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Mitochondrial Peptide

Elamipretide for Friedreich Ataxia Vision Loss (ELViS-FA Trial)

Phase 1 & 2
Waitlist Available
Led By David Lynch, MD, PhD
Research Sponsored by Children's Hospital of Philadelphia
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Ejection Fraction (EF) less than 50% at last evaluation (within 1 year before screening), with a history consistent with cardiomyopathy from FRDA, and VA 20/25- 20/40
Disease onset before 18 years of age
Must not have
Presence of severe renal disease (eGFR <30 mL/min) or hepatic disease [aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >2x the upper limit of normal] as evidenced by laboratory results at Screening
Current use of Metformin
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 52 weeks
Awards & highlights

Summary

This trial is testing Elamipretide, a drug that may help improve vision in patients with Friedreich Ataxia (FRDA). Elamipretide has been studied for its protective effects on eye cells. The study will compare high and low doses of the drug to see if it improves vision.

Who is the study for?
This trial is for individuals over 16 with Friedreich Ataxia (FRDA) causing vision loss and/or cardiomyopathy. Participants must have stable medication use for 30 days prior, visual acuity worse than 20/40 not correctable by glasses, and an ejection fraction less than 50%. They should agree to contraception use and not be pregnant or planning pregnancy. Genetic confirmation of FRDA is required.
What is being tested?
The study tests Elamipretide's safety and effectiveness in treating vision loss associated with FRDA. It aims to understand how well the drug can be tolerated by patients and its impact on their condition.
What are the potential side effects?
While specific side effects of Elamipretide are not listed here, common ones may include reactions at the injection site, fatigue, gastrointestinal issues like nausea or diarrhea, headache, muscle pain or weakness.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My heart's pumping ability is reduced, and I have vision issues but can see fairly well.
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My condition started before I turned 18.
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I am older than 16 years.
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My Friedreich's ataxia is confirmed through genetic testing.
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My condition started before I turned 18.
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I am older than 16 years.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I do not have severe kidney or liver disease.
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I am currently taking Metformin.
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I have an active HIV or Hepatitis B/C infection.
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I do not have any active eye nerve issues or heart disease caused by vitamin deficiencies.
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I am currently taking medication that weakens my immune system.
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I have an irregular heartbeat that is not under control.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to 52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to 52 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change in High Contrast Visual Acuity
Secondary study objectives
Change Cardiac Stroke Volume
Change in Cardiac Fibrosis
Change in Cardiac Strain
+4 more

Side effects data

From 2021 Phase 2 & 3 trial • 12 Patients • NCT03098797
100%
Dizziness
60%
Injection site pruritus
60%
Injection site erythema
60%
Injection site pain
40%
Headache
40%
Injection site urticaria
40%
Injection site induration
40%
Nausea
40%
Drug eruption
20%
Gastroenteritis
20%
Viral upper respiratory tract infection
20%
Pain in extremity
20%
Aphthous ulcer
20%
Eczema
20%
Leukopenia
20%
Palpitations
20%
Feeling cold
20%
Vessel puncture site bruise
20%
Injection site cellulitis
20%
Procedural vomiting
20%
Echocardiogram abnormal
20%
Myalgia
20%
Anxiety
20%
Initial insomnia
20%
Urticaria
20%
Vaccination site rash
20%
Eosinophil count increased
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part 2 (OLE): Elamipretide (Sequence AB)
Part 2 (OLE): Elamipretide (Sequence BA)
Part 1: Elamipretide
Part 1: Placebo

Trial Design

2Treatment groups
Experimental Treatment
Group I: Low Dose (20-30mg)Experimental Treatment1 Intervention
Subjects will receive daily subcutaneous (SC) dosing of Elamipretide (20-30 mg) for 52 weeks
Group II: High Dose (40-60 mg)Experimental Treatment1 Intervention
Subjects will receive daily subcutaneous (SC) dosing of Elamipretide (40-60 mg) for 52 weeks
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Elamipretide
2016
Completed Phase 3
~150

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Elamipretide works by stabilizing cardiolipin, a key lipid in the inner mitochondrial membrane, thereby improving mitochondrial function and energy production. This is crucial for Friedreich Ataxia patients, as the disease is characterized by mitochondrial dysfunction leading to impaired energy production and oxidative stress. By enhancing mitochondrial stability and function, treatments like Elamipretide aim to mitigate these core issues, potentially slowing disease progression and improving patient outcomes.

Find a Location

Who is running the clinical trial?

Stealth BioTherapeutics Inc.Industry Sponsor
28 Previous Clinical Trials
2,230 Total Patients Enrolled
Children's Hospital of PhiladelphiaLead Sponsor
721 Previous Clinical Trials
8,604,307 Total Patients Enrolled
9 Trials studying Friedreich Ataxia
2,550 Patients Enrolled for Friedreich Ataxia
Friedreich's Ataxia Research AllianceOTHER
18 Previous Clinical Trials
6,240 Total Patients Enrolled
18 Trials studying Friedreich Ataxia
6,240 Patients Enrolled for Friedreich Ataxia

Media Library

Elamipretide (Mitochondrial Peptide) Clinical Trial Eligibility Overview. Trial Name: NCT05168774 — Phase 1 & 2
Friedreich Ataxia Research Study Groups: Low Dose (20-30mg), High Dose (40-60 mg)
Friedreich Ataxia Clinical Trial 2023: Elamipretide Highlights & Side Effects. Trial Name: NCT05168774 — Phase 1 & 2
Elamipretide (Mitochondrial Peptide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05168774 — Phase 1 & 2
~6 spots leftby Sep 2025
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