NCT00897221

A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia

An Open-label, Single Treatment, Safety and Efficacy, Long-term Study of Deferiprone in Subjects With Friedreich's Ataxia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 36 (actual)
• Sponsor: ApoPharma · Industry
• Sex: All
• Age: 7 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to evaluate the long-term safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA). The secondary objective is to evaluate the long-term efficacy of deferiprone for the treatment of FRDA. The tertiary objectives are to evaluate the effect of deferiprone on: 1. cardiac function, 2. quality of life, and 3. functional status.

Detailed description

This is a multi-centre, open-label, non-randomized, single treatment, safety and efficacy study. All subjects who completed the LA29-0207 study are eligible for participation. Participants will receive deferiprone oral solution at the same dose (20 or 40 mg/kg/day) that they were assigned for LA29-0207. The duration of treatment will be 52 weeks.

Conditions

• Friedreich's Ataxia

Interventions

Type	Name	Description
DRUG	Deferiprone oral solution 100mg/mL	Deferiprone oral solution (20 mg/kg/day)
DRUG	Deferiprone oral solution 100 mg/mL	Deferiprone oral solution(40mg/kg/day)

Timeline

• Start date: 2009-06-01
• Primary completion: 2011-03-01
• Completion: 2011-03-01
• First posted: 2009-05-12
• Last updated: 2011-06-27

Locations

4 sites across 4 countries: Belgium, France, Italy, Spain