Efficacy of Epoetin Alfa in Patients With Friedreich's Ataxia
Status:
Completed
Trial end date:
2009-06-01
Target enrollment:
Participant gender:
Summary
Friedreich's ataxia is a rare genetic disorder characterized by severe neurological
disability and cardiomyopathy. Friedreich's ataxia is the consequence of frataxin deficiency.
Although several drugs have been proposed, there is no available treatment. It was recently
demonstrated that erythropoietin can increase the intracellular levels of frataxin in an
in-vitro model.
The present project is aimed at testing the possible therapeutic approach of erythropoietin,
which is an already available and commercialized drug. The investigators will perform both
in-vitro and in-vivo tests, in order to asses its efficacy and safety in patients. The
results will be useful to plan further clinical trials.