Peptide Library

Human study PLS-00229

A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy

Other design · 12 participants enrolled

Version 1 · published November 8, 2026 · evidence through November 8, 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.

Reports of this study

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

  • A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathyregistry entry · NCT02693119 (COMPLETED, results posted, planned enrollment 12)

Design and participants

Design
Other design
Population
People with Leber's hereditary optic neuropathy
Conditions
Leber's Hereditary Optic Neuropathy
Planned enrollment (registry)
12
Enrolled
12
Randomized
Not reported
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 topical ophthalmic solution (intervention) · elamipretide · Elamipretide (MTP-131, Bendavia) topical ophthalmic solution · topical ophthalmic · Not reported assigned
  • Vehicle topical ophthalmic solution (comparator) · Vehicle topical ophthalmic solution without elamipretide · placebo · topical ophthalmic · Not reported assigned

What the authors reported

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

Safety and tolerability

unclear outcome · safety

  • No results reported.

Efficacy (unspecified)

unclear outcome · patient-important

  • No results reported.

Adverse events

None reported. That is not evidence of safety.

Monitoring: The title states that safety and tolerability were investigated. No monitoring methods are described in the record.

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

  • abstract only: Only the registry entry is available. No outcome definitions, result values or adverse event data appear in the record.
  • small or imprecise: Actual enrollment was 12 participants, which is too few to give precise estimates of safety or efficacy.
  • inadequate randomization: The record lists a vehicle comparator but does not state whether allocation was randomized or masked.
  • selective reporting: The registry flags results as available, but none are shown here, so complete reporting cannot be verified.
  • other: Only registry metadata is available. No outcomes, results, allocation method, or adverse event data appear in the record, so the study cannot be appraised in detail.
  • inappropriate population: The population has Leber's hereditary optic neuropathy and the route is topical ocular, which differs from the populations and systemic use in the claim questions.

No risk-of-bias assessment has been published for this study’s results.

Claims this study informs

Where each value comes from

  • arms: NCT NCT02693119, registry (interventions field) · extracted by AI (automated, not verified by a person)
  • design: NCT NCT02693119, registry (phases, study_type and interventions fields) · extracted by AI (automated, not verified by a person)
  • limitations: NCT NCT02693119, registry (enrollment and has_results fields) · extracted by AI (automated, not verified by a person)
  • outcomes: NCT NCT02693119, title (study title) · extracted by AI (automated, not verified by a person)
  • participants: NCT NCT02693119, registry (enrollment and enrollment_type fields) · extracted by AI (automated, not verified by a person)
  • population: NCT NCT02693119, registry (conditions field) · extracted by AI (automated, not verified by a person)

  • Version 1 · published November 8, 2026 · evidence through November 8, 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).