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

Leber's Hereditary Optic Neuropathy Clinical Trial

— 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00747487
• Other study ID #: SNT-II-003
• Status: Completed
• Phase: Phase 2
• First received: September 4, 2008
• Last updated: May 24, 2013
• Start date: November 2007
• Est. completion date: February 2010

Study information

• Verified date: May 2013
• Source: Santhera Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: Germany: Federal Institute for Drugs and Medical DevicesUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyCanada: Health Canada
• Study type: Interventional

Clinical Trial 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.

Description:

The study involves 6 clinic visits.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 85
• Est. completion date: February 2010
• Est. primary completion date: February 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 14 Years to 65 Years

Eligibility

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 Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Leber's Hereditary Optic Neuropathy
• Optic Atrophy, Hereditary, Leber
• Optic Nerve Diseases

Intervention

Drug:
• Idebenone
Idebenone 900 mg/day
• Placebo
Placebo

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Santhera Pharmaceuticals

Countries where clinical trial is conducted

Canada,  Germany,  United Kingdom, 

References & Publications (2)

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 — View Citation

Rudolph G, Dimitriadis K, Büchner 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 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye		24 weeks	No
Secondary	Change in the patient's best logMAR visual acuity between baseline and week 24		24 weeks	No
Secondary	Change in scotoma area in both eyes		Day -1, Week 4, Week 12, Week 24	No
Secondary	Change in optic nerve fibre layer thickness in both eyes		Day -1, Week 4, Week 12, Week 24	No
Secondary	Colour contrast sensitivity in both eyes (in a subset of patients)		Day -1, Week 4, Week 12, Week 24	No
Secondary	logMAR visual acuity as a continuous variable in both eyes		Screening, Day -1, Week 4, Week 12, Week 24, Week 28	No
Secondary	Clinical Global Impression of Change		Week 4, Week 12 and Week 24	No
Secondary	Change in Health-Related Quality of Life (HRQOL)		Day -1, Week 4, Week 12, Week 24	No
Secondary	Change in self-reported general energy levels		Day -1, Week 4, Week 12, Week 24, Week 28	No
Secondary	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	No
Secondary	Plasma levels of idebenone matched to measures of efficacy and safety		24 weeks	Yes
Secondary	• Best visual acuity at Week 24 (best eye at Week 24) compared to best visual acuity at Baseline (best eye at Baseline)		24 weeks	No
Secondary	• Count of eyes/ patients for which the visual acuity improves between baseline and week 24		24 weeks	No

External Links

•   Website for LHON patients in the U.K.