NCT00530127

A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia

A Six-month Double-blind, Randomized, Placebo-controlled Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 80 (estimated)
• Sponsor: ApoPharma · Industry
• Sex: All
• Age: 7 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

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).

Detailed description

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.

Conditions

• Friedreich's Ataxia

Interventions

Type	Name	Description
DRUG	placebo	Same dose and frequency as treatment
DRUG	deferiprone	100 mg/mL
DRUG	deferiprone	100 mg/mL
DRUG	placebo	Same dosage and frequency as study drug
DRUG	deferiprone	100 mg/mL

Timeline

• Start date: 2008-04-01
• Primary completion: 2009-07-01
• Completion: 2009-07-01
• First posted: 2007-09-17
• Last updated: 2010-06-02

Locations

6 sites across 6 countries: Australia, Belgium, Canada, France, Italy, Spain