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

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).

Conditions

• Friedreich's Ataxia

Interventions

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

Sponsors & Collaborators

• ApoPharma

  lead INDUSTRY

Principal Investigators

• Massimo Pandolfo, M.D. · Hospital Erasme, Brussels, Belgium

• Arnold Munnich, M.D. · Hospital Necker-Enfants Malades, Paris, France

• Franco Taroni · Fondazione IRCCS Istituto Neurologico "C. Besta"

• Martin Delatycki · Murdoch Children's Research Institute, Vicotria, Australia

• Javier Arpa · La Fundaction Para la Investigacion Biomedica, Madrid, Spain

• Mark Tarnopolsky, MD · McMaster University

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Model

PARALLEL

Eligibility

Min Age

7 Years

Max Age

35 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2008-04-30

Primary Completion

2009-07-31

Completion

2009-07-31

Countries

• Australia
• Belgium
• Canada
• France
• Italy
• Spain

Study Locations