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

Phase 2

Completed

• Conditions: Leber's Hereditary Optic Neuropathy
• Interventions: Drug: Idebenone; Drug: Placebo

• Registration Number: NCT00747487
• Lead Sponsor: Santhera Pharmaceuticals

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 Description

The study involves 6 clinic visits.

Study Timeline

• Study Start: 2007-11
• Study First Submitted: 2008-09-04
• Study First Submitted QC: 2008-09-04
• Study First Posted: 2008-09-05
• Primary Completion: 2010-02
• Study Completion: 2010-02
• Status Verified: 2013-05
• Last Update Submitted: 2013-05-24
• Last Update Posted: 2013-05-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 85

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
1	Idebenone	Idebenone
2	Placebo	Placebo

Primary Outcome Measures

Name	Time	Method
Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye	24 weeks

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (3)

Unité de recherche clinique Ophtalmologie- Hopital Notre-Dame; 🇨🇦 Montreal, Quebec, Canada

Clinical Research Facility, 4th Floor Leazes Wing, Royal Victoria Infirmary; 🇬🇧 Newcastle Upon Tyne, United Kingdom

Klinikum der Universität München - Grosshadern, Neurologische Klinik und Poliklinik; 🇩🇪 Munich, Germany