Peptide Library

Human study PLS-00108

Use of Elamipretide in patients assigned treatment in the compassionate use program: Case series in pediatric patients with rare orphan diseases.

Case series · Not reported 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.

  • Use of Elamipretide in patients assigned treatment in the compassionate use program: Case series in pediatric patients with rare orphan diseases.primary · JIMD reports · 2022-09-21 · PMID 36636586 · DOI

Design and participants

Design
Case series
Population
Pediatric patients aged under 12 years with rare mitochondrial (orphan) disorders treated with elamipretide under a compassionate use program; the specific diagnoses are not stated in the abstract.
Conditions
Mitochondrial disorders, Rare orphan diseases
Setting
Compassionate use program; setting not otherwise stated
Planned enrollment (registry)
Not reported
Enrolled
Not reported
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 compassionate use (intervention) · elamipretide · Elamipretide given under a compassionate use program · Not reported assigned

What the authors reported

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

Safety of elamipretide treatment

unclear outcome · safety

  • No results reported.

Efficacy of elamipretide treatment (clinical response)

unclear outcome · patient-important

  • No results reported.

Adverse events

None reported. That is not evidence of safety.

Monitoring: Not stated in the abstract; the authors say the case reports address safety.

The authors concluded (paraphrased): The authors present these as the first case reports on the safety and efficacy of elamipretide in children under 12 with mitochondrial disorders. They describe its use to provide dosing parameters for this age group.

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: Case series of compassionate use patients with no comparison group, so any effect cannot be attributed to elamipretide.
  • abstract only: Only the abstract is available, and it reports no results, patient numbers, adverse events or follow-up duration.
  • selection bias: Patients were selected for compassionate use treatment, not enrolled consecutively or by defined criteria.
  • small or imprecise: This is a case series of rare-disease patients; the number of patients is not stated, so precision cannot be assessed.
  • inappropriate population: Children under 12 with unspecified mitochondrial disorders differ from the adult primary mitochondrial myopathy and Barth syndrome populations in the claims.

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

Claims this study informs

Where each value comes from

  • arms: PMID 36636586, title (title) · extracted by AI (automated, not verified by a person) · abstract only
  • author_conclusion: PMID 36636586, abstract (sentences 4-5) · extracted by AI (automated, not verified by a person) · abstract only
  • design: PMID 36636586, title (title) · extracted by AI (automated, not verified by a person) · abstract only
  • limitations: PMID 36636586, abstract (sentences 1-5) · extracted by AI (automated, not verified by a person) · abstract only
  • outcomes: PMID 36636586, abstract (sentence 4) · extracted by AI (automated, not verified by a person) · abstract only
  • population: PMID 36636586, abstract (sentences 4-5) · 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).