A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension
Stealth BioTherapeutics Inc.218 participants9 Oct 2017 to 10 Feb 2020
Primary Mitochondrial Myopathy
In PepBank now:147 peptides, 6 blends and 13 other compounds97,577 study results from 17,383 studies8,473 clinical trialsUpdated
Comparing 2 records · Data version 308560
Swipe sideways to see every peptide.
| Peptide | Synthetic analog Elamipretide (SS-31) | ||
|---|---|---|---|
| Identity | |||
| Kind | Synthetic analog | Small molecule | |
| Area | Longevity and mitochondria | Longevity and mitochondria | |
| Evidence | |||
| Amount of evidence | 1,784 | 1,442 (plus 2 on natural levels, listed separately) | |
| Evidence level | Has results from studies in people | Has results from studies in people | |
| Safety | |||
| Safety results | 35 (29 in people) | 19 (12 in people) | |
| Status | |||
| Approval | |||
| Sport (WADA) | |||
| Registered trials | 30 · 3 active | 3 | |
Shared outcomes
From the regulatory data of each peptide.
The FDA label for Forzinity says: “To improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg” No central EU authorisation (EMA).
Outcome groups with study results for at least two of these peptides. Counts include only results from studies that gave the peptide. Bar length is the number of results, on one scale. The bars show how much was studied, not how large an effect was. A peptide with more studies can look stronger here without being stronger. For results, see the randomized results above.
| Outcome group | Elamipretide (SS-31) | AICAR (acadesine) |
|---|---|---|
| Receptor activity and mechanismRespiratory | 883850 | 5382294 |
PepBank is a research reference. Talk to a doctor before you use any peptide.
Its most advanced trial is in phase 3. The FDA, the EMA and the other sources we use show no approval (as of 21 Oct 2026).
| Mitochondrial functionWhole body | 4795258216 | 713536 |
|---|
| Inflammation markersWhole body | 17312548 | 1219328 |
|---|
| Heart functionCardiovascular | 120208614 | 73133822 |
|---|
| NeuroprotectionNervous system and cognition | 17814335 | 544410 |
|---|
| Kidney functionKidney and urinary | 130611410 | 38344 |
|---|
| Adverse eventsWhole body | 292621 | 12102 |
|---|
| Exercise and physical functionWhole body | 6220357 | 1414 |
|---|
| Glycemic controlMetabolic and weight | 11101 | 9857023 |
|---|
| Oxidative stressSkin and wound | 914645 | 311615 |
|---|
| Cell viability and deathWhole body | 561937 | 29623 |
|---|
| LipidsMetabolic and weight | 532 | 7133137 |
|---|
Registered trials, ranked by phase (late phases first), then posted results and enrollment. Status is the registry status. Study results come from published studies. Results posted at ClinicalTrials.gov are shown on each trial page.
Top 3 of 30 trials, ranked by phase, then posted results and enrollment: 1 ongoing · 1 completed · 1 stopped early · 2 with posted results · source ClinicalTrials.gov (opens in a new tab)
Stealth BioTherapeutics Inc.218 participants9 Oct 2017 to 10 Feb 2020
Primary Mitochondrial Myopathy
Stealth BioTherapeutics Inc.12 participants5 Jul 2017 to 11 Oct 2021
Barth Syndrome
Stealth BioTherapeutics Inc.313 participants30 May 2024 to est. Sept 2027
Age Related Macular Degeneration (ARMD)
3 trials · 1 completed · 2 stopped early · 1 with posted results · source ClinicalTrials.gov (opens in a new tab)
Merck Sharp & Dohme LLC3,080 participantsApr 2009 to Oct 2010
Coronary Artery Bypass · Myocardial Infarction · Ventricular Dysfunction, Left · Stroke · Cardiopulmonary Bypass
Advancell - Advanced In Vitro Cell Technologies, S.A.40 participantsDec 2007 to Dec 2010
Leukemia, B-Cell, Chronic
Groupe Francophone des Myelodysplasies5 participantsJun 2013 to Jun 2015
SMD
For each outcome that two or more peptides have results for: the best result of a single randomized trial, on the same measure (the same kind of number, the same unit). When a trial has several dose arms, the cell shows the highest dose arm. The trials differ in people, length and dose, so a gap between two cells is not proof that one peptide works better.
No outcome has a randomized-trial result on the same measure for two or more of these peptides yet.
The other peptides have no randomized result on the same measure, so these results cannot be set side by side.
| Outcome | Elamipretide (SS-31) | AICAR (acadesine) |
|---|---|---|
| Cardiovascular eventsCardiovascularCompared as: value in the arm that got the peptide, in % | No comparable result 2 randomized-trial results measure it in another way or another unit. | 5.1% Primary outcome Placebo: 5% Dose studied: 0.1 mg/kg per minute7 hoursn = 2,9862012 |
| Heart functionCardiovascularCompared as: value in the arm that got the peptide, in % | No comparable result 9 randomized-trial results measure it in another way or another unit. | 3% Prebypass ECG ischemia Placebo: 0% Dose studied: Low dose, amount not stated7 hn not reported1994 |
| Exercise and physical functionMusculoskeletalCompared as: difference from placebo, in m | −3.2 m 6MWT least squares mean difference Dose studied: 40 mg/d24 weeksn = 2182023 | No randomized trial result 14 results from other study designs. |
| Kidney functionKidney and urinaryCompared as: value in the arm that got the peptide, in % | −6% Change in fractional hypoxia Placebo: 47% Dose studied: 0.05 mg/kg per hourbefore and during PTRAn = 142017 | |
| MortalityWhole bodyCompared as: value in the arm that got the peptide, in % | No comparable result 1 randomized-trial result measure it in another way or another unit. | 2.7% Overall mortality Placebo: 3.4% Dose studied: 0.1 mg/kg per minute intravenously and 5 micrograms/ml in cardioplegic solution7 hoursn = 8211995 |
| Adverse eventsEyeCompared as: value in the arm that got the peptide, in % | 86% Adverse event rate Placebo: 71% Dose studied: 40 mg daily48 weeksn = 1762025 |
No comparable result
2 randomized-trial results measure it in another way or another unit.
5.1%
Primary outcome
Placebo: 5%
Dose studied: 0.1 mg/kg per minute7 hoursn = 2,9862012
No randomized trial result
38 results from other study designs.
No comparable result 4 randomized-trial results measure it in another way or another unit. |
No comparable result
9 randomized-trial results measure it in another way or another unit.
3%
Prebypass ECG ischemia
Placebo: 0%
Dose studied: Low dose, amount not stated7 hn not reported1994
−3.2 m
6MWT least squares mean difference
Dose studied: 40 mg/d24 weeksn = 2182023
No randomized trial result
14 results from other study designs.
−6%
Change in fractional hypoxia
Placebo: 47%
Dose studied: 0.05 mg/kg per hourbefore and during PTRAn = 142017
No randomized trial result
38 results from other study designs.
No comparable result
1 randomized-trial result measure it in another way or another unit.
2.7%
Overall mortality
Placebo: 3.4%
Dose studied: 0.1 mg/kg per minute intravenously and 5 micrograms/ml in cardioplegic solution7 hoursn = 8211995
86%
Adverse event rate
Placebo: 71%
Dose studied: 40 mg daily48 weeksn = 1762025
No comparable result
4 randomized-trial results measure it in another way or another unit.