Human study PLS-00120

Novel Mitochondria-Targeting Peptide in Heart Failure Treatment: A Randomized, Placebo-Controlled Trial of Elamipretide.

Randomized controlled trial · 36 participants enrolled

Version 1 · published November 7, 2026 · evidence through November 7, 2026 · research snapshot 2026.1.1 · methodology ev-1 · Automated assessment by AI (claude-opus-5-5 and claude-sonnet-5-5), not reviewed by a person.

Only the abstract was available. Essential methods may be missing, which limits what can be judged.

Reports of this study

Every publication, registration and abstract describing this one study. Participants are counted once.

Novel Mitochondria-Targeting Peptide in Heart Failure Treatment: A Randomized, Placebo-Controlled Trial of Elamipretide.primary · Circulation. Heart failure · 2017-12-01 · PMID 29217757 · DOI
A Study Investigating the Safety, Tolerability, and Pharmacokinetics of MTP-131 in Subjects With Congestive Heart Failureregistry entry · NCT02388464 (COMPLETED, no results posted, planned enrollment 36)

Design and participants

Design: Randomized controlled trial
Population: Patients with heart failure with reduced ejection fraction (ejection fraction at or below 35%)
Conditions: Heart failure with reduced ejection fraction, Congestive heart failure
Planned enrollment (registry): Not reported
Enrolled: 36
Randomized: 36
Completed follow-up: Not reported

Intervention and comparator

What each group received. Related peptides, analogs and combinations are never treated as the compound itself.

Elamipretide cohort 1 (lowest dose) (intervention) · elamipretide · Elamipretide (MTP-131), lowest of three ascending dose levels · intravenous infusion · Single 4-hour infusion · 8 assigned
Elamipretide cohort 2 (middle dose) (intervention) · elamipretide · Elamipretide (MTP-131), middle of three ascending dose levels · intravenous infusion · Single 4-hour infusion · 8 assigned
Elamipretide cohort 3 (highest dose) (intervention) · elamipretide · Elamipretide (MTP-131), highest of three ascending dose levels · intravenous infusion · Single 4-hour infusion · 8 assigned
Placebo (comparator) · Placebo infusion · placebo · intravenous infusion · Single 4-hour infusion · 12 assigned

What the authors reported

As stated in the sources. This is the authors’ account, not Peptide Library’s assessment.

Left ventricular end-diastolic volume

unclear outcome · surrogate · End of infusion (also pre-, mid-infusion and 6, 8, 12, 24 hours after infusion start) · Echocardiography

Elamipretide cohort 3 (highest dose) vs Placebo: mean difference -18, p = 0.009

Left ventricular end-systolic volume

unclear outcome · surrogate · End of infusion (also pre-, mid-infusion and 6, 8, 12, 24 hours after infusion start) · Echocardiography

Elamipretide cohort 3 (highest dose) vs Placebo: mean difference -14, p = 0.005

Serious adverse events

unclear outcome · safety · Up to 24 hours after infusion start · Clinical and laboratory safety assessments

—: narrative — No serious adverse events occurred

Blood pressure and heart rate

unclear outcome · safety · Pre-, mid- and end-infusion and up to 24 hours after infusion start · Clinical vital-sign assessments

—: narrative — Blood pressure and heart rate remained stable in all cohorts

Plasma elamipretide pharmacokinetics

unclear outcome · surrogate · Through 24 hours after infusion start · Plasma concentrations

—: narrative — Peak plasma levels occurred at end of infusion and were undetectable by 24 hours

Adverse events

Serious adverse events (serious): 0 participants

Monitoring: Clinical, laboratory and echocardiographic assessments at pre-, mid- and end-infusion and at 6, 8, 12 and 24 hours after infusion start.

The authors concluded (paraphrased): A single infusion of elamipretide was safe and well tolerated in HFrEF. The high dose produced favorable changes in LV volumes that correlated with peak plasma levels, suggesting a dose effect. Longer studies are needed to assess safety and efficacy.

Peptide Library’s assessment

An automated assessment, made by AI separately from the authors’ conclusions and not reviewed by a person. How risk of bias is assessed.

Limitations

small or imprecise: Only 8 patients per dose cohort and 12 on placebo, so estimates are imprecise and safety detection is limited.
short follow up: Single 4-hour infusion with follow-up to 24 hours only; long-term safety and efficacy are unknown.
inappropriate endpoint: Efficacy findings are echocardiographic LV volumes (surrogates), not symptoms, function or clinical events.
selective reporting: Many timepoints and three dose cohorts were assessed, but only the significant end-infusion highest-dose comparison is reported.
abstract only: Only the abstract and a registry entry without results are available; methods, adverse event detail and funding are not reported.

Left ventricular end-diastolic volume: High risk of bias (Cochrane RoB 2)

Risk of bias in one result of a randomized trial, judged in five domains.

Domain judgments
Randomization process	Some concerns	Abstract states randomization but gives no method of sequence generation or allocation concealment.
Deviations from intended interventions	Some concerns	Described as double-blind, but adherence and deviations are not reported in the abstract.
Missing outcome data	Some concerns	Numbers analyzed and any missing echocardiographic data are not reported in the abstract.
Measurement of the outcome	Some concerns	Echocardiographic volumes were measured under double-blinding, but blinding of the echo reader is not described.
Selection of the reported result	High risk of bias	Multiple doses and seven timepoints were assessed, yet only the significant highest-dose end-infusion contrast is reported.

Selective reporting of one significant contrast among many timepoints and doses, plus sparse methods detail, gives high risk.

Version 1 · published November 7, 2026 · evidence through November 7, 2026 · research snapshot 2026.1.1 · methodology ev-1 · RoB 2 (2019-08-22) · Automated assessment by AI (claude-opus-5-5 and claude-sonnet-5-5), not reviewed by a person.

Left ventricular end-systolic volume: High risk of bias (Cochrane RoB 2)

Risk of bias in one result of a randomized trial, judged in five domains.

Domain judgments
Randomization process	Some concerns	Abstract states randomization but gives no method of sequence generation or allocation concealment.
Deviations from intended interventions	Some concerns	Described as double-blind, but adherence and deviations are not reported in the abstract.
Missing outcome data	Some concerns	Numbers analyzed and any missing echocardiographic data are not reported in the abstract.
Measurement of the outcome	Some concerns	Echocardiographic volumes were measured under double-blinding, but blinding of the echo reader is not described.
Selection of the reported result	High risk of bias	Multiple doses and seven timepoints were assessed, yet only the significant highest-dose end-infusion contrast is reported.

Selective reporting of one significant contrast among many timepoints and doses, plus sparse methods detail, gives high risk.

Serious adverse events: Some concerns (Cochrane RoB 2)

Risk of bias in one result of a randomized trial, judged in five domains.

Domain judgments
Randomization process	Some concerns	Abstract states randomization but gives no method of sequence generation or allocation concealment.
Deviations from intended interventions	Some concerns	Double-blind design is stated, but deviations from intended infusion are not reported.
Missing outcome data	Some concerns	Completion and analysis numbers are not given in the abstract or the registry entry.
Measurement of the outcome	Some concerns	Safety was assessed clinically under blinding, but adverse event ascertainment methods are not described.
Selection of the reported result	Some concerns	Only a narrative statement of no serious adverse events is given; non-serious adverse events are not reported.

Blinded randomized design, but sparse abstract-level reporting of methods and adverse events raises some concerns.

Claims this study informs

Improves function in Barth syndrome: not eligible (wrong population) for “Muscle strength, exercise capacity and fatigue”
Improves exercise capacity in primary mitochondrial myopathy: not eligible (wrong population) for “Six-minute walk distance and fatigue”
Is safe for human administration: eligible for “Adverse events, including injection-site reactions, and discontinuations”, indirect evidence

Insufficient information to assess Adverse events, including injection-site reactions, and discontinuations

Where each value comes from

adverse_events: PMID 29217757, abstract (Methods and results, sentence 4) · extracted by AI (automated, not verified by a person) · abstract only
arms: PMID 29217757, abstract (Methods and results, sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
author_conclusion: PMID 29217757, abstract (Conclusions, sentences 1-3) · extracted by AI (automated, not verified by a person) · abstract only
design: PMID 29217757, abstract (Methods and results, sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
limitations: PMID 29217757, abstract (Methods and results and Conclusions) · extracted by AI (automated, not verified by a person) · abstract only
outcomes: PMID 29217757, abstract (Methods and results, sentences 2-6) · extracted by AI (automated, not verified by a person) · abstract only
participants: PMID 29217757, abstract (Methods and results, sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
participants: NCT NCT02388464, registry (enrollment) · extracted by AI (automated, not verified by a person) · abstract only
population: PMID 29217757, abstract (Methods and results, sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
results: PMID 29217757, abstract (Methods and results, sentence 6) · extracted by AI (automated, not verified by a person) · abstract only

Version 1 · published November 7, 2026 · evidence through November 7, 2026 · research snapshot 2026.1.1 · methodology ev-1 · Automated assessment by AI (claude-opus-5-5 and claude-sonnet-5-5), not reviewed by a person.

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).