PROTI-Idebenone in FRDA, Patient reported outcome study, SNT-III-004

• Research type

  Research Study

• Full title

  A Phase IIIb, Double-Blind, Randomised, Placebo-controlled Study of Patient Reported Outcomes in Friedreich's Ataxia Patients after withdrawal from Treatment with Idebenone

• IRAS ID

  73095

• Sponsor organisation

  Santhera Pharm.(Switzerland) Ltd

• Eudract number

  2010-023388-16

• Clinicaltrials.gov Identifier

  01303406

• Research summary

  This study looks at patient reported symptoms after interupting or stopping treatment with idebenone. Patients who are currently participating in an open-label extension study (MICONOS Extension Study - MES) are being invited to take part in this study. These patients are currently taking idebenone at a daily dose of 2,250mg and patients at this London site (all patients greater than 45kg at this aite)are still due to attend the centre for visits after 18 months treatment(visit 6)starting from end of May 2011, and after 2 years treatment (Visit 7 - end of study)starting November 2011. At these timepoints patients will undergo the assessments scheduled for the MES and if they agree to participate they will be randomly assigned in a blinded fashion to either continue treatment with idebenone at the same dose or to receive placebo for a 2 month period. Patients may have the opportunity to take place twice in this PROTI study (after Visit 6 and /or Visit 7 of MES) and will be allocated to different treatment groups on each occasion (i.e. idebenone or placebo) but still in a blinded fashion. At the start of each study period patients will undergo the assessments already schedulled in the MES and patient will also be asked to complete the Modified Fatigue Impact Scale, Patient Status questionnaire, speech assessments and neurological assessments (if these are not already scheduled for MES i.e. after visit 6). At the end of the 2 month treatment period these assessments will be repeated and patients will be asked to complete a change questionnaire and be asked to assess which treatment they consider they were receiving since the start of the PROTI study. Patient will be asked to keep a daily diary during the 2 month study period recording symptoms of fatigue and activities of daily living.

• REC name

  Wales REC 3

• REC reference

  11/WA/0087

• Date of REC Opinion

  7 Jun 2011

• REC opinion

  Further Information Favourable Opinion