Status:

COMPLETED

Safety and Efficacy of Etravirine in Friedreich Ataxia Patients

Lead Sponsor:

IRCCS Eugenio Medea

Collaborating Sponsors:

University of Rome Tor Vergata

Conditions:

Friedreich Ataxia

Eligibility:

All Genders

10-40 years

Phase:

PHASE2

Brief Summary

A drug repositioning effort provided evidence supporting the possible use of Etravirine, a drug approved for the treatment of HIV infections in patients starting from 2 years of age, as a treatment fo...

Detailed Description

Friedreich ataxia is a rare progressive neurodegenerative disease presenting in childhood or late adolescence for which no treatment is currently available. The disease is caused by the defective synt...

Eligibility Criteria

Inclusion

  • Molecularly defined FA diagnosis (at least one expanded FXN allele),
  • ability to complete maximal exercise testing (being able to ride a recumbent exercise ergometer at 60 rpm with no added resistance for at least 3 minutes),
  • willingness to participate, as shown by the signed consent form.

Exclusion

  • Known intolerance to any of the components of the drug preparation.
  • Ongoing treatment with Etravirine or other experimental therapeutics under study for FA (e.g. IFNy, erythropoietin, nicotinamide, etc.).
  • Previous treatment, if any, should have been stopped at least 4 weeks before study entry.
  • Severe medical condition likely to interfere with drug absorbance and distribution (liver or kidney failure, severe metabolic unbalance), significant cardiac disease

Key Trial Info

Start Date :

September 17 2020

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

January 17 2023

Estimated Enrollment :

30 Patients enrolled

Trial Details

Trial ID

NCT04273165

Start Date

September 17 2020

End Date

January 17 2023

Last Update

December 4 2024

Active Locations (1)

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IRCCS Medea Scientific Institute, Pieve di Soligo Research Centre

Pieve di Soligo, TV, Italy, 31953