Effect of Pioglitazone Administered to Patients With Friedreich's Ataxia: Proof of Concept
Status:
Completed
Trial end date:
2013-03-01
Target enrollment:
Participant gender:
Summary
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.