Human study PLS-00103

Expanded-access use of elamipretide in a patient with membrane protein-associated neurodegeneration.

Case report · 1 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 in a patient with membrane protein-associated neurodegeneration.primary · Clinical case reports · 2024-06-24 · PMID 38919884 · DOI

Design and participants

Design: Case report
Population: A single progressively declining 17-year-old female patient with membrane protein-associated neurodegeneration (MPAN)
Conditions: Membrane protein-associated neurodegeneration
Setting: Expanded-access (compassionate) use
Planned enrollment (registry): Not reported
Enrolled: 1
Randomized: Not reported
Completed follow-up: Not reported
Funding: Not stated
Authors’ conflicts: Not stated

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 · 1 assigned

What the authors reported

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

Symptoms (dysarthria, dysphagia, gait)

unclear outcome · patient-important · During elamipretide treatment · Symptomatic assessment; method not stated in abstract

Elamipretide: narrative — Symptomatic improvements in dysarthria, dysphagia and gait during treatment

6-minute walk test

unclear outcome · patient-important · During elamipretide treatment · 6-minute walk test

Elamipretide: narrative — Objective improvement in the 6-minute walk test during treatment

5 times sit-to-stand test

unclear outcome · patient-important · During elamipretide treatment · 5 times sit-to-stand test

Elamipretide: narrative — Objective improvement in the 5 times sit-to-stand test during treatment

Adverse events

None reported. That is not evidence of safety.

Monitoring: Not reported in the available abstract

The authors concluded (paraphrased): The authors report that a progressively declining 17-year-old with MPAN showed symptomatic improvements in dysarthria, dysphagia and gait, and objective improvements in walking and sit-to-stand tests, during elamipretide treatment.

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-patient case report with no comparator, so improvement cannot be attributed to elamipretide.
small or imprecise: Only one patient was treated, so the findings cannot be generalized and give no precision.
abstract only: Only a one-sentence abstract is available. Dosing, duration, magnitude of change and safety data are not reported.
inadequate blinding: Symptom assessments and effort-dependent function tests were done open-label in an expanded-access setting.
selective reporting: The abstract reports only improvements. Adverse events and any outcomes that did not improve are not described.
inappropriate population: MPAN is a rare neurodegeneration, unlike Barth syndrome or primary mitochondrial myopathy in the claims.

Symptoms (dysarthria, dysphagia, gait): 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	Single case report with no control or comparison condition described in the abstract.
Selection of participants	Serious concern	Single patient selected for expanded access, likely reported because of a perceived benefit.
Confounding	Serious concern	Natural fluctuation, co-therapies and placebo effects cannot be separated from any drug effect in one patient.
Follow-up and attrition	Not reported	Treatment duration and follow-up length are not stated in the one-sentence abstract.
Outcome measurement	Serious concern	Symptomatic improvements are subjective and were assessed unblinded; the method is not described.
Completeness of reporting	Not reported	Abstract only; no prespecified outcomes or full results are available to check reporting.
Precision	Serious concern	n=1 and no quantitative values reported, so no estimate of precision is possible.
Funding and conflicts of interest	Not reported	Funding and conflicts of interest are not stated in the record.

Uncontrolled single-patient report with subjective 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.

6-minute walk test: 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; single-patient case report described in the abstract.
Selection of participants	Serious concern	Single expanded-access patient; selection for publication likely favours positive findings.
Confounding	Serious concern	Practice effects, motivation and natural course cannot be separated from drug effect in one case.
Follow-up and attrition	Not reported	Duration of treatment and timing of the walk test are not stated in the abstract.
Outcome measurement	Serious concern	Effort-dependent test assessed open-label, so it is susceptible to expectation and encouragement effects.
Completeness of reporting	Not reported	Abstract gives no values or prespecified analysis for the walk test.
Precision	Serious concern	Single patient and no numeric change reported, so precision cannot be assessed.
Funding and conflicts of interest	Not reported	Funding and conflicts of interest are not stated in the record.

Single uncontrolled case with an effort-dependent unblinded test and no numeric results in the abstract.

5 times sit-to-stand test: 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; single-patient case report described in the abstract.
Selection of participants	Serious concern	Single expanded-access patient; selection for publication likely favours positive findings.
Confounding	Serious concern	Practice effects and natural course cannot be separated from drug effect in one case.
Follow-up and attrition	Not reported	Duration of treatment and timing of the test are not stated in the abstract.
Outcome measurement	Serious concern	Effort-dependent performance test assessed open-label, so it is susceptible to expectation bias.
Completeness of reporting	Not reported	Abstract gives no values or prespecified analysis for the sit-to-stand test.
Precision	Serious concern	Single patient and no numeric change reported, so precision cannot be assessed.
Funding and conflicts of interest	Not reported	Funding and conflicts of interest are not stated in the record.

Single uncontrolled case with an unblinded performance test and no numeric results in the abstract.

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: not eligible (insufficient data) for “Adverse events, including injection-site reactions, and discontinuations”

Where each value comes from

arms: PMID 38919884, abstract (sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
author_conclusion: PMID 38919884, abstract (sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
design: PMID 38919884, title (title) · extracted by AI (automated, not verified by a person) · abstract only
limitations: PMID 38919884, abstract (sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
outcomes: PMID 38919884, abstract (sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
participants: PMID 38919884, abstract (sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
population: PMID 38919884, abstract (sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
results: PMID 38919884, abstract (sentence 1) · 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).