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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief 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 Summary: 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.
Sponsor: ApoPharma

Current Primary Outcome: The patient's tolerance of treatment will be determined, as assessed by the occurrence of adverse events [ Time Frame: 6 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome: 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 ]

Original Secondary Outcome: Same as current

Information By: ApoPharma

Dates:
Date Received: September 13, 2007
Date Started: April 2008
Date Completion:
Last Updated: May 31, 2010
Last Verified: May 2010