Human evidence quality
Elamipretide (SS-31): what the human research shows
Evidence overview
Research volume and reviewed human evidence are different numbers, kept apart.
Indexed publications
398
Research snapshot 2026.1.1
Human clinical publications
30
PubMed records classified as human research
Distinct human studies reviewed
51
of 54 identified
Randomized controlled studies
16
among reviewed studies
Studies with reported results
27
Registered, no results available
14
Unpublished is not negative
Study results assessed for risk of bias
100
Outcomes with a certainty assessment
3
Claims evaluated
Claims are things to investigate, never established benefits. Evidence for one outcome doesn’t carry over to another.
| Claim being evaluated | Eligible human studies | Certainty by outcome |
|---|---|---|
| Improves exercise capacity in primary mitochondrial myopathy | 4 |
|
| Improves function in Barth syndrome | 2 |
|
| Is safe for human administrationSafety | 15 |
|
Human studies
Each distinct study once, however many papers, abstracts or registrations describe it.
Elamipretide in pediatric Barth syndrome: from heart failure to school return.Case report · 1 participants · first reported 2026 · automated, not human-reviewed
Population: A 5-year-old (preschool-aged) boy with genetically confirmed Barth syndrome and advanced heart failure. At start he had severe left ventricular dysfunction and moderate-to-severe mitral regurgitation, depended on gastrostomy feeding and was listed for heart transplantation.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Left ventricular ejection fraction (LVEF): Serious concern (Structured limitations appraisal); Mitral regurgitation severity: Serious concern (Structured limitations appraisal); Ross heart failure class: Serious concern (Structured limitations appraisal); Functional capacity / exercise tolerance: Serious concern (Structured limitations appraisal); Nutritional status: Serious concern (Structured limitations appraisal); Health-related quality of life: Serious concern (Structured limitations appraisal); Return to school: Serious concern (Structured limitations appraisal); Heart transplant listing status: Serious concern (Structured limitations appraisal); Adverse events / tolerability: Serious concern (Structured limitations appraisal)
Full study record (PLS-00097)Expanded-Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series.Case series · 2 participants · first reported 2024 · automated, not human-reviewed
Population: Two patients with different progressive mitochondrial disorders with ophthalmic features: chronic progressive external ophthalmoplegia (CPEO) plus, and neuropathy, ataxia and retinitis pigmentosa (NARP) syndrome.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Symptoms and quality of life: Serious concern (Structured limitations appraisal); Tolerability: Serious concern (Structured limitations appraisal)
Full study record (PLS-00098)Expanded-access use of elamipretide in a newborn with Barth syndrome: a case report.Case report · 1 participants · first reported 2025 · automated, not human-reviewed
Population: A male infant diagnosed in utero with Barth syndrome. At birth he had a moderately dilated left ventricle with reduced ejection fraction and needed intubation and haemodynamic support.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Left ventricular ejection fraction: Serious concern (Structured limitations appraisal); Oxygen weaning and hospital discharge: Serious concern (Structured limitations appraisal)
Full study record (PLS-00099)Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial.Randomized controlled trial · 218 participants · first reported 2017 · automated, not human-reviewed
Population: Adults with genetically confirmed primary mitochondrial myopathy in MMPOWER-3. 74% had mtDNA pathogenic variants or single large-scale mtDNA deletions. The rest had nDNA variants, mostly in mtDNA replisome genes such as POLG and TWNK. Analyzed post hoc by genotype subgroup.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Six-minute walk test distance, overall trial population: Some concerns (Cochrane RoB 2); Six-minute walk test distance, prespecified nDNA and mtDNA subgroups: Some concerns (Cochrane RoB 2); Six-minute walk test change, mtDNA replisome post hoc cohort: High risk of bias (Cochrane RoB 2); Six-minute walk test change, replisome subjects with CPEO: High risk of bias (Cochrane RoB 2); Exposure-response: plasma elamipretide concentration vs 6MWT improvement, nDNA cohort: High risk of bias (Cochrane RoB 2)
Full study record (PLS-00100)Cardiac pathology in a patient with a novel pathogenic variant c.703del (p.Ile235SerfsTer4) of the TAFAZZIN gene.Case report · 1 participants · first reported 2025 · automated, not human-reviewed
Population: A full-term male infant with Barth syndrome due to a novel pathogenic TAFAZZIN c.703del variant. He presented with postnatal cardiogenic shock that required VA-ECMO and was waitlisted for cardiac transplantation.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Tolerability of elamipretide: Serious concern (Structured limitations appraisal); Cardiac transplantation and clinical course: Serious concern (Structured limitations appraisal); Explanted heart pathology: Serious concern (Structured limitations appraisal)
Full study record (PLS-00101)Elamipretide in the Management of Barth Syndrome: Current Evidence and a Case Report.Case report · 1 participants · first reported 2025 · automated, not human-reviewed
Population: A single neonate with prenatally identified Barth syndrome. The infant had severe left ventricular non-compaction cardiomyopathy and clinical heart failure, and was on the heart transplant list.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Clinical heart failure status and heart transplant listing status: Serious concern (Structured limitations appraisal)
Full study record (PLS-00102)Expanded-access use of elamipretide in a patient with membrane protein-associated neurodegeneration.Case report · 1 participants · first reported 2024 · automated, not human-reviewed
Population: A single progressively declining 17-year-old female patient with membrane protein-associated neurodegeneration (MPAN)
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Symptoms (dysarthria, dysphagia, gait): Serious concern (Structured limitations appraisal); 6-minute walk test: Serious concern (Structured limitations appraisal); 5 times sit-to-stand test: Serious concern (Structured limitations appraisal)
Full study record (PLS-00103)ReCLAIM-2: A Randomized Phase II Clinical Trial Evaluating Elamipretide in Age-related Macular Degeneration, Geographic Atrophy Growth, Visual Function, and Ellipsoid Zone Preservation.Randomized controlled trial · 176 participants · first reported 2019 · automated, not human-reviewed
Population: Patients aged 55 years or older with at least one eye with dry age-related macular degeneration and noncentral geographic atrophy.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Mean change from baseline in low-luminance best-corrected visual acuity (LL BCVA): Some concerns (Cochrane RoB 2); Change from baseline in square-root converted geographic atrophy area: Some concerns (Cochrane RoB 2); Progression of macular percentage of total ellipsoid zone attenuation/loss: Some concerns (Cochrane RoB 2); Progression of macular percentage of partial ellipsoid zone attenuation/degradation: Some concerns (Cochrane RoB 2); Proportion of patients with a gain of 10 or more letters in LL BCVA: Some concerns (Cochrane RoB 2); Incidence and severity of adverse events: Some concerns (Cochrane RoB 2)
Full study record (PLS-00104)Elamipretide Topical Ophthalmic Solution for the Treatment of Subjects with Leber Hereditary Optic Neuropathy: A Randomized Trial.Randomized controlled trial · 12 participants · first reported 2023 · automated, not human-reviewed
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Adverse events: Some concerns (Cochrane RoB 2); Change from baseline in best-corrected visual acuity (BCVA): Some concerns (Cochrane RoB 2); Central visual field mean deviation (Humphrey automated visual field central region): High risk of bias (Cochrane RoB 2)
Full study record (PLS-00105)Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER.Single-arm trial · Not reported participants · first reported 2017 · automated, not human-reviewed
Population: Patients with Barth syndrome who had completed the 28-week randomized, double-blind, placebo-controlled phase of TAZPOWER and entered the 168-week open-label extension.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: 6-minute walk test distance: Serious concern (Structured limitations appraisal); BTHS-SA Total Fatigue score: Serious concern (Structured limitations appraisal); Safety and tolerability: Serious concern (Structured limitations appraisal); 3D echocardiographic left ventricular volumes: Serious concern (Structured limitations appraisal); MLCL/CL ratio: Serious concern (Structured limitations appraisal)
Full study record (PLS-00106)Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial.Randomized controlled trial · 218 participants · first reported 2023 · automated, not human-reviewed
Population: Participants with genetically confirmed primary mitochondrial myopathy, most with mtDNA alterations and the rest with nDNA defects. Mean age 45.6 years, 64% women, 94% White.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: 6-minute walk test distance: Some concerns (Cochrane RoB 2); PMMSA total fatigue score: Some concerns (Cochrane RoB 2); Adverse events: High risk of bias (Cochrane RoB 2)
Full study record (PLS-00107)Use of Elamipretide in patients assigned treatment in the compassionate use program: Case series in pediatric patients with rare orphan diseases.Case series · Not reported participants · first reported 2022 · automated, not human-reviewed
Population: Pediatric patients aged under 12 years with rare mitochondrial (orphan) disorders treated with elamipretide under a compassionate use program; the specific diagnoses are not stated in the abstract.
Assessed from the abstract only: essential methods may be missing.
Full study record (PLS-00108)Identifying responders to elamipretide in Barth syndrome: Hierarchical clustering for time series data.Randomized controlled trial · 12 participants · first reported 2023 · automated, not human-reviewed
Population: Patients with Barth syndrome enrolled in a randomized, double-blind, placebo-controlled crossover trial of elamipretide.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Accuracy of clustering patients by functional status from wearable physiological data: High risk of bias (Cochrane RoB 2); Accuracy of clustering responders vs non-responders to elamipretide: High risk of bias (Cochrane RoB 2)
Full study record (PLS-00109)Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome.Retrospective cohort · Not reported participants · first reported 2022 · automated, not human-reviewed
Population: Patients with Barth syndrome: 8 elamipretide-treated patients from the TAZPOWER open-label extension and 19 untreated natural history controls, 12 of whom had serial echocardiographic assessments.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: 6-minute walk test distance: No information (ROBINS-I); Muscle strength by handheld dynamometry: No information (ROBINS-I); 5 times sit-to-stand: No information (ROBINS-I); Multi-domain responder index: No information (ROBINS-I); Left ventricular stroke volume: No information (ROBINS-I)
Full study record (PLS-00110)Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy.Randomized controlled trial · 36 participants · first reported 2018 · automated, not human-reviewed
Population: Adults with genetically confirmed primary mitochondrial myopathy (PMM)
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Change in 6-minute walk test distance: High risk of bias (Cochrane RoB 2); Cardiopulmonary exercise testing parameters: Some concerns (Cochrane RoB 2); Participant-reported symptoms: Some concerns (Cochrane RoB 2); Serum and urinary biomarkers: Some concerns (Cochrane RoB 2); Safety and tolerability: Some concerns (Cochrane RoB 2)
Full study record (PLS-00111)A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism.Randomized controlled trial · 12 participants · first reported 2020 · automated, not human-reviewed
Population: Subjects with Barth syndrome (BTHS), a genetic condition caused by TAZ defects that produce abnormal mitochondrial cardiolipin. Age and sex are not stated in the abstract.
Assessed from the abstract only: essential methods may be missing.
Full study record (PLS-00112)Phase 1 Clinical Trial of Elamipretide in Intermediate Age-Related Macular Degeneration and High-Risk Drusen: ReCLAIM High-Risk Drusen Study.Single-arm trial · 21 participants · first reported 2021 · automated, not human-reviewed
Population: Adults aged 55 years or older with intermediate age-related macular degeneration and high-risk drusen (preplanned HRD cohort)
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Adverse events and tolerability: Serious concern (Structured limitations appraisal); Normal-luminance best-corrected visual acuity (BCVA): Serious concern (Structured limitations appraisal); Low-luminance best-corrected visual acuity (LLVA): Serious concern (Structured limitations appraisal); Normal-luminance binocular reading acuity (NLRA): Serious concern (Structured limitations appraisal); Low-luminance binocular reading acuity (LLRA): Serious concern (Structured limitations appraisal); RPE-drusen complex volume by OCT: Serious concern (Structured limitations appraisal); Fundus autofluorescence: Serious concern (Structured limitations appraisal); Mesopic microperimetry: Serious concern (Structured limitations appraisal); Dark adaptation: Serious concern (Structured limitations appraisal)
Full study record (PLS-00113)Phase 1 Clinical Trial of Elamipretide in Dry Age-Related Macular Degeneration and Noncentral Geographic Atrophy: ReCLAIM NCGA Study.Single-arm trial · 19 participants · first reported 2021 · automated, not human-reviewed
Population: Adults aged 55 years or older with dry age-related macular degeneration and noncentral geographic atrophy
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Safety and tolerability (adverse events): Serious concern (Structured limitations appraisal); Normal-luminance best-corrected visual acuity (BCVA): Serious concern (Structured limitations appraisal); Low-luminance best-corrected visual acuity (LLBCVA): Serious concern (Structured limitations appraisal); Low-luminance binocular reading acuity (LLBRA): Serious concern (Structured limitations appraisal); Normal-luminance binocular reading acuity (NLBRA): Serious concern (Structured limitations appraisal); Geographic atrophy area: Serious concern (Structured limitations appraisal); Low-luminance questionnaire (LLQ) self-reported function: Serious concern (Structured limitations appraisal)
Full study record (PLS-00114)Effects of Elamipretide on Left Ventricular Function in Patients With Heart Failure With Reduced Ejection Fraction: The PROGRESS-HF Phase 2 Trial.Randomized controlled trial · 71 participants · first reported 2020 · automated, not human-reviewed
Population: Patients with stable heart failure with reduced ejection fraction (LVEF at or below 40%). Mean age 65 years, 24% female, mean EF 31%.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Change in left ventricular end systolic volume (LVESV): Some concerns (Cochrane RoB 2); Change in left ventricular ejection fraction (LVEF): Some concerns (Cochrane RoB 2); Study drug-related adverse events: Some concerns (Cochrane RoB 2)
Full study record (PLS-00115)A randomized crossover trial of elamipretide in adults with primary mitochondrial myopathy.Randomized controlled trial · 30 participants · first reported 2020 · automated, not human-reviewed
Population: Adults with genetically confirmed primary mitochondrial myopathy
Assessed from the abstract only: essential methods may be missing.
Risk of bias: 6-minute walk test distance: Some concerns (Cochrane RoB 2); PMMSA Total Fatigue score: Some concerns (Cochrane RoB 2); PMMSA Total Fatigue During Activities score: Some concerns (Cochrane RoB 2); Neuro-QoL Fatigue Short Form: Some concerns (Cochrane RoB 2); Patient Global Assessment: Some concerns (Cochrane RoB 2); Physician Global Assessment: Some concerns (Cochrane RoB 2); Triple Timed Up and Go test: Some concerns (Cochrane RoB 2); Wrist and hip accelerometry: Some concerns (Cochrane RoB 2); Adverse events: Some concerns (Cochrane RoB 2)
Full study record (PLS-00116)EMBRACE STEMI study: a Phase 2a trial to evaluate the safety, tolerability, and efficacy of intravenous MTP-131 on reperfusion injury in patients undergoing primary percutaneous coronary intervention.Randomized controlled trial · Not reported participants · first reported 2015 · automated, not human-reviewed
Population: Adults with first-time anterior ST-elevation myocardial infarction due to a proximal or mid left anterior descending artery occlusion who were undergoing primary percutaneous coronary intervention.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Infarct size by CK-MB area under the curve over 72 h: Some concerns (Cochrane RoB 2); Pre-specified MRI, angiographic and electrocardiographic outcomes: High risk of bias (Cochrane RoB 2); Clinical outcomes: High risk of bias (Cochrane RoB 2); Safety and tolerability: High risk of bias (Cochrane RoB 2)
Full study record (PLS-00117)Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study.Randomized controlled trial · 12 participants · first reported 2025 · automated, not human-reviewed
Population: Adolescent and adult white males with genetically confirmed Barth syndrome (TAZ mutations). Mean age 19.5 years (SD 7.7). Overall symptoms were rated mild to very severe.
Assessed from the abstract only: essential methods may be missing.
Full study record (PLS-00118)Novel Mitochondria-Targeting Peptide in Heart Failure Treatment: A Randomized, Placebo-Controlled Trial of Elamipretide.Randomized controlled trial · 36 participants · first reported 2015 · automated, not human-reviewed
Population: Patients with heart failure with reduced ejection fraction (ejection fraction at or below 35%)
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Left ventricular end-diastolic volume: High risk of bias (Cochrane RoB 2); Left ventricular end-systolic volume: High risk of bias (Cochrane RoB 2); Serious adverse events: Some concerns (Cochrane RoB 2)
Full study record (PLS-00120)In vivo mitochondrial ATP production is improved in older adult skeletal muscle after a single dose of elamipretide in a randomized trial.Randomized controlled trial · 39 participants · first reported 2021 · automated, not human-reviewed
Population: Healthy older adults aged 60 to 85 years (46% female), enrolled because they had poorly functioning skeletal muscle mitochondria. The first dorsal interosseous muscle was studied.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Mitochondrial energetic capacity (ATPmax) during exercise: Some concerns (Cochrane RoB 2); Resting mitochondrial coupling (P/O, ATP supply per O2 uptake): Some concerns (Cochrane RoB 2); Volitional fatigue resistance: Some concerns (Cochrane RoB 2)
Full study record (PLS-00121)Phase 2a Clinical Trial of Mitochondrial Protection (Elamipretide) During Stent Revascularization in Patients With Atherosclerotic Renal Artery Stenosis.Randomized controlled trial · 16 participants · first reported 2012 · automated, not human-reviewed
Population: Patients with severe atherosclerotic renal artery stenosis scheduled for percutaneous transluminal renal angioplasty (PTRA) and stenting; age and baseline glomerular filtration rate did not differ between groups.
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Stenotic kidney fractional hypoxia: High risk of bias (Cochrane RoB 2); Stenotic kidney renal blood flow: High risk of bias (Cochrane RoB 2); Estimated glomerular filtration rate: High risk of bias (Cochrane RoB 2); Systolic blood pressure: High risk of bias (Cochrane RoB 2)
Full study record (PLS-00122)The mitochondria-targeting peptide elamipretide diminishes circulating HtrA2 in ST-segment elevation myocardial infarction.Randomized controlled trial · 35 participants · first reported 2017 · automated, not human-reviewed
Population: Patients with first-time acute anterior ST-segment elevation myocardial infarction after percutaneous coronary intervention (n=19, a subset of the EMBRACE trial), plus a reference group of healthy blood donors (n=16).
Assessed from the abstract only: essential methods may be missing.
Risk of bias: Serum HtrA2 level: High risk of bias (Cochrane RoB 2)
Full study record (PLS-00123)Urinary mitochondrial DNA copy number identifies renal mitochondrial injury in renovascular hypertensive patients undergoing renal revascularization: A Pilot Study.Other design · 19 participants · first reported 2019 · automated, not human-reviewed
Population: Patients with renovascular hypertension undergoing percutaneous transluminal renal angioplasty (PTRA), plus healthy volunteers as reference controls.
Assessed from the abstract only: essential methods may be missing.
Full study record (PLS-00124)Relation of Left Ventricular Mass and Infarct Size in Anterior Wall ST-Segment Elevation Acute Myocardial Infarction (from the EMBRACE STEMI Clinical Trial).Randomized controlled trial · 300 participants · first reported 2012 · automated, not human-reviewed
Population: Patients with a first anterior ST-segment elevation myocardial infarction, an occluded left anterior descending artery and an available LV mass measurement in the EMBRACE STEMI trial (n = 100 in this post hoc analysis).
Assessed from the abstract only: essential methods may be missing.
Risk of bias: CK-MB area under the curve (biomarker infarct size): High risk of bias (Cochrane RoB 2); Troponin I area under the curve (biomarker infarct size): High risk of bias (Cochrane RoB 2); Myocardial salvage index: High risk of bias (Cochrane RoB 2)
Full study record (PLS-00125)Study to Evaluate Safety, Tolerability, and Pharmacokinetics (PK) of Intravenous (IV) Infusion of MTP-131 (Bendavia™) in Healthy AdultsOther design · 40 participants · first reported 2010 · automated, not human-reviewed
Population: Healthy adults
Full study record (PLS-00213)Study to Assess the Pharmacodynamic Effects of Unfractionated Heparin (UFH) in Healthy Volunteers With and Without BendaviaOther design · 12 participants · first reported 2012 · automated, not human-reviewed
Population: Healthy volunteers
Full study record (PLS-00216)The Impact of Intravenous Bendavia™ on Endothelial Reactivity Dysfunction in Cigarette SmokingOther design · 6 participants · first reported 2012 · automated, not human-reviewed
Population: Healthy volunteers exposed to cigarette smoking. Smoking status and other eligibility details are not stated in the record.
Full study record (PLS-00217)A Phase 1 Study Examining the Pharmacokinetics and Tolerability of a Single Oral Dose of Bendavia (MTP-131)Other design · 30 participants · first reported 2012 · automated, not human-reviewed
Population: Healthy volunteers
Full study record (PLS-00218)Safety and Pharmacokinetic (PK) Study of Oral Bendavia Administered for 7 DaysOther design · 30 participants · first reported 2013 · automated, not human-reviewed
Population: Healthy volunteers
Full study record (PLS-00219)A Phase 2 Study to Evaluate the Impact of MTP-131 (Bendavia™) on Skeletal Muscle Function in ElderlyOther design · 41 participants · first reported 2014 · automated, not human-reviewed
Population: Elderly people with skeletal muscle mitochondrial dysfunction
Full study record (PLS-00220)A Study of MTP-131 Topical Ophthalmic Solution in Subjects With Diabetic Macular Edema and Non-Exudative Intermediate Age-related Macular DegenerationOther design · 20 participants · first reported 2014 · automated, not human-reviewed
Population: Subjects with diabetic macular edema or non-exudative intermediate age-related macular degeneration
Full study record (PLS-00221)Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial MyopathyOther design · 36 participants · first reported 2015 · automated, not human-reviewed
Population: People with mitochondrial myopathy. Age and other eligibility details are not stated in the record.
Full study record (PLS-00222)A Phase 1 Study Investigating the Safety and Pharmacokinetics of Repeat-dose Intravenous Infusion of MTP-131 in Subjects With Impaired Renal FunctionOther design · 23 participants · first reported 2015 · automated, not human-reviewed
Population: Subjects with impaired renal function (registered condition: normal and impaired renal function)
Full study record (PLS-00224)A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED)Other design · 22 participants · first reported 2016 · automated, not human-reviewed
Population: People with Fuchs' corneal endothelial dystrophy (FCED).
Full study record (PLS-00228)A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic NeuropathyOther design · 12 participants · first reported 2016 · automated, not human-reviewed
Population: People with Leber's hereditary optic neuropathy
Full study record (PLS-00229)Effect of Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Reduced Ejection FractionOther design · 71 participants · first reported 2016 · automated, not human-reviewed
Population: Adults with stable heart failure with reduced ejection fraction (per study title)
Full study record (PLS-00230)Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER StudyOther design · 30 participants · first reported 2016 · automated, not human-reviewed
Population: Subjects with primary mitochondrial disease drawn from the earlier MMPOWER study. Age range and other eligibility details are not stated in the record.
Full study record (PLS-00231)A Study to Evaluate the Effects of 4 Weeks Treatment With Subcutaneous Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Preserved Ejection FractionOther design · 46 participants · first reported 2016 · automated, not human-reviewed
Population: Adults with stable heart failure with preserved ejection fraction (registered condition: chronic heart failure).
Full study record (PLS-00232)An Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Intermediate Age-Related Macular DegenerationSingle-arm trial · 40 participants · first reported 2016 · automated, not human-reviewed
Population: Subjects with intermediate age-related macular degeneration
Full study record (PLS-00233)A Phase 2 Study to Evaluate the Cardiac and Renal Effects of Short Term Treatment With Elamipretide in Patients Hospitalized With Congestion Due to Heart FailureOther design · 308 participants · first reported 2016 · automated, not human-reviewed
Population: Patients hospitalized with congestion due to heart failure
Full study record (PLS-00234)Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)Single-arm trial · 28 participants · first reported 2016 · automated, not human-reviewed
Population: Subjects with genetically confirmed primary mitochondrial myopathy (PMM) entering an open-label extension
Full study record (PLS-00236)An Intermediate Size Expanded Access Protocol of ElamipretideOther design · Not reported participants · first reported 2020 · automated, not human-reviewed
Population: People with mitochondrial diseases, including Barth syndrome, receiving elamipretide under an intermediate-size expanded access protocol.
Full study record (PLS-00240)Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)Other design · 102 participants · first reported 2021 · automated, not human-reviewed
Population: Subjects with primary mitochondrial disease caused by nuclear DNA mutations (nPMD). Age and other eligibility details are not stated in the record.
Full study record (PLS-00241)FRDA Investigator Initiated Study (IIS) With ElamipretideOther design · 20 participants · first reported 2021 · automated, not human-reviewed
Population: People with Friedreich ataxia
Full study record (PLS-00242)ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD)Other design · 313 participants · first reported 2024 · automated, not human-reviewed
Population: Subjects with dry age-related macular degeneration (dry AMD)
Full study record (PLS-00244)Study of Healthy Aging and Physical Function With ElamipretideOther design · Not reported participants · first reported 2025 · automated, not human-reviewed
Population: Healthy aging adults (registered condition: Aging, Healthy); eligibility details not provided in the record
Full study record (PLS-00245)Clinical Trial in Patients With Barth Syndrome- 4TAZPowerOther design · Not reported participants · first reported 2026 · automated, not human-reviewed
Population: Patients with Barth syndrome. Eligibility criteria and age range are not stated in the record.
Full study record (PLS-00250)Records reviewed and excluded (3)
- The Mitochondrial Antioxidant SS-31 Modulates Oxidative Stress, Endoplasmic Reticulum Stress, and Autophagy in Type 2 Diabetes.: not human
- The mitochondrial antioxidant SS-31 increases SIRT1 levels and ameliorates inflammation, oxidative stress and leukocyte-endothelium interactions in type 2 diabetes.: not human
- A Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Bendavia™ (MTP-131) in Patients With Heart Failure: other
Why the counts differ
Publications, studies and registrations are different units.
These numbers count different things. The research snapshot 2026.1.1 counts 30 human clinical publications: PubMed records, classified by publication type. Here, each reviewed study is counted once (51 in all), however many reports describe it (30 PubMed records, 30 registrations, 0 other reports such as meeting abstracts outside PubMed).
| Study | Source record | Role |
|---|---|---|
| PLS-00036 (excluded) | PMID 31466264The Mitochondrial Antioxidant SS-31 Modulates Oxidative Stress, Endoplasmic Reticulum Stress, and Autophagy in Type 2 Diabetes. | primary |
| PLS-00097 | PMID 42415111Elamipretide in pediatric Barth syndrome: from heart failure to school return. | primary |
| PLS-00098 | PMID 39619320Expanded-Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series. | primary |
| PLS-00099 | PMID 39917770Expanded-access use of elamipretide in a newborn with Barth syndrome: a case report. | primary |
| PLS-00100 | PMID 39574155Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial. | primary |
| PLS-00100 | NCT03323749 · results postedA Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension | registry entry |
| PLS-00101 | PMID 40645388Cardiac pathology in a patient with a novel pathogenic variant c.703del (p.Ile235SerfsTer4) of the TAFAZZIN gene. | primary |
| PLS-00102 | PMID 40816230Elamipretide in the Management of Barth Syndrome: Current Evidence and a Case Report. | primary |
| PLS-00103 | PMID 38919884Expanded-access use of elamipretide in a patient with membrane protein-associated neurodegeneration. | primary |
| PLS-00104 | PMID 39605874ReCLAIM-2: A Randomized Phase II Clinical Trial Evaluating Elamipretide in Age-related Macular Degeneration, Geographic Atrophy Growth, Visual Function, and Ellipsoid Zone Preservation. | primary |
| PLS-00104 | NCT03891875 · results postedReCLAIM-2 Study to Evaluate Safety,Efficacy & Pharmacokinetics of Elamipretide in Subjects With AMD With Non-central GA | registry entry |
| PLS-00105 | PMID 37923251Elamipretide Topical Ophthalmic Solution for the Treatment of Subjects with Leber Hereditary Optic Neuropathy: A Randomized Trial. | primary |
| PLS-00106 | PMID 38602181Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER. | primary |
| PLS-00106 | NCT03098797 · results postedA Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome | registry entry |
| PLS-00107 | PMID 37268435Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial. | primary |
| PLS-00108 | PMID 36636586Use of Elamipretide in patients assigned treatment in the compassionate use program: Case series in pediatric patients with rare orphan diseases. | primary |
| PLS-00109 | PMID 37041653Identifying responders to elamipretide in Barth syndrome: Hierarchical clustering for time series data. | primary |
| PLS-00110 | PMID 36056411Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome. | primary |
| PLS-00111 | PMID 29500292Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy. | primary |
| PLS-00112 | PMID 33077895A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism. | primary |
| PLS-00113 | PMID 36246187Phase 1 Clinical Trial of Elamipretide in Intermediate Age-Related Macular Degeneration and High-Risk Drusen: ReCLAIM High-Risk Drusen Study. | primary |
| PLS-00114 | PMID 36246181Phase 1 Clinical Trial of Elamipretide in Dry Age-Related Macular Degeneration and Noncentral Geographic Atrophy: ReCLAIM NCGA Study. | primary |
| PLS-00115 | PMID 32068002Effects of Elamipretide on Left Ventricular Function in Patients With Heart Failure With Reduced Ejection Fraction: The PROGRESS-HF Phase 2 Trial. | primary |
| PLS-00116 | PMID 32096613A randomized crossover trial of elamipretide in adults with primary mitochondrial myopathy. | primary |
| PLS-00117 | PMID 26586786EMBRACE STEMI study: a Phase 2a trial to evaluate the safety, tolerability, and efficacy of intravenous MTP-131 on reperfusion injury in patients undergoing primary percutaneous coronary intervention. | primary |
| PLS-00118 | PMID 40281531Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study. | primary |
| PLS-00119 (excluded) | PMID 30367115The mitochondrial antioxidant SS-31 increases SIRT1 levels and ameliorates inflammation, oxidative stress and leukocyte-endothelium interactions in type 2 diabetes. | primary |
| PLS-00120 | PMID 29217757Novel Mitochondria-Targeting Peptide in Heart Failure Treatment: A Randomized, Placebo-Controlled Trial of Elamipretide. | primary |
| PLS-00120 | NCT02388464 · no results postedA Study Investigating the Safety, Tolerability, and Pharmacokinetics of MTP-131 in Subjects With Congestive Heart Failure | registry entry |
| PLS-00121 | PMID 34264994In vivo mitochondrial ATP production is improved in older adult skeletal muscle after a single dose of elamipretide in a randomized trial. | primary |
| PLS-00122 | PMID 28916603Phase 2a Clinical Trial of Mitochondrial Protection (Elamipretide) During Stent Revascularization in Patients With Atherosclerotic Renal Artery Stenosis. | primary |
| PLS-00122 | NCT01755858 · results postedEffects of Intravenous Bendavia™ on Reperfusion Injury in Patients Undergoing Angioplasty of the Renal Artery | registry entry |
| PLS-00123 | PMID 28534645The mitochondria-targeting peptide elamipretide diminishes circulating HtrA2 in ST-segment elevation myocardial infarction. | primary |
| PLS-00124 | PMID 30803138Urinary mitochondrial DNA copy number identifies renal mitochondrial injury in renovascular hypertensive patients undergoing renal revascularization: A Pilot Study. | primary |
| PLS-00125 | PMID 27392509Relation of Left Ventricular Mass and Infarct Size in Anterior Wall ST-Segment Elevation Acute Myocardial Infarction (from the EMBRACE STEMI Clinical Trial). | primary |
| PLS-00125 | NCT01572909 · results postedEvaluation of Myocardial Effects of MTP-131 for Reducing Reperfusion Injury in Patients With Acute Coronary Events | registry entry |
| PLS-00213 | NCT01115920 · no results postedStudy to Evaluate Safety, Tolerability, and Pharmacokinetics (PK) of Intravenous (IV) Infusion of MTP-131 (Bendavia™) in Healthy Adults | registry entry |
| PLS-00216 | NCT01513200 · no results postedStudy to Assess the Pharmacodynamic Effects of Unfractionated Heparin (UFH) in Healthy Volunteers With and Without Bendavia | registry entry |
| PLS-00217 | NCT01518985 · no results postedThe Impact of Intravenous Bendavia™ on Endothelial Reactivity Dysfunction in Cigarette Smoking | registry entry |
| PLS-00218 | NCT01754818 · no results postedA Phase 1 Study Examining the Pharmacokinetics and Tolerability of a Single Oral Dose of Bendavia (MTP-131) | registry entry |
| PLS-00219 | NCT01786915 · no results postedSafety and Pharmacokinetic (PK) Study of Oral Bendavia Administered for 7 Days | registry entry |
| PLS-00220 | NCT02245620 · results postedA Phase 2 Study to Evaluate the Impact of MTP-131 (Bendavia™) on Skeletal Muscle Function in Elderly | registry entry |
| PLS-00221 | NCT02314299 · no results postedA Study of MTP-131 Topical Ophthalmic Solution in Subjects With Diabetic Macular Edema and Non-Exudative Intermediate Age-related Macular Degeneration | registry entry |
| PLS-00222 | NCT02367014 · results postedSafety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy | registry entry |
| PLS-00223 (excluded) | NCT02388529 · no results postedA Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Bendavia™ (MTP-131) in Patients With Heart Failure | registry entry |
| PLS-00224 | NCT02436447 · no results postedA Phase 1 Study Investigating the Safety and Pharmacokinetics of Repeat-dose Intravenous Infusion of MTP-131 in Subjects With Impaired Renal Function | registry entry |
| PLS-00228 | NCT02653391 · results postedA Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED) | registry entry |
| PLS-00229 | NCT02693119 · results postedA Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy | registry entry |
| PLS-00230 | NCT02788747 · results postedEffect of Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Reduced Ejection Fraction | registry entry |
| PLS-00231 | NCT02805790 · results postedSafety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study | registry entry |
| PLS-00232 | NCT02814097 · no results postedA Study to Evaluate the Effects of 4 Weeks Treatment With Subcutaneous Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Preserved Ejection Fraction | registry entry |
| PLS-00233 | NCT02848313 · results postedAn Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Intermediate Age-Related Macular Degeneration | registry entry |
| PLS-00234 | NCT02914665 · no results postedA Phase 2 Study to Evaluate the Cardiac and Renal Effects of Short Term Treatment With Elamipretide in Patients Hospitalized With Congestion Due to Heart Failure | registry entry |
| PLS-00236 | NCT02976038 · results postedOpen-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM) | registry entry |
| PLS-00240 | NCT04689360 · no results postedAn Intermediate Size Expanded Access Protocol of Elamipretide | registry entry |
| PLS-00241 | NCT05162768 · no results postedStudy to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) | registry entry |
| PLS-00242 | NCT05168774 · results postedFRDA Investigator Initiated Study (IIS) With Elamipretide | registry entry |
| PLS-00244 | NCT06373731 · no results postedReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD) | registry entry |
| PLS-00245 | NCT07275424 · no results postedStudy of Healthy Aging and Physical Function With Elamipretide | registry entry |
| PLS-00250 | NCT07531251 · no results postedClinical Trial in Patients With Barth Syndrome- 4TAZPower | registry entry |
Evidence gaps
Research activity gap: No gap identified
Little indexed research of any kind.
Human research gap: No gap identified
Laboratory or animal research exists, but little human research.
Evidence quality gap: Gap identified
Human research exists, but no assessed outcome reaches moderate or high certainty.
Clinical relevance gap: No gap identified
Studies exist but none directly address a claim being evaluated.
Safety evidence gap: Gap identified
Safety is not established with moderate or high certainty for the populations, routes or durations studied.
Timeline
Registrations, publications, posted results and every change to the assessments.
2010-05-04
Registered: NCT01115920
2012-01-20
Registered: NCT01513200
2012-01-26
Registered: NCT01518985
2012-04-06
Registered: NCT01572909
2012-12-21
Registered: NCT01754818
2012-12-24
Registered: NCT01755858
2013-02-08
Registered: NCT01786915
2014-09-19
Registered: NCT02245620
2014-12-11
Registered: NCT02314299
2015-02-20
Registered: NCT02367014
2015-03-17
Registered: NCT02388464
2015-03-17
Registered: NCT02388529
2015-05-06
Registered: NCT02436447
2015-11-19
Published: EMBRACE STEMI study: a Phase 2a trial to evaluate the safety, tolerability, and efficacy of intravenous MTP-131 on reperfusion injury in patients undergoing primary percutaneous coronary intervention.
2016-01-12
Registered: NCT02653391
2016-02-26
Registered: NCT02693119
2016-06-02
Registered: NCT02788747
2016-06-15
Published: Relation of Left Ventricular Mass and Infarct Size in Anterior Wall ST-Segment Elevation Acute Myocardial Infarction (from the EMBRACE STEMI Clinical Trial).
2016-06-20
Registered: NCT02805790
2016-06-27
Registered: NCT02814097
2016-07-28
Registered: NCT02848313
2016-09-26
Registered: NCT02914665
2016-11-29
Registered: NCT02976038
2017-04-04
Registered: NCT03098797
2017-05-23
Published: The mitochondria-targeting peptide elamipretide diminishes circulating HtrA2 in ST-segment elevation myocardial infarction.
2017-09-01
Published: Phase 2a Clinical Trial of Mitochondrial Protection (Elamipretide) During Stent Revascularization in Patients With Atherosclerotic Renal Artery Stenosis.
2017-10-27
Registered: NCT03323749
2017-12-01
Published: Novel Mitochondria-Targeting Peptide in Heart Failure Treatment: A Randomized, Placebo-Controlled Trial of Elamipretide.
2018-03-02
Published: Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy.
2018-10-26
Published: The mitochondrial antioxidant SS-31 increases SIRT1 levels and ameliorates inflammation, oxidative stress and leukocyte-endothelium interactions in type 2 diabetes.
2019-03-13
Published: Urinary mitochondrial DNA copy number identifies renal mitochondrial injury in renovascular hypertensive patients undergoing renal revascularization: A Pilot Study.
2019-03-27
Registered: NCT03891875
2019-08-28
Published: The Mitochondrial Antioxidant SS-31 Modulates Oxidative Stress, Endoplasmic Reticulum Stress, and Autophagy in Type 2 Diabetes.
2019-12-27
Results posted: NCT02367014
2020-02-14
Published: Effects of Elamipretide on Left Ventricular Function in Patients With Heart Failure With Reduced Ejection Fraction: The PROGRESS-HF Phase 2 Trial.
2020-02-25
Published: A randomized crossover trial of elamipretide in adults with primary mitochondrial myopathy.
2020-04-22
Results posted: NCT02788747
2020-06-11
Results posted: NCT01572909
2020-06-23
Results posted: NCT02245620
2020-07-07
Results posted: NCT02805790
2020-08-11
Results posted: NCT01755858
2020-09-21
Results posted: NCT02848313
2020-10-20
Published: A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism.
2020-12-30
Registered: NCT04689360
2021-04-02
Results posted: NCT03323749
2021-07-15
Published: In vivo mitochondrial ATP production is improved in older adult skeletal muscle after a single dose of elamipretide in a randomized trial.
2021-09-17
Results posted: NCT02653391
2021-10-12
Results posted: NCT02693119
2021-11-27
Published: Phase 1 Clinical Trial of Elamipretide in Dry Age-Related Macular Degeneration and Noncentral Geographic Atrophy: ReCLAIM NCGA Study.
2021-12-17
Registered: NCT05162768
2021-12-17
Results posted: NCT02976038
2021-12-22
Published: Phase 1 Clinical Trial of Elamipretide in Intermediate Age-Related Macular Degeneration and High-Risk Drusen: ReCLAIM High-Risk Drusen Study.
2021-12-23
Registered: NCT05168774
2022-09-02
Published: Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome.
2022-09-21
Published: Use of Elamipretide in patients assigned treatment in the compassionate use program: Case series in pediatric patients with rare orphan diseases.
2023-04-11
Published: Identifying responders to elamipretide in Barth syndrome: Hierarchical clustering for time series data.
2023-06-02
Published: Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial.
2023-10-17
Results posted: NCT03891875
2023-11-03
Published: Elamipretide Topical Ophthalmic Solution for the Treatment of Subjects with Leber Hereditary Optic Neuropathy: A Randomized Trial.
2024-04-08
Published: Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER.
2024-04-16
Results posted: NCT03098797
2024-04-18
Registered: NCT06373731
2024-06-24
Published: Expanded-access use of elamipretide in a patient with membrane protein-associated neurodegeneration.
2024-10-09
Published: ReCLAIM-2: A Randomized Phase II Clinical Trial Evaluating Elamipretide in Age-related Macular Degeneration, Geographic Atrophy Growth, Visual Function, and Ellipsoid Zone Preservation.
2024-11-21
Published: Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial.
2024-11-29
Published: Expanded-Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series.
2025-01-22
Published: Expanded-access use of elamipretide in a newborn with Barth syndrome: a case report.
2025-04-25
Published: Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study.
2025-07-10
Published: Cardiac pathology in a patient with a novel pathogenic variant c.703del (p.Ile235SerfsTer4) of the TAFAZZIN gene.
2025-08-11
Published: Elamipretide in the Management of Barth Syndrome: Current Evidence and a Case Report.
2025-12-10
Registered: NCT07275424
2025-12-12
Results posted: NCT05168774
2026-04-15
Registered: NCT07531251
2026-07-07
Published: Elamipretide in pediatric Barth syndrome: from heart failure to school return.
2026-11-07
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Sources and method
Every assessment links its sources and keeps its full version history. Methodology · All assessments (CSV) · All study records (CSV)
These assessments describe published research. Records marked automated were produced by AI under the published methodology and have not been reviewed by a person; they can contain errors. None of this is medical advice, not a statement about any product’s safety for human use, and not a regulatory approval. Peptide Library’s vendor and partner relationships play no part in them (editorial independence).