Clinical Trials

Numerous clinical trials evaluate the mitochondria-targeted peptide elamipretide across Phase 1 through Phase 4 investigations for diverse indications, sponsored by industry developers such as Stealth BioTherapeutics alongside academic institutions like University Hospital Montpellier. Key conditions under evaluation include heart failure, age-related macular degeneration, impaired renal function, and primary mitochondrial diseases such as Barth syndrome and mitochondrial myopathies. With statuses ranging from completed and active to currently recruiting, these trials reflect ongoing therapeutic development for targeted mitochondrial dysfunction.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07531251 RECRUITING
Barth Syndrome
Stealth BioTherapeutics Inc.
 2026-07-02 PHASE4
NCT06373731 ACTIVE_NOT_RECRUITING
Age Related Macular Degeneration (ARMD)
Stealth BioTherapeutics Inc.
 2024-05-30 PHASE3
NCT07275424 RECRUITING
Aging, Healthy
David Marcinek
 2025-11-26 PHASE2
NCT05162768 COMPLETED
Mitochondrial Myopathies; Mitochondrial Pathology; Mitochondrial DNA Mutation; Mitochondrial Diseases; Mitochondrial DNA Deletion; Mitochondrial DNA Depletion; Mitochondrial Metabolism Defect; Mitochondrial Complex I Deficiency
Stealth BioTherapeutics Inc.
 2022-04-29 PHASE3
NCT05168774 COMPLETED
Friedreich Ataxia
Children's Hospital of Philadelphia
 2022-03-03 PHASE1; PHASE2
NCT03891875 COMPLETED
Age-related Macular Degeneration
Stealth BioTherapeutics Inc.
 2019-03-27 PHASE2
NCT05194631 Recruiting
Diaphragm Injury|Mechanical Ventilation Complication
University Hospital Montpellier
 2022-02-18 Not Applicable
NCT02976038 TERMINATED
Primary Mitochondrial Disease
Stealth BioTherapeutics Inc.
 2016-12 PHASE2
NCT03323749 TERMINATED
Primary Mitochondrial Myopathy
Stealth BioTherapeutics Inc.
 2017-10-09 PHASE3
NCT02693119 COMPLETED
Leber's Hereditary Optic Neuropathy
Stealth BioTherapeutics Inc.
 2016-03 PHASE2
NCT03098797 COMPLETED
Barth Syndrome
Stealth BioTherapeutics Inc.
 2017-07-05 PHASE2; PHASE3
NCT02848313 COMPLETED
Age-Related Macular Degeneration
Stealth BioTherapeutics Inc.
 2016-10-28 PHASE1
NCT02653391 COMPLETED
Fuchs' Corneal Endothelial Dystrophy (FCED)
Stealth BioTherapeutics Inc.
 2016-02 PHASE1; PHASE2
NCT02914665 COMPLETED
Heart Failure
Stealth BioTherapeutics Inc.
 2016-10-20 PHASE2
NCT02788747 COMPLETED
Heart Failure
Stealth BioTherapeutics Inc.
 2016-06 PHASE2
NCT02814097 COMPLETED
Chronic Heart Failure
Stealth BioTherapeutics Inc.
 2016-09-02 PHASE2
NCT02805790 COMPLETED
Primary Mitochondrial Disease
Stealth BioTherapeutics Inc.
 2016-08-22 PHASE2
NCT02914665 Completed
Heart Failure
Stealth BioTherapeutics Inc.
 2016-10-20 Phase 2
NCT02848313 Completed
Age-Related Macular Degeneration
Stealth BioTherapeutics Inc.
 2016-10-28 Phase 1
NCT02814097 Completed
Chronic Heart Failure
Stealth BioTherapeutics Inc.|Charite University Berlin Germany|SCIRENT Clinical Research and Science d.o.o.
 2016-09-02 Phase 2
NCT02245620 COMPLETED
Skeletal Muscle Mitochondrial Dysfunction in the Elderly
Stealth BioTherapeutics Inc.
 2015-01-15 PHASE2
NCT02367014 COMPLETED
Mitochondrial Myopathy
Stealth BioTherapeutics Inc.
 2015-02 PHASE1; PHASE2
NCT02388529 WITHDRAWN
Heart Failure
Stealth BioTherapeutics Inc.
 2015-05 PHASE1
NCT02436447 COMPLETED
Normal and Impaired Renal Function
Stealth BioTherapeutics Inc.
 2015-05 PHASE1
NCT02436447 Completed
Normal and Impaired Renal Function
Stealth BioTherapeutics Inc.
 2015-05 Phase 1
NCT02314299 COMPLETED
Diabetic Macular Edema; Age-related Macular Degeneration
Stealth BioTherapeutics Inc.
 2014-11 PHASE1; PHASE2
NCT02388464 COMPLETED
Congestive Heart Failure
Stealth BioTherapeutics Inc.
 2014-07 PHASE1
NCT01572909 COMPLETED
Reperfusion Injury; STEMI
Stealth BioTherapeutics Inc.
 2012-04 PHASE2
NCT01518985 COMPLETED
Healthy Volunteers
Stealth BioTherapeutics Inc.
 2012-01 PHASE1
NCT01115920 COMPLETED
Healthy
Stealth BioTherapeutics Inc.
 2010-05 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-07-13)

Check the Elamipretide (SS-31, MTP-131) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Elamipretide selectively targets cardiolipin within the inner mitochondrial membrane to inhibit cardiolipin peroxidase activity, thereby preventing cytochrome c release and mitigating electron transport chain disruption. By preserving mitochondrial energetic efficiency and limiting reactive oxygen species accumulation, this peptide restores cellular homeostasis and alleviates organ dysfunction in clinically evaluated conditions such as primary mitochondrial disease and chronic heart failure.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.