Efficacy Study of Epoetin Alfa in Friedreich Ataxia
Status:
Completed
Trial end date:
2015-06-01
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 2
Details
Lead Sponsor:
Federico II University
Collaborators:
Associazione Italiana per la lotta alle Sindromi Atassiche (AISA) Friedreich's Ataxia Research Alliance