Friedreich's Ataxia MICONOS Extension study

• Research type

  Research Study

• Full 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

• IRAS ID

  33279

• Sponsor organisation

  Santhera Pharm.(Switzerland) Ltd

• Eudract number

  2007-001646-40

• ISRCTN Number

  n/a

• Clinicaltrials.gov Identifier

  n/a

• Research summary

  This is an extension study providing open-label treatment with idebenone to patients who have completed a double-blind, dose ranging, placebo-controlled phase III study of idebenone in Friedreich's Ataxia patients (SNT-III-001). Patients who complete this 52 week study and are found to be eligible by the investigator may enter this open-label extension study. In this extension study all patients above 45kg will receive a daily dose of 2,250mg/day idebenone for up to 24 months. This study aims to gather long-term safety and tolerability data on idebenone in the Friedreich's Ataxia population by monitoring adverse events, haematological and biochemical parameters, physical examination and vital signs and ECG. The effect of longer term administration of idebenone on neurological symptoms and signs will also be explored as assessed by the International Cooperative Ataxia Rating Scale (ICARS) and the Friedreich's Ataxia Rating Scale (FARS).

• REC name

  East of England - Cambridge Central Research Ethics Committee

• REC reference

  09/H0308/177

• Date of REC Opinion

  2 Nov 2009

• REC opinion

  Further Information Favourable Opinion