Text Size

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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00993967
First received: October 13, 2009
Last updated: August 28, 2012
Last verified: August 2012
History of Changes
  Purpose

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: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: 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:

U.S. FDA Resources

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 ] [ Designated as safety issue: Yes ]
  • Measures of safety and tolerability: haematological and biochemical laboratory parameters [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ] [ Designated as safety issue: Yes ]
  • Measures of safety and tolerability: physical examinations and vital signs [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ] [ Designated as safety issue: Yes ]
  • Measures of safety and tolerability: electrocardiograms (ECGs) [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ] [ Designated as safety issue: Yes ]

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 ] [ Designated as safety issue: No ]

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
  Eligibility

Ages Eligible for Study:   9 Years and older
Genders Eligible for Study:   Both
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: 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
  More Information

No publications provided

Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00993967     History of Changes
Other Study ID Numbers: SNT-III-001-E
Study First Received: October 13, 2009
Last Updated: August 28, 2012
Health Authority: Austria: Federal Office for Safety in Health Care
Belgium: Institutional Review Board
Belgium: Federal Agency for Medicinal Products and Health Products
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
France: Institutional Ethical Committee
Germany: Ethics Commission
Germany: Federal Institute for Drugs and Medical Devices
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Netherlands: Medical Ethics Review Committee (METC)
Netherlands: Ministry of Health, Welfare and Sport

Keywords provided by Santhera Pharmaceuticals:
FRDA
idebenone
FRDA disease

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

ClinicalTrials.gov processed this record on June 18, 2013