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

May 24, 2013 updated by: Santhera Pharmaceuticals

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

The study involves 6 clinic visits.

Study Type

Interventional

Enrollment (Actual)

85

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Quebec
      • Montreal, Quebec, Canada, H2L 4M1
        • Unité de recherche clinique Ophtalmologie- Hopital Notre-Dame
  • Germany
      • Munich, Germany, 81377
        • Klinikum der Universität München - Grosshadern, Neurologische Klinik und Poliklinik
  • United Kingdom
      • Newcastle Upon Tyne, United Kingdom, NE1 4LP
        • Clinical Research Facility, 4th Floor Leazes Wing, Royal Victoria Infirmary

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years to 63 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: 2
Placebo
Drug: Placebo
Placebo
Experimental: 1
Idebenone
Drug: Idebenone
Idebenone 900 mg/day

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Santhera Pharmaceuticals

Investigators

  • Principal Investigator: Prof Patrick F Chinnery, MD, Clinical Research Facility, 4th Floor Leazes Wing, Royal Victoria Infirmary
  • Principal Investigator: Prof Thomas Klopstock, MD, Klinikum der Universität München - Grosshadern, Neurologische Klinik und Poliklinik

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2007

Primary Completion (Actual)

February 1, 2010

Study Completion (Actual)

February 1, 2010

Study Registration Dates

First Submitted

September 4, 2008

First Submitted That Met QC Criteria

September 4, 2008

First Posted (Estimate)

September 5, 2008

Study Record Updates

Last Update Posted (Estimate)

May 27, 2013

Last Update Submitted That Met QC Criteria

May 24, 2013

Last Verified

May 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SNT-II-003

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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