A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients

Last updated: May 19, 2016
Sponsor: Santhera Pharmaceuticals
Overall Status: Completed

Phase

3

Condition

Friedreich's Ataxia

Dyskinesias

Spinocerebellar Disorders

Treatment

N/A

Clinical Study ID

NCT00905268
SNT-III-001
  • Ages > 8
  • All Genders

Study Summary

The purpose of this trial is to study the efficacy, safety and tolerability of idebenone in 12 months of treatment in children and adults with Friedreich's Ataxia. This is a randomised placebo-controlled double-blind trial conducted in Europe. Efficacy outcomes include measures of neurological impairment and function, and measures of the heart.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Documented diagnosis of FRDA with confirmed FRDA mutations

  • Patients 8 years of age or older at baseline

  • Patients with body weight ≥ 25kg

  • Patients who in the opinion of the investigator are able to comply with therequirements of the study, including swallowing the medication

  • Negative urine pregnancy test at screening and at baseline (women of childbearingpotential)

Exclusion

Exclusion Criteria:

  • Treatment with idebenone or Coenzyme Q10 within the past 1 month

  • Pregnancy and/or breast-feeding

  • Clinically significant abnormalities of clinical haematology or biochemistryincluding, but not limited to, elevations greater than 1.5 times the upper limit ofnormal of SGOT, SGPT, or creatinine

  • Past or present history of abuse of drugs or alcohol

Study Design

Total Participants: 232
Study Start date:
April 01, 2006
Estimated Completion Date:
January 31, 2010

Study Description

Idebenone, a short-chain analogue of Co-enzyme Q10 (CoQ10), has the potential to moderate underlying causes of Friedreich's Ataxia through its antioxidant activity and its role as an electron carrier in the respiratory chain promoting mitochondrial ATP production.

The current 12-month placebo-controlled treatment study in 232 patients aims to confirm the positive effect of idebenone on neurological function, as for instance observed in the recent 48-patient, 6-month NICOSIA study in children, using the International Cooperative Ataxia Rating Scale (ICARS) and the newly developed Friedreich's Ataxia Rating Scale (FARS).

In addition, the study aims to confirm the positive effect on cardiomyopathy associated with FRDA observed in several small studies. Cardiac anatomy and function will be assessed using echocardiography, tissue Doppler imaging and cardiac MRI methods in patients with FRDA cardiomyopathy. In addition exercise capacity, measured as peak workload, will be assessed in patients able to comply with a modified exercise protocol.

Connect with a study center

  • Universitätsklinik Innsbruck

    Innsbruck, 6020
    Austria

    Site Not Available

  • Hôpital Erasme - Université Libre de Bruxelles

    Bruxelles, 1070
    Belgium

    Site Not Available

  • Hôpital de la Salpêtrière - INSERM U679, Neurologie et Thérapeutique expérimentale

    Paris, 75651
    France

    Site Not Available

  • HELIOS Klinikum BerlinBuch

    Berlin, 13125
    Germany

    Site Not Available

  • Neurologische Universitätsklinik und Poliklinik- Universitätsklinikum Bonn

    Bonn, 53105
    Germany

    Site Not Available

  • Klinik II, Neuropädiatrie u.Muskelerkrankungen- Universitätsklinik Freiburg

    Freiburg, 79106
    Germany

    Site Not Available

  • Zentrum für Neurologische Medizin

    Göttingen, 37073
    Germany

    Site Not Available

  • Zentrum für Neurologische Medizin

    Göttingen, 37073
    Germany

    Site Not Available

  • UKE Hamburg Neuropädiatrie-Zentum für Frauen, Kinder und Jugendmedizin

    Hamburg, 20246
    Germany

    Site Not Available

  • Neurologische Klinik- klinikum Grosshadern

    München, 81377
    Germany

    Site Not Available

  • Neurologische Klinik- klinikum Grosshadern

    München, 81377
    Germany

    Site Not Available

  • Neurologische Universitätsklinik und Poliklinik

    Tübingen, 72076
    Germany

    Site Not Available

  • Neurologische Universitätsklinik und Poliklinik

    Tübingen, 72076
    Germany

    Site Not Available

  • University Medical Center Groningen

    Groningen, 9700 RB
    Netherlands

    Site Not Available

  • National Hospital for Neurology & Neurosurgery

    London, WC1N 3BG
    United Kingdom

    Site Not Available

  • University of Newcastle upon Tyne -Mitochondrial Research Group

    Newcastle, NE2 4HH
    United Kingdom

    Site Not Available

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