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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 52 weeks
Awards & highlights
ELViS-FA Trial Summary
This trial is testing a drug to see if it is safe and effective in treating vision loss in people with Friedreich Ataxia.
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.Check my eligibility
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.See study design
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.
ELViS-FA Trial 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.
ELViS-FA Trial 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
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change in High Contrast Visual Acuity
Secondary outcome measures
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 pain
60%
Injection site erythema
40%
Drug eruption
40%
Injection site induration
40%
Injection site urticaria
40%
Headache
40%
Nausea
20%
Echocardiogram abnormal
20%
Pain in extremity
20%
Gastroenteritis
20%
Aphthous ulcer
20%
Eczema
20%
Feeling cold
20%
Vessel puncture site bruise
20%
Injection site cellulitis
20%
Procedural vomiting
20%
Myalgia
20%
Anxiety
20%
Initial insomnia
20%
Urticaria
20%
Vaccination site rash
20%
Eosinophil count increased
20%
Palpitations
20%
Leukopenia
20%
Viral upper respiratory tract infection
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part 2 (OLE): Elamipretide (Sequence AB)
Part 1: Placebo
Part 1: Elamipretide
Part 2 (OLE): Elamipretide (Sequence BA)
ELViS-FA 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
~130
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
709 Previous Clinical Trials
8,587,699 Total Patients Enrolled
9 Trials studying Friedreich Ataxia
2,550 Patients Enrolled for Friedreich Ataxia
Friedreich's Ataxia Research AllianceOTHER
16 Previous Clinical Trials
5,930 Total Patients Enrolled
16 Trials studying Friedreich Ataxia
5,930 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.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What is the maximum capacity of participants in this clinical trial?
"This medical research is no longer recruiting participants. Initially posted on March 3rd of 2022, the trial was last updated on July 27th of 2022. Nevertheless, there are currently 46 studies for Friedreich Ataxia and 2 trials concerning Elamipectide that are actively enlisting individuals."
Answered by AI
Are recruitment efforts ongoing for this clinical experiment?
"The clinical trial posted on March 3rd 2022 is not currently taking applicants. It was last updated in July 27th 2022. Despite this, there are 48 other trials that require patient enrolment at the present time."
Answered by AI
Is this research groundbreaking in its methodology?
"Presently, two ongoing trials of the drug Elamipretide are taking place in 14 cities and 10 countries. The original study, sponsored by Stealth BioTherapeutics Inc., commenced back in 2022 with 18 participants and successfully completed Phase 1 & 2 of its clinical trial process. Since then, 17 additional studies have been concluded."
Answered by AI
Are there additional investigations centered around Elamipretide?
"At this moment, two clinical trials are actively researching the effectiveness of elamipretide. Of these, one is in its final stage (phase 3). Most of the studies take place at a single location in Portland; however there are 32 other research sites taking part as well."
Answered by AI
Who else is applying?
What state do they live in?
Pennsylvania
How old are they?
18 - 65
What site did they apply to?
Children's Hospital of Philadelphia - Neurology
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
How responsive is this trial?
Typically responds via
Email
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