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

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ClinicalTrials.gov Identifier: NCT00747487
Recruitment Status : Completed
First Posted : September 5, 2008
Last Update Posted : May 27, 2013
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals

Study Description
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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
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Study Type : Interventional  (Clinical Trial)
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 : November 2007
Actual Primary Completion Date : February 2010
Actual Study Completion Date : February 2010

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 1
Idebenone
 Drug: Idebenone
Idebenone 900 mg/day
Placebo Comparator: 2
Placebo
 Drug: Placebo
Placebo


Outcome Measures
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Primary Outcome Measures :
  1. Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye [ Time Frame: 24 weeks ]

Secondary Outcome Measures :
  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 ]

Eligibility Criteria
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Ages Eligible for Study:   14 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00747487


Locations
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Canada, Quebec
Unité de recherche clinique Ophtalmologie- Hopital Notre-Dame
Montreal, Quebec, Canada, H2L 4M1
Germany
Klinikum der Universität München - Grosshadern, Neurologische Klinik und Poliklinik
Munich, Germany, 81377
United Kingdom
Clinical Research Facility, 4th Floor Leazes Wing, Royal Victoria Infirmary
Newcastle Upon Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
Santhera Pharmaceuticals
Investigators
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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
More Information
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Additional Information:

Publications of Results:

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Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00747487    
Other Study ID Numbers: SNT-II-003
First Posted: September 5, 2008    Key Record Dates
Last Update Posted: May 27, 2013
Last Verified: May 2013
Keywords provided by Santhera Pharmaceuticals:
Leber
LHON
Leber's Hereditary Optic Neuropathy
Idebenone
Additional relevant MeSH terms:
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Peripheral Nervous System Diseases
Optic Nerve Diseases
Optic Atrophy, Hereditary, Leber
Neuromuscular Diseases
Nervous System Diseases
Cranial Nerve Diseases
Eye Diseases
Optic Atrophies, Hereditary
Optic Atrophy
Heredodegenerative Disorders, Nervous System
 Neurodegenerative Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Idebenone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs