Clinical Trial: A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: An Open-label, Single Treatment, Safety and Efficacy, Long-term Study of Deferiprone in Subjects With Friedreich's Ataxia
Brief 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:
- cardiac function,
- quality of life, and
- functional status.
Detailed Summary: 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.
Sponsor: ApoPharma
Current Primary Outcome: The patient's long-term tolerance of treatment will be assessed by the occurence of adverse events. [ Time Frame: 52 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome: The long-term efficacy of deferiprone will be assessed. Efficacy measures include the 9HPT, T25FW, LCLA, ICARS and FARS. [ Time Frame: 52 weeks ]
Original Secondary Outcome: Same as current
Information By: ApoPharma
Dates:
Date Received: May 8, 2009
Date Started: June 2009
Date Completion:
Last Updated: June 24, 2011
Last Verified: June 2011
