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FRDA Investigator Initiated Study (IIS) With Elamipretide

NCT05168774 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2025-12-12

Study results available
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Summary

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

Conditions

  • Friedreich Ataxia

Interventions

DRUG

Elamipretide

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.

Sponsors & Collaborators

Principal Investigators

  • David Lynch, MD, PhD · Children's Hospital of Philadelphia

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-03-03
Primary Completion
2024-06-25
Completion
2024-07-25
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

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 NCT05168774 on ClinicalTrials.gov