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

Sponsor

Santhera Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT00747487

Collaborator

(none)

Enrollment

Locations

Arms

Duration (Months)

28.3

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Leber's Hereditary Optic Neuropathy	Drug: Idebenone Drug: Placebo	Phase 2

Detailed Description

The study involves 6 clinic visits.

Study Design

Study Type:

Interventional

Actual Enrollment :

85 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official 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

Study Start Date :

Nov 1, 2007

Actual Primary Completion Date :

Feb 1, 2010

Actual Study Completion Date :

Feb 1, 2010

Arms and Interventions

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

Arm

Intervention/Treatment

Experimental: 1

Idebenone

Drug: Idebenone

Idebenone 900 mg/day

Placebo Comparator: 2

Placebo

Drug: Placebo

Placebo

Outcome Measures

Primary Outcome Measures

Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye [24 weeks]

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

14 Years to 65 Years

Sexes Eligible for Study:

All

Accepts 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

Contacts and Locations

Locations

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

Sponsors and Collaborators

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