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

Santhera

Status and phase

Completed

Phase 2

Conditions

Leber's Hereditary Optic Neuropathy

Treatments

Drug: Placebo

Drug: Idebenone

Study type

Interventional

Funder types

Industry

Identifiers

NCT00747487

SNT-II-003

Details and patient eligibility

About

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.

Full description

The study involves 6 clinic visits.

Enrollment

85 patients

Sex

All

Ages

14 to 65 years old

Volunteers

No Healthy Volunteers

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