Skip to main content
OpenTrials
Completed

NCT Number: NCT00747487

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Leber's Hereditary Optic Neuropathy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cranial Nerve Diseases Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Optic Atrophies, Hereditary Optic Atrophy Optic Atrophy, Hereditary, Leber Optic Nerve Diseases

Age range

14 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

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

Loading trial locations.

About this study

The study involves 6 clinic visits.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Idebenone

Drug

Idebenone 900 mg/day

Placebo

Drug

Placebo

Primary outcomes

  1. Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye

    Time frame: 24 weeks

Secondary outcomes

  1. Change in the patient's best logMAR visual acuity between baseline and week 24

    Time frame: 24 weeks

  2. Change in scotoma area in both eyes

    Time frame: Day -1, Week 4, Week 12, Week 24

  3. Change in optic nerve fibre layer thickness in both eyes

    Time frame: Day -1, Week 4, Week 12, Week 24

  4. Colour contrast sensitivity in both eyes (in a subset of patients)

    Time frame: Day -1, Week 4, Week 12, Week 24

  5. logMAR visual acuity as a continuous variable in both eyes

    Time frame: Screening, Day -1, Week 4, Week 12, Week 24, Week 28

  6. Clinical Global Impression of Change

    Time frame: Week 4, Week 12 and Week 24

  7. Change in Health-Related Quality of Life (HRQOL)

    Time frame: Day -1, Week 4, Week 12, Week 24

  8. Change in self-reported general energy levels

    Time frame: Day -1, Week 4, Week 12, Week 24, Week 28

  9. 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

  10. Plasma levels of idebenone matched to measures of efficacy and safety

    Time frame: 24 weeks

  11. • 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

  12. • Count of eyes/ patients for which the visual acuity improves between baseline and week 24

    Time frame: 24 weeks

Sponsors and collaborators

Lead sponsor

Santhera Pharmaceuticals

Industry

Registry information

Official study title

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

Acronym: RHODOS

Important dates

Study start
2007
Primary completion
2010
Study completion
2010
First posted
Sep 5, 2008
Registry last updated
May 27, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.