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FRDA Investigator Initiated Study (IIS) With Elamipretide — ELViS-FA  

Friedreich Ataxia Clinical Trial

Official title:
A Pilot Investigator Initiated Study to Evaluate the Safety, Tolerability and Efficacy of Elamipretide in the Treatment of Advanced Symptoms of Friedreich Ataxia (FRDA)

Clinical Trial Summary

To evaluate the safety, tolerability, and activity of Elamipretide in treating vision loss in Friedreich Ataxia (FRDA).

Clinical Trial Description

To evaluate the effect of high dose (40-60mg) versus low dose (20-30mg) Elamipretide on high contrast visual acuity in FRDA compared to baseline at 52 weeks with the option to extend for an additional 52 weeks if there are objective signs of clinical improvement on primary or secondary endpoints. The interim analysis will be based on data from a 36-week visit. For subjects worse than 20/800 at study start, they will be followed using low vision alternatives only. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05168774
Study type Interventional
Source Children's Hospital of Philadelphia
Contact
Status Active, not recruiting
Phase Phase 1/Phase 2
Start date March 3, 2022
Completion date December 31, 2024
View Details
See also
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