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Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)

This study has been completed.
ClinicalTrials.gov Identifier:
First Posted: October 14, 2009
Last Update Posted: October 12, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Santhera Pharmaceuticals
This is an Extension study of the MICONOS main randomised placebo-controlled trial (NCT00905268), and open to those patients completing the main study. The scientific aim of this extension study is to monitor safety and tolerability of idebenone over two years in patients with Friedreich's Ataxia.

Condition Intervention Phase
Freidreich's Ataxia Drug: idebenone Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase III Open-Label, Single-Group, Extension Study to Obtain Long-Term Safety and Tolerability Data of Idebenone in the Treatment of Friedreich's Ataxia Patients.

Resource links provided by NLM:

Further study details as provided by Santhera Pharmaceuticals:

Primary Outcome Measures:
  • Measures of safety and tolerability: nature and frequency of adverse events (AEs) [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]
  • Measures of safety and tolerability: haematological and biochemical laboratory parameters [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]
  • Measures of safety and tolerability: physical examinations and vital signs [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]
  • Measures of safety and tolerability: electrocardiograms (ECGs) [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]

Secondary Outcome Measures:
  • Absolute change in The International Cooperative Ataxia Rating Scale (ICARS) and Friedreich Ataxia Rating Scale (FARS) scores [ Time Frame: Baseline, Month 12 and month 24 ]

Estimated Enrollment: 204
Study Start Date: June 2007
Study Completion Date: June 2012
Primary Completion Date: June 2012 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: idebenone
    Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg

Information from the National Library of Medicine

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Ages Eligible for Study:   9 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Completion of 52 weeks in study SNT-III-001
  • Body weight ≥ 25 kg
  • Negative urine pregnancy test
  • Eligibility to participate in the present extension study as confirmed by investigator

Exclusion Criteria:

  • Safety or tolerability issues arising during the course of SNT-III-001 which in the opinion of the investigator preclude further treatment with idebenone
  • Clinically significant abnormalities of haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of SGOT, SGPT or creatinine
  • Parallel participation in another clinical drug trial
  • Pregnancy or breast-feeding
  • Abuse of drugs or alcohol
  Contacts and Locations
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): NCT00993967

Universitätsklinik Innsbruck
Innsbruck, Austria
Hôpital Erasme - Univeristé Libre de Bruxelles
Bruxelles, Belgium, 1070
Hôpital de la Salpétrière - INSERM U679, Neurologie et Thérapeutique expérimentale
Paris, France, 75651
HELIOS Klinikum Berlin
Berlin, Germany, 13125
Neurologische Universitätsklinik und Ploklinik - Universitätsklimikum Bonn
Bonn, Germany, 53105
Klinik II, Neuropädiatrie und Muskelerkrankungen - Universitätsklinik Freiburg
Freiburg, Germany, 79106
Zentrum für Neurologische Medizin
Göttingen, Germany, 37073
UKE Hamburg Neuropädiatrie - Zentrum für Frauen, Kinder und Jugendmedizin
Hamburg, Germany, 20246
Neurologische Klinik - Klinikum Grosshadern
München, Germany, 81377
Neurologische Universitätsklinik und Poliklinik
Tübingen, Germany, 72076
University medical Center Groningen
Groningen, Netherlands, 9700 RB
Sponsors and Collaborators
Santhera Pharmaceuticals
Principal Investigator: Nick Wood, Professor Dept of Molecular Neuroscience, Institute of Neurology. The National Hospital, University College London
  More Information

Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00993967     History of Changes
Other Study ID Numbers: SNT-III-001-E
First Submitted: October 13, 2009
First Posted: October 14, 2009
Last Update Posted: October 12, 2017
Last Verified: August 2012

Keywords provided by Santhera Pharmaceuticals:
FRDA disease

Additional relevant MeSH terms:
Cerebellar Ataxia
Friedreich Ataxia
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Growth Substances