Back to results

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

Primary Purpose

Leber's Hereditary Optic Neuropathy

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

Idebenone

Placebo

About this trial

This is an interventional treatment trial for Leber's Hereditary Optic Neuropathy focused on measuring Leber, LHON, Leber's Hereditary Optic Neuropathy, Idebenone

Eligibility Criteria

14 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

Unité de recherche clinique Ophtalmologie- Hopital Notre-Dame
Klinikum der Universität München - Grosshadern, Neurologische Klinik und Poliklinik
Clinical Research Facility, 4th Floor Leazes Wing, Royal Victoria Infirmary

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Arm Description

Idebenone

Placebo

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Change in scotoma area in both eyes

Change in optic nerve fibre layer thickness in both eyes

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

logMAR visual acuity as a continuous variable in both eyes

Clinical Global Impression of Change

Change in Health-Related Quality of Life (HRQOL)

Change in self-reported general energy levels

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)

Plasma levels of idebenone matched to measures of efficacy and safety

• Best visual acuity at Week 24 (best eye at Week 24) compared to best visual acuity at Baseline (best eye at Baseline)

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

Full Information

NCT ID

NCT00747487

First Posted

September 4, 2008

Last Updated

May 24, 2013

Sponsor

Santhera Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT00747487

Brief Title

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

Acronym

RHODOS

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 Type

Interventional

2. Study Status

Record Verification Date

May 2013

Overall Recruitment Status

Completed

Study Start Date

November 2007 (undefined)

Primary Completion Date

February 2010 (Actual)

Study Completion Date

February 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Santhera Pharmaceuticals

4. Oversight

Data Monitoring Committee

Yes

5. 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.

Detailed Description

The study involves 6 clinic visits.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Leber's Hereditary Optic Neuropathy

Keywords

Leber, LHON, Leber's Hereditary Optic Neuropathy, Idebenone

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

85 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Arm Type

Experimental

Arm Description

Idebenone

Arm Title

Arm Type

Placebo Comparator

Arm Description

Placebo

Intervention Type

Drug

Intervention Name(s)

Idebenone

Intervention Description

Idebenone 900 mg/day

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Placebo

Primary Outcome Measure Information:

Title

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

Time Frame

24 weeks

Secondary Outcome Measure Information:

Title

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

Time Frame

24 weeks

Title

Change in scotoma area in both eyes

Time Frame

Day -1, Week 4, Week 12, Week 24

Title

Change in optic nerve fibre layer thickness in both eyes

Time Frame

Day -1, Week 4, Week 12, Week 24

Title

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

Time Frame

Day -1, Week 4, Week 12, Week 24

Title

logMAR visual acuity as a continuous variable in both eyes

Time Frame

Screening, Day -1, Week 4, Week 12, Week 24, Week 28

Title

Clinical Global Impression of Change

Time Frame

Week 4, Week 12 and Week 24

Title

Change in Health-Related Quality of Life (HRQOL)

Time Frame

Day -1, Week 4, Week 12, Week 24

Title

Change in self-reported general energy levels

Time Frame

Day -1, Week 4, Week 12, Week 24, Week 28

Title

Time Frame

24 weeks

Title

Plasma levels of idebenone matched to measures of efficacy and safety

Time Frame

24 weeks

Title

• 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

Title

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

Time Frame

24 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

14 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Prof Patrick F Chinnery, MD

Organizational Affiliation

Clinical Research Facility, 4th Floor Leazes Wing, Royal Victoria Infirmary

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Prof Thomas Klopstock, MD

Organizational Affiliation

Klinikum der Universität München - Grosshadern, Neurologische Klinik und Poliklinik

Official's Role

Principal Investigator

Facility Information:

Facility Name

Unité de recherche clinique Ophtalmologie- Hopital Notre-Dame

City

Montreal

State/Province

Quebec

ZIP/Postal Code

H2L 4M1

Country

Canada

Facility Name

Klinikum der Universität München - Grosshadern, Neurologische Klinik und Poliklinik

City

Munich

ZIP/Postal Code

81377

Country

Germany

Facility Name

Clinical Research Facility, 4th Floor Leazes Wing, Royal Victoria Infirmary

City

Newcastle Upon Tyne

ZIP/Postal Code

NE1 4LP

Country

United Kingdom

12. IPD Sharing Statement

Citations:

PubMed Identifier

21788663

Citation

Klopstock T, Yu-Wai-Man P, Dimitriadis K, Rouleau J, Heck S, Bailie M, Atawan A, Chattopadhyay S, Schubert M, Garip A, Kernt M, Petraki D, Rummey C, Leinonen M, Metz G, Griffiths PG, Meier T, Chinnery PF. A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy. Brain. 2011 Sep;134(Pt 9):2677-86. doi: 10.1093/brain/awr170. Epub 2011 Jul 25.

Results Reference

result

PubMed Identifier

23263355

Citation

Rudolph G, Dimitriadis K, Buchner B, Heck S, Al-Tamami J, Seidensticker F, Rummey C, Leinonen M, Meier T, Klopstock T. Effects of idebenone on color vision in patients with leber hereditary optic neuropathy. J Neuroophthalmol. 2013 Mar;33(1):30-6. doi: 10.1097/WNO.0b013e318272c643.

Results Reference

result

Links:

URL

http://www.santhera.com/index.php?mid=101&vid=&lang=en

Description

Website for LHON patients in Canada

URL

http://www.nefo.med.uni-muenchen.de/Forschungshauptseite/klinstudien/therapeutic-trial-of-idebenone-in-lhon

Description

Website for LHON patients in Germany

Learn more about this trial

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

We'll reach out to this number within 24 hrs