Study to Assess the Safety and Tolerability of Idebenone in the (IONIA-E)
This study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase III Open-Label, Single Group Extension Study of the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients|
- Change in ICARS [ Time Frame: baseline and 12 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 (Friedreich's Ataxia Rating Scale) [ Time Frame: baseline and 12 Months ] [ Designated as safety issue: No ]
- Nature and Frequency of Adverse Events [ Time Frame: 12 Months ] [ Designated as safety issue: Yes ]
|Study Start Date:||July 2008|
|Study Completion Date:||May 2010|
|Primary Completion Date:||May 2010 (Final data collection date for primary outcome measure)|
high dose Idebenone
Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day
The study involves 6 clinic visits.
|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|