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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00747487
First received: September 4, 2008
Last updated: May 24, 2013
Last verified: May 2013
  Purpose

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 Intervention Phase
Leber's Hereditary Optic Neuropathy
Drug: Idebenone
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:


Further study details as provided by Santhera Pharmaceuticals:

Primary Outcome Measures:
  • Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in the patient's best logMAR visual acuity between baseline and week 24 [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
  • Change in scotoma area in both eyes [ Time Frame: Day -1, Week 4, Week 12, Week 24 ] [ Designated as safety issue: No ]
  • Change in optic nerve fibre layer thickness in both eyes [ Time Frame: Day -1, Week 4, Week 12, Week 24 ] [ Designated as safety issue: No ]
  • Colour contrast sensitivity in both eyes (in a subset of patients) [ Time Frame: Day -1, Week 4, Week 12, Week 24 ] [ Designated as safety issue: No ]
  • logMAR visual acuity as a continuous variable in both eyes [ Time Frame: Screening, Day -1, Week 4, Week 12, Week 24, Week 28 ] [ Designated as safety issue: No ]
  • Clinical Global Impression of Change [ Time Frame: Week 4, Week 12 and Week 24 ] [ Designated as safety issue: No ]
  • Change in Health-Related Quality of Life (HRQOL) [ Time Frame: Day -1, Week 4, Week 12, Week 24 ] [ Designated as safety issue: No ]
  • Change in self-reported general energy levels [ Time Frame: Day -1, Week 4, Week 12, Week 24, Week 28 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Plasma levels of idebenone matched to measures of efficacy and safety [ Time Frame: 24 weeks ] [ Designated as safety issue: Yes ]
  • • 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 ] [ Designated as safety issue: No ]
  • • Count of eyes/ patients for which the visual acuity improves between baseline and week 24 [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]

Enrollment: 85
Study Start Date: November 2007
Study Completion Date: February 2010
Primary Completion Date: February 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
Idebenone
Drug: Idebenone
Idebenone 900 mg/day
Placebo Comparator: 2
Placebo
Drug: Placebo
Placebo

Detailed Description:

The study involves 6 clinic visits.

  Eligibility

Ages Eligible for Study:   14 Years to 65 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00747487

Locations
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
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

Additional Information:
Publications:
Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00747487     History of Changes
Other Study ID Numbers: SNT-II-003
Study First Received: September 4, 2008
Last Updated: May 24, 2013
Health Authority: Germany: Federal Institute for Drugs and Medical Devices
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Canada: Health Canada

Keywords provided by Santhera Pharmaceuticals:
Leber
LHON
Leber's Hereditary Optic Neuropathy
Idebenone

Additional relevant MeSH terms:
Optic Atrophy, Hereditary, Leber
Optic Nerve Diseases
Peripheral Nervous System Diseases
Cranial Nerve Diseases
Eye Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolic Diseases
Mitochondrial Diseases
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Diseases
Optic Atrophies, Hereditary
Optic Atrophy
Idebenone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs
Protective Agents

ClinicalTrials.gov processed this record on November 27, 2014