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

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ClinicalTrials.gov Identifier: NCT00993967
Recruitment Status : Completed
First Posted : October 14, 2009
Last Update Posted : August 29, 2012
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals

Brief Summary:
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 or disease Intervention/treatment Phase
Freidreich's Ataxia Drug: idebenone Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 204 participants
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.
Study Start Date : June 2007
Primary Completion Date : June 2012
Study Completion Date : June 2012



Intervention Details:
    Drug: idebenone
    Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg


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

Secondary Outcome Measures :
  1. 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 ]


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
Criteria

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

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


Locations
Austria
Universitätsklinik Innsbruck
Innsbruck, Austria
Belgium
Hôpital Erasme - Univeristé Libre de Bruxelles
Bruxelles, Belgium, 1070
France
Hôpital de la Salpétrière - INSERM U679, Neurologie et Thérapeutique expérimentale
Paris, France, 75651
Germany
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
Netherlands
University medical Center Groningen
Groningen, Netherlands, 9700 RB
Sponsors and Collaborators
Santhera Pharmaceuticals
Investigators
Principal Investigator: Nick Wood, Professor Dept of Molecular Neuroscience, Institute of Neurology. The National Hospital, University College London

Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00993967     History of Changes
Other Study ID Numbers: SNT-III-001-E
First Posted: October 14, 2009    Key Record Dates
Last Update Posted: August 29, 2012
Last Verified: August 2012

Keywords provided by Santhera Pharmaceuticals:
FRDA
idebenone
FRDA disease

Additional relevant MeSH terms:
Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Dyskinesias
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
Idebenone
Ubiquinone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Micronutrients
Growth Substances