Principal Investigator:    Alessandro Filla
Grant Title:    A double-blind, randomized, placebo-controlled, clinical trial to test the efficacy of Epoetin alfa on physical performance of Friedreich Ataxia patients.
Status:    Closed
Start Date:    11/15/2011
Duration:    12 months
Description:    Friedreich’s ataxia (FRDA) is a rare genetic disorder characterised 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. Four trials recently demonstrated that erythropoietin can increase the intracellular levels of frataxin. The present project is aimed at testing a long term therapeutic approach using erythropoietin, which is an already available and commercialised drug. The study will test the effect of erythropoietin on exercise capacity, which is reduced in patients with FRDA. Additional objectives of the study will be the drug’s safety and tolerability, and its effect on frataxin, blood vessel reactivity, heart functional indexes, and disease progression.