Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (IONIA)
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.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|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|
- ICARS [ Time Frame: baseline and 6 months ] [ Designated as safety issue: No ]
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).
- FARS [ Time Frame: 6 Months ] [ Designated as safety issue: No ]Friedreich's Ataxia Rating Scale
- ADL of FARS [ Time Frame: 6 Months ] [ Designated as safety issue: No ]ADL=Activities of Daily Living
- FACT [ Time Frame: 6 Months ] [ Designated as safety issue: No ]Friedreich's Ataxia Composite Test
|Study Start Date:||December 2007|
|Study Completion Date:||April 2009|
|Primary Completion Date:||April 2009 (Final data collection date for primary outcome measure)|
mid dose Idebenone
Patients ≤ 45 kg/99 lbs: idebenone 450 mg/day; Patients > 45 kg/99 lbs: idebenone 900 mg/day
high dose Idebenone
Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day
|Placebo Comparator: 3||
Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.
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.
|United States, California|
|David Geffen School of Medicine, UCLA|
|Los Angeles, California, United States, 90095-1769|
|United States, Pennsylvania|
|The Children's Hopsital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|Principal Investigator:||Susan Perlman, MD||University of California, Los Angeles|
|Principal Investigator:||David Lynch, MD||Children's Hospital of Philadelphia|