This study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.
The study involves 6 clinic visits.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
68
Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients \> 45 kg/99 lbs: idebenone 2250 mg/day
David Geffen School of Medicine, UCLA
Los Angeles, California, United States
The Children's Hopsital of Philadelphia
Philadelphia, Pennsylvania, United States
Change in ICARS
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. A total score of 0 points represents the best possible score, and a value of 100 points is the worst possible score. Therefore, a negative change in ICARS score represents an improvement. A higher score also indicates more disability.
Time frame: baseline and 12 months
FARS (Friedreich's Ataxia Rating Scale)
FARS consists of a 25 manoeuvre exam along with 4 quantitative performance measures. The neurological exam covers bulbar function, upper limb coordination, lower limb coordination, peripheral nervous system function, deep tendon reflexes, stability and gait. The use of FARS has been recently validated as a sensitive scale for this population. The FARS consists of three subscales, comprising a general score for ataxia, a score for activities of daily living (ADL) and a neurological examination. The scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability.
Time frame: baseline and 12 Months
Nature of Adverse Events
Time frame: 12 Months
Frequency/Number of Mild, Moderate, and Severe Adverse Events
Time frame: 12 months
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