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Efficacy Study of Epoetin Alfa in Friedreich Ataxia

NCT01493973 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 56

Last updated 2015-08-11

No results posted yet for this study

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.

Conditions

  • Friedreich Ataxia

Interventions

DRUG

Epoetin alfa

Epoetin alfa will be administered s.c. at 1200 IU/Kg every 12 weeks

DRUG

Placebo

Placebo

Sponsors & Collaborators

  • Friedreich's Ataxia Research Alliance

    collaborator OTHER

  • Associazione Italiana per la lotta alle Sindromi Atassiche (AISA)

    collaborator UNKNOWN

  • Federico II University

    lead OTHER

Principal Investigators

  • Francesco Saccà, MD · University Federico II, Naples Italy

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-01-31
Primary Completion
2014-10-31
Completion
2015-06-30

Countries

  • Italy

Study Locations

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Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01493973 on ClinicalTrials.gov