Human study PLS-00098

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 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.

Expanded-Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series.primary · Clinical case reports · 2024-11-29 · PMID 39619320 · DOI

Design and participants

Design: Case series
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.
Conditions: Chronic progressive external ophthalmoplegia plus, Neuropathy, ataxia, and retinitis pigmentosa (NARP) syndrome, Mitochondrial disease
Setting: Expanded-access (compassionate) use
Planned enrollment (registry): Not reported
Enrolled: 2
Randomized: Not reported
Completed follow-up: Not reported
Funding: Not reported in the record.
Authors’ conflicts: Not reported in the record.

Intervention and comparator

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

Elamipretide (intervention) · elamipretide · Elamipretide given under expanded access · 2 assigned

What the authors reported

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

Symptoms and quality of life

unclear outcome · patient-important · While on therapy · Not specified in the abstract; the title refers to quality of life.

Elamipretide: narrative — Both patients showed symptom improvement while on therapy

Tolerability

unclear outcome · safety · While on therapy · Not specified

Elamipretide: narrative — Elamipretide was well tolerated

Adverse events

None reported. That is not evidence of safety.

Monitoring: Not described in the abstract.

The authors concluded (paraphrased): The authors report that elamipretide was well tolerated and that both patients with CPEO plus or NARP improved in symptoms while on therapy.

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: Uncontrolled case series with no comparator, so improvement cannot be attributed to elamipretide.
small or imprecise: Only two patients were treated, each with a different disorder, so results are highly imprecise.
abstract only: Only the abstract was available: no outcome measures, timepoints, doses or adverse event details.
selection bias: Expanded-access cases chosen for publication may be those with favourable responses.
inappropriate endpoint: Symptom improvement is described only narratively; no validated measures are named in the abstract.
other: Symptom and quality-of-life improvement was probably assessed without blinding and may be subject to expectation effects.

Symptoms and quality of life: Serious concern (Structured limitations appraisal)

For uncontrolled studies, case series and case reports, where no validated comparative tool applies. It lists limitations; it is not a risk-of-bias rating.

Domain judgments
Control group	Serious concern	No control group: two single patients treated under expanded access.
Selection of participants	Serious concern	Expanded-access patients reported as a case series; selection for publication is likely.
Confounding	Serious concern	No adjustment is possible; concurrent care and natural fluctuation are not addressed in the abstract.
Follow-up and attrition	Not reported	Duration of treatment and follow-up are not stated in the abstract.
Outcome measurement	Serious concern	Symptom and quality-of-life improvement are self-reported or unblinded, and the instruments are not stated.
Completeness of reporting	Some concern	Results are given only narratively in the abstract, with no quantitative data.
Precision	Serious concern	Two patients only; no estimates or variability are reported.
Funding and conflicts of interest	Not reported	Funding and conflicts of interest are not given in the record.

Uncontrolled two-patient case series with narrative, unblinded outcomes and abstract-only information.

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

Tolerability: Serious concern (Structured limitations appraisal)

For uncontrolled studies, case series and case reports, where no validated comparative tool applies. It lists limitations; it is not a risk-of-bias rating.

Domain judgments
Control group	Serious concern	No comparator group to contrast adverse event rates against.
Selection of participants	Serious concern	Two expanded-access cases selected for reporting; not representative.
Confounding	Some concern	Attribution of any events to elamipretide versus underlying disease is not addressed in the abstract.
Follow-up and attrition	Not reported	The length of exposure and observation is not stated in the abstract.
Outcome measurement	Not reported	How adverse events were collected or graded is not described in the abstract.
Completeness of reporting	Not reported	Only a summary statement of tolerability; no event details are available.
Precision	Serious concern	Two patients cannot detect or exclude uncommon adverse events.
Funding and conflicts of interest	Not reported	Funding and conflicts are not reported in the record.

Safety rests on a narrative statement in two uncontrolled patients, with no adverse event details.

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 outcome) 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

arms: PMID 39619320, abstract (sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
author_conclusion: PMID 39619320, abstract (sentence 2) · extracted by AI (automated, not verified by a person) · abstract only
design: PMID 39619320, title (title) · extracted by AI (automated, not verified by a person) · abstract only
limitations: PMID 39619320, abstract (sentences 1-2) · extracted by AI (automated, not verified by a person) · abstract only
outcomes: PMID 39619320, abstract (sentence 2) · extracted by AI (automated, not verified by a person) · abstract only
participants: PMID 39619320, abstract (sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
population: PMID 39619320, abstract (sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
results: PMID 39619320, abstract (sentence 2) · 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).