Effect of Pioglitazone Administered to Patients With Friedreich's Ataxia: Proof of Concept (ACTFRIE)
Friedreich's ataxia (FA) is a rare progressive neurological disorder affecting approximately 1/30, 000 individuals. No treatment is presently available to counteract the neurodegeneration of this extremely severe disease.
Pioglitazone, a well known PPAR gamma (peroxysome proliferators-activated receptor gamma) ligand induces the expression of many enzymes involved in the mitochondrial metabolism, including the superoxide dismutases. This agent may be therapeutic by counteracting the disabled recruitment of antioxidant enzymes in FA patients. This potential neuroprotective agent crosses the brain blood barrier in human.
Primary objective: To explore the effects of Pioglitazone on neurological function in FA patients. We expect neurological benefits taking into account the natural course of the disease.
Population: Subjects for this study will be limited to patients not older than 25 years Methodology: Prospective, randomized double-blind trial of Pioglitazone versus placebo in FA patients. Patients will be treated two years and will undergo clinical exams and testing during three days each six months at the clinical investigation centre.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
|Official Title:||Effect of Pioglitazone Administered to Patients With Friedreich's ATAXIA:Proof of Concept|
- evaluate the efficacy of Pioglitazone on the neurological function of FA patients. Success will be defined as a stabilisation or improvement on ICARS designed as no more than 2 points maximum increment on this scale in two year. [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
- tolerance of Pioglitazone [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
- efficacy of Pioglitazone on neurological function [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
- efficacy of Pioglitazone on functional handicap and quality of life [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
- effect of Pioglitazone on cardiac parameters [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
|Study Start Date:||December 2008|
|Study Completion Date:||March 2013|
|Primary Completion Date:||March 2013 (Final data collection date for primary outcome measure)|
administered orally once a day after the first visit and for a total of 2 years. Initially, Pioglitazone will be started at 15mg /day. Dosage will then increase by 15mg /d/ week up to the maximal dose of 45mg /day
Other Name: pioglitazone
Placebo Comparator: Control
a placebo administered orally once a day after the first visit and for a total of 2 years.
Other Name: Control
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