Human study PLS-00114

Phase 1 Clinical Trial of Elamipretide in Dry Age-Related Macular Degeneration and Noncentral Geographic Atrophy: ReCLAIM NCGA Study.

Single-arm trial · 19 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.

Phase 1 Clinical Trial of Elamipretide in Dry Age-Related Macular Degeneration and Noncentral Geographic Atrophy: ReCLAIM NCGA Study.primary · Ophthalmology science · 2021-11-27 · PMID 36246181 · DOI

Design and participants

Design: Single-arm trial
Population: Adults aged 55 years or older with dry age-related macular degeneration and noncentral geographic atrophy
Conditions: Dry age-related macular degeneration, Noncentral geographic atrophy
Setting: Single-center, open-label phase 1 clinical trial with 24 weeks of treatment
Planned enrollment (registry): Not reported
Enrolled: 19
Randomized: 0
Completed follow-up: 15

Intervention and comparator

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

Elamipretide (intervention) · elamipretide · Elamipretide (mitochondria-targeted peptide) given daily by subcutaneous administration · subcutaneous · 24 weeks · 19 assigned

What the authors reported

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

Safety and tolerability (adverse events)

primary outcome · safety · Throughout 24 weeks · Adverse events assessed throughout the study, graded by severity

Elamipretide: narrative — All 19 participants had at least one nonocular AE; all AEs were mild or moderate, and no serious AEs occurred
Elamipretide: proportion — AEs were mild in 73.7%
Elamipretide: proportion — AEs were moderate in 26.3%

Normal-luminance best-corrected visual acuity (BCVA)

exploratory outcome · patient-important · Week 24 · Letters read, change from baseline

Elamipretide: change from baseline 4.6, p = 0.0032 — Mean change of +4.6 (SD 5.1) letters from baseline to week 24 among completers

Low-luminance best-corrected visual acuity (LLBCVA)

exploratory outcome · patient-important · Week 24 · Letters read under low luminance, change from baseline

Elamipretide: change from baseline 5.4, p = 0.0245 — Mean change of +5.4 (SD 7.9) letters among completers

Low-luminance binocular reading acuity (LLBRA)

exploratory outcome · patient-important · Week 24 · logMAR units, change from baseline

Elamipretide: change from baseline -0.52, p = 0.005 — Mean change of -0.52 (SD 0.75) logMAR; the abstract labels this value LLBCVA although it is given in logMAR units in the reading-acuity sentence

Normal-luminance binocular reading acuity (NLBRA)

exploratory outcome · patient-important · Week 24 · Reading acuity, change from baseline

Elamipretide: narrative — Minimal change in NLBRA

Geographic atrophy area

exploratory outcome · surrogate · Week 24 · Square-root-transformed GA area by fundus autofluorescence and by spectral-domain OCT

Elamipretide: change from baseline 0.14 — Mean change of 0.14 (SD 0.08) mm by FAF
Elamipretide: change from baseline 0.13 — Mean change of 0.13 (SD 0.14) mm by OCT

Low-luminance questionnaire (LLQ) self-reported function

exploratory outcome · patient-important · Week 24 · Patient-reported low-luminance questionnaire

Elamipretide: narrative — Improvement observed in the dim light reading and general dim light vision domains

Adverse events

Any nonocular adverse event · Elamipretide: 19 participants
Serious adverse events (serious) · Elamipretide: 0 participants
Conversion to neovascular AMD leading to study exit · Elamipretide: 1 participants
Intolerable injection site reaction leading to study exit · Elamipretide: 1 participants

Monitoring: Safety and tolerability were assessed throughout the 24-week study. Adverse events were graded by severity (mild or moderate) and serious adverse events were recorded.

The authors concluded (paraphrased): Elamipretide appeared well tolerated without serious adverse events in dry AMD with noncentral GA. Exploratory analyses suggested a possible positive effect on visual function, especially under low luminance, and a phase 2b trial is underway.

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

no control group: Single-arm open-label design with no placebo group, so visual changes cannot be attributed to elamipretide rather than learning, regression or natural variation.
inadequate blinding: Open-label study; visual acuity, reading tests and questionnaires were assessed with participants and staff aware of treatment.
small or imprecise: Only 19 participants treated and 15 analyzed for efficacy outcomes, giving imprecise estimates.
incomplete follow up: Four of 19 participants left the study, and efficacy analyses were restricted to completers.
short follow up: Treatment and follow-up lasted only 24 weeks, which is short for a progressive atrophic disease.
abstract only: Only the abstract was available; funding, conflicts and full methods could not be checked.
other: The abstract appears to mislabel the low-luminance reading acuity result as LLBCVA, which creates ambiguity about that outcome.
inappropriate population: Participants had dry AMD with noncentral GA, not Barth syndrome or mitochondrial myopathy.

Safety and tolerability (adverse events): Serious concern (Structured limitations appraisal)