The primary objective of this study is to demonstrate the safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA). The secondary objective is to evaluate the efficacy of deferiprone for the treatment of FRDA, as assessed by a 9-Hole Peg Test (9HPT), Timed 25-Foot Walk (T25FW), Low-Contrast Letter Acuity test (LCLA), International Cooperative Ataxia Rating Scale (ICARS), and Friedreich's Ataxia Rating Scale (FARS). The tertiary objectives are to evaluate the effect of deferiprone on: 1. cardiac function as measured by changes in Left Ventricular Shortening Fraction (LVSF), Left Ventricular Ejection Fraction (LVEF) and Left Ventricular (LV) mass using echocardiogram (ECHO), 2. quality of life using quality-of-life surveys, and 3. functional status using Activities of Daily Living (ADL).
This will be a multi-centre, double-blind, randomized, placebo-controlled clinical trial. A total of 80 patients with Friedreich's ataxia will be enrolled. Eligible patients will receive deferiprone oral solution or placebo at a total daily dose of 20 mg/kg/day, 40 mg/kg/day or 60 mg/kg/day, divided into two-daily doses for 6 months.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
80
Same dose and frequency as treatment
100 mg/mL
100 mg/mL
Same dosage and frequency as study drug
100 mg/mL
Murdoch Children's Research Institute
Victoria, Australia
Hospital Erasme
Brussels, Belgium
McMaster University
Hamilton, Ontario, Canada
Hospital Necker-Enfants Malades
Paris, France
Fondazione IRCCS Istituto Neurologico "C. Besta"
Milan, Italy
La Fundacion Para la Investigacion Biomedica
Madrid, Spain
The patient's tolerance of treatment will be determined, as assessed by the occurrence of adverse events
Time frame: 6 months
The efficacy endpoints will be change in the score for 9-Hole Peg Test (9HPT), Timed 25-Foot Walk (T25FW), Low-Contrast Letter Acuity test (LCLA), International Cooperative Ataxia Rating Scale (ICARS), and Friedreich's Ataxia Rating Scale (FARS).
Time frame: 6 months
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