and at Baseline (women of childbearing potential). Exclusion Criteria: Treatment with Coenzyme Q10 or idebenone within 1 month prior to Baseline Pregnancy and/or breast-feeding Weekly alcohol intake 35 units (men) or 24 units (women) Current drug abuse Clinically significant abnormalities of clinical haematology or biochemistry including, but not limited to, elevations greater than 2 times the upper limit of normal of AST, ALT or creatinine Participation in another clinical trial of any investigational drug within 3 months prior to Baseline Other factor that, in the investigator's opinion, excludes the patient from entering the study

Ages Eligible for Study
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Minimum Age: 14 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>Idebenone | Drug: Idebenone<br>* Idebenone 900 mg/day<br>|
| Placebo Comparator: 2<br>Placebo | Drug: Placebo<br>* Placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye |  | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in the patient's best logMAR visual acuity between baseline and week 24 |  | 24 weeks | 
| Change in scotoma area in both eyes |  | Day -1, Week 4, Week 12, Week 24 | 
| Change in optic nerve fibre layer thickness in both eyes |  | Day -1, Week 4, Week 12, Week 24 | 
| Colour contrast sensitivity in both eyes (in a subset of patients) |  | Day -1, Week 4, Week 12, Week 24 | 
| logMAR visual acuity as a continuous variable in both eyes |  | Screening, Day -1, Week 4, Week 12, Week 24, Week 28 | 
| Clinical Global Impression of Change |  | Week 4, Week 12 and Week 24 | 
| Change in Health-Related Quality of Life (HRQOL) |  | Day -1, Week 4, Week 12, Week 24 | 
| Change in self-reported general energy levels |  | 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) |  | 24 weeks | 
| Plasma levels of idebenone matched to measures of efficacy and safety |  | 24 weeks | 
| • Best visual acuity at Week 24 (best eye at Week 24) compared to best visual acuity at Baseline (best eye at Baseline) |  | 24 weeks | 
| • Count of eyes/ patients for which the visual acuity improves between baseline and week 24 |  | 24 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Leber, LHON, Leber's Hereditary Optic Neuropathy, Idebenone