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

• Status: Completed
• Trial end date: 2010-02-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Santhera Pharmaceuticals
• Treatments: Idebenone; Ubiquinone

Criteria

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