Peptide LibraryHuman study PLS-00102
Elamipretide in the Management of Barth Syndrome: Current Evidence and a Case Report.
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.
- Elamipretide in the Management of Barth Syndrome: Current Evidence and a Case Report.primary · Molecular genetics and metabolism · 2025-08-11 · PMID 40816230 · DOI
Design and participants
- Design
- Case report
- Population
- A single neonate with prenatally identified Barth syndrome. The infant had severe left ventricular non-compaction cardiomyopathy and clinical heart failure, and was on the heart transplant list.
- Conditions
- Barth syndrome, Left ventricular non-compaction cardiomyopathy, Heart failure
- Planned enrollment (registry)
- Not reported
- Enrolled
- 1
- Randomized
- Not reported
- Completed follow-up
- Not reported
- Funding
- Not reported
- Authors’ conflicts
- Not reported
Intervention and comparator
What each group received. Related peptides, analogs and combinations are never treated as the compound itself.
- Elamipretide (intervention) · elamipretide · Elamipretide (ELAM) started shortly after birth for clinical heart failure. · Initiated shortly after birth; total duration not stated · 1 assigned
What the authors reported
As stated in the sources. This is the authors’ account, not Peptide Library’s assessment.
Clinical heart failure status and heart transplant listing status
unclear outcome · patient-important · Not stated (described as sustained) · Narrative clinical course, including change in heart transplant list status
- Elamipretide: narrative — Treatment was associated with significant, sustained clinical improvement, leading to inactive transplant-list status with anticipated delisting.
Adverse events
None reported. That is not evidence of safety.
Monitoring: Not reported in the abstract.
The authors concluded (paraphrased): In a neonate with Barth syndrome-related severe LV non-compaction cardiomyopathy, elamipretide started shortly after birth was associated with significant, sustained clinical improvement. The patient became inactive on the heart transplant list, with delisting anticipated.
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 case report with no comparator, so improvement cannot be attributed to elamipretide rather than natural course or other care.
- small or imprecise: Only one patient was treated, so the findings cannot be generalized or quantified.
- confounding: Concurrent heart failure management is not described in the abstract and could explain the clinical improvement.
- abstract only: Only the abstract is available. Dosing details, follow-up length, measurements and adverse events are not reported.
- selective reporting: Favorable single cases are more likely to be published. No safety outcomes are reported in the abstract.
- inappropriate endpoint: The outcome is a narrative clinical judgment and transplant-list status, not standardized function measures.
Clinical heart failure status and heart transplant listing status: 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.
| Control group | Serious concern | Single case report with no control or comparison group described in the abstract. |
|---|---|---|
| Selection of participants | Serious concern | One selected patient is described; it is unclear how or why this case was chosen for publication. |
| Confounding | Serious concern | Other heart failure treatments and the natural disease course are not described and could account for the improvement. |
| Follow-up and attrition | Not reported | The abstract describes improvement as sustained but does not state the follow-up duration. |
| Outcome measurement | Serious concern | Improvement is a narrative, unblinded clinical judgment plus transplant-list status, with no standardized measure reported. |
| Completeness of reporting | Not reported | Only the abstract is available. Measures, timepoints and full results are not reported. |
| Precision | Serious concern | A single patient gives no basis for estimating an effect or its uncertainty. |
| Funding and conflicts of interest | Not reported | Funding and conflicts of interest are not reported in the available record. |
Uncontrolled single case with unaddressed confounding and narrative outcome assessment, and only an abstract available. These give serious concerns overall.
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.
Claims this study informs
- Improves function in Barth syndrome: not eligible (wrong outcome) 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 40816230, abstract (sentence 5) · extracted by AI (automated, not verified by a person) · abstract only
- author_conclusion: PMID 40816230, abstract (sentence 5) · extracted by AI (automated, not verified by a person) · abstract only
- design: PMID 40816230, abstract (sentence 5) · extracted by AI (automated, not verified by a person) · abstract only
- limitations: PMID 40816230, abstract (sentences 5-6) · extracted by AI (automated, not verified by a person) · abstract only
- outcomes: PMID 40816230, abstract (sentence 5) · extracted by AI (automated, not verified by a person) · abstract only
- participants: PMID 40816230, abstract (sentence 5) · extracted by AI (automated, not verified by a person) · abstract only
- population: PMID 40816230, abstract (sentence 5) · extracted by AI (automated, not verified by a person) · abstract only
- results: PMID 40816230, abstract (sentence 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).