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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
No Placebo-Only Group
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 belowSelect...
My heart's pumping ability is reduced, and I have vision issues but can see fairly well.
Select...
My condition started before I turned 18.
Select...
I am older than 16 years.
Select...
My Friedreich's ataxia is confirmed through genetic testing.
Select...
My condition started before I turned 18.
Select...
I am older than 16 years.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I do not have severe kidney or liver disease.
Select...
I am currently taking Metformin.
Select...
I have an active HIV or Hepatitis B/C infection.
Select...
I do not have any active eye nerve issues or heart disease caused by vitamin deficiencies.
Select...
I am currently taking medication that weakens my immune system.
Select...
I have an irregular heartbeat that is not under control.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline to 52 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to 52 weeks
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 moreSide effects data
From 2021 Phase 2 & 3 trial • 12 Patients • NCT03098797100%
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
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
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
726 Previous Clinical Trials
8,470,230 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
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have very low white blood cell count, low hemoglobin, or very high or very low platelet count based on a blood test.My medications and supplements have been the same for the last 30 days.I am currently taking Metformin.I do not have severe kidney or liver disease.My vision is worse than 20/40 due to FRDA and cannot be corrected.My heart's pumping ability is reduced, and I have vision issues but can see fairly well.I have an active HIV or Hepatitis B/C infection.My condition started before I turned 18.I do not have any active eye nerve issues or heart disease caused by vitamin deficiencies.If you are a woman, you are not pregnant, breastfeeding, or planning to get pregnant during the study or within 30 days after the study ends. If you could become pregnant, you need to have a negative pregnancy test before starting the study.My medications and supplements have been the same for the last 30 days.I am currently taking medication that weakens my immune system.You have a history of using drugs or alcohol in a harmful way.I am older than 16 years.Your heart's pumping ability is less than 35% based on the latest heart ultrasound.This criterion does not apply to me.My Friedreich's ataxia is confirmed through genetic testing.I have an irregular heartbeat that is not under control.My condition started before I turned 18.I am older than 16 years.
Research Study Groups:
This trial has the following groups:- Group 1: Low Dose (20-30mg)
- Group 2: High Dose (40-60 mg)
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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