CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 20 enrolled
Drug / intervention
Elamipretidedrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT05168774
NCT05168774Phase 2Completed
CompareCT.gov

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

Children's Hospital of Philadelphia·interventional·Posted Dec 23, 2021·Updated Dec 12, 2025

In Brief

A Phase 2 clinical trial evaluating Elamipretide for Friedreich Ataxia. Completed, enrolled 20 participants across 1 site.

Detailed Summary

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

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsFriedreich Ataxia
CountriesUnited States
CollaboratorsFriedreich's Ataxia Research Alliance, Stealth BioTherapeutics Inc.

Timeline

Phase 2CompletedFinished
20222023202420252026
First PostedDec 23, 2021
Enrollment StartMar 3, 2022
Primary CompletionJun 25, 2024
Study CompletionJul 25, 2024
TodayJul 2, 2026
Enrollment to primary: 2.3 yearsPosted 4.5 years ago

Interventions

Elamipretidedrug

Elamipretide is a tetra peptide with limited blood brain barrier penetration being developed for use in a variety of mitochondrial disorders, including FRDA, mitochondrial myopathy and Barth Syndrome.