Clinical Trial: Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Phase III Double-Blind, Randomized, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients
Brief Summary: This study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.
Detailed Summary: The study involves 6 clinic visits and upon completion the possibility to join a 12 months extension study where all patients will receive high dose Idebenone.
Sponsor: Santhera Pharmaceuticals
Current Primary Outcome: ICARS [ Time Frame: baseline and 6 months ]
International Cooperative Ataxia Rating Scale (ICARS):
ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability.
Scores for each subscale quantify the extent of ataxia in each clinically important area. Subscale scores are summed to give a total score ranging from 0 (best) to 100 (worst).
Original Primary Outcome: ICARS [ Time Frame: 6 months ]
Current Secondary Outcome:
- FARS [ Time Frame: 6 Months ]Friedreich's Ataxia Rating Scale
- ADL of FARS [ Time Frame: 6 Months ]ADL=Activities of Daily Living
- FACT [ Time Frame: 6 Months ]Friedreich's Ataxia Composite Test
Original Secondary Outcome:
- FARS [ Time Frame: 6 Months ]
- ADL of FARS [ Time Frame: 6 Months ]
- FACT [ Time Frame: 6 Months ]
Information By: Santhera Pharmaceuticals
Dates:
Date Received: September 7, 2007
Date Started: December 2007
Date Completion:
Last Updated: February 2, 2012
Last Verified: February 2012
