Peptide LibraryHuman study PLS-00097
Elamipretide in pediatric Barth syndrome: from heart failure to school return.
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 pediatric Barth syndrome: from heart failure to school return.primary · Orphanet journal of rare diseases · 2026-07-07 · PMID 42415111 · DOI
Design and participants
- Design
- Case report
- Population
- A 5-year-old (preschool-aged) boy with genetically confirmed Barth syndrome and advanced heart failure. At start he had severe left ventricular dysfunction and moderate-to-severe mitral regurgitation, depended on gastrostomy feeding and was listed for heart transplantation.
- Conditions
- Barth syndrome, Heart failure, Cardiomyopathy, Mitral regurgitation
- Setting
- Compassionate use program approved by the French Drug Administration (ANSM) (France)
- Planned enrollment (registry)
- Not reported
- Enrolled
- 1
- Randomized
- Not reported
- Completed follow-up
- Not reported
- Funding
- Not stated in the record
- Authors’ conflicts
- Not stated 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 · Daily subcutaneous elamipretide given alongside maximal conventional heart failure therapy · subcutaneous · Six months reported · 1 assigned
What the authors reported
As stated in the sources. This is the authors’ account, not Peptide Library’s assessment.
Left ventricular ejection fraction (LVEF)
unclear outcome · surrogate · Treatment initiation and six months · LVEF (method not stated in abstract)
- Elamipretide: narrative — LVEF improved from 20% at initiation to 50% over six months
Mitral regurgitation severity
unclear outcome · surrogate · Treatment initiation and six months · Graded mitral regurgitation severity
- Elamipretide: narrative — Mitral regurgitation decreased from moderate-to-severe to mild
Ross heart failure class
unclear outcome · patient-important · Treatment initiation and six months · Ross classification of pediatric heart failure
- Elamipretide: narrative — Ross class improved from IV to I
Functional capacity / exercise tolerance
unclear outcome · patient-important · Over six months · Not specified in abstract
- Elamipretide: narrative — Substantial gains in functional capacity were observed
Nutritional status
unclear outcome · surrogate · Over six months · Not specified in abstract
- Elamipretide: narrative — Substantial gains in nutritional status were observed
Health-related quality of life
unclear outcome · patient-important · Over six months · HRQoL instrument not specified in abstract
- Elamipretide: narrative — Substantial gains in health-related quality of life were observed
Return to school
unclear outcome · patient-important · Over six months · School attendance status
- Elamipretide: narrative — The patient returned to full-time school
Heart transplant listing status
unclear outcome · patient-important · Over six months · Transplant waiting list status
- Elamipretide: narrative — The patient remained temporarily suspended from the transplant list
Adverse events / tolerability
unclear outcome · safety · During six months of treatment · Clinical reporting of adverse events
- Elamipretide: narrative — Generally well tolerated; one transient non-serious episode of abdominal pain with moderate pancreatic enzyme elevation resolved without stopping treatment
Adverse events
- Abdominal pain with moderate pancreatic enzyme elevation · Elamipretide: 1 participants
Monitoring: Not described in the abstract beyond the report of a pancreatic enzyme elevation detected during treatment.
The authors concluded (paraphrased): The authors suggest elamipretide may improve cardiac function, exercise tolerance, nutrition and quality of life in preschool-aged children with Barth syndrome and may delay transplantation. They call for broader pediatric evaluation and complementary cardiac rehabilitation.
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 the natural course or other care.
- small or imprecise: Only one patient was treated, so the findings cannot be generalized and their precision cannot be judged.
- confounding: Elamipretide was started alongside maximal conventional heart failure therapy, which could explain the cardiac and functional gains.
- selection bias: A favorable single compassionate-use case selected for publication may not represent typical responses.
- short follow up: Outcomes are reported over six months only; durability and longer-term safety are unknown.
- inappropriate endpoint: Several outcomes are surrogates (LVEF, mitral regurgitation), and functional gains are described without named instruments in the abstract.
- abstract only: Only the abstract is available in the record, so methods, measurement details, funding and conflicts cannot be checked.
Left ventricular ejection fraction (LVEF): 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-patient case report with no control group or comparator. |
|---|---|---|
| Selection of participants | Serious concern | One compassionate-use patient was reported; selection for publication may favor good outcomes. |
| Confounding | Serious concern | Concurrent maximal conventional heart failure therapy could account for the LVEF change. |
| Follow-up and attrition | Some concern | Follow-up of six months reported; no longer-term data in the abstract. |
| Outcome measurement | Not reported | The abstract does not state the imaging method or whether the assessor was blinded. |
| Completeness of reporting | Some concern | Only baseline and six-month values reported in the abstract; intermediate data not shown. |
| Precision | Serious concern | Single patient; no measure of precision is possible. |
| Funding and conflicts of interest | Not reported | Funding and conflicts of interest are not stated in the record. |
An uncontrolled single case with major confounding from concurrent therapy; serious concern 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.
Mitral regurgitation severity: 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-patient case report with no control group or comparator. |
|---|---|---|
| Selection of participants | Serious concern | One compassionate-use patient was reported; selection for publication may favor good outcomes. |
| Confounding | Serious concern | Concurrent maximal heart failure therapy could explain the reduced regurgitation. |
| Follow-up and attrition | Some concern | Six-month follow-up only, as reported in the abstract. |
| Outcome measurement | Not reported | Grading method and assessor blinding are not described in the abstract. |
| Completeness of reporting | Some concern | Qualitative grades only (moderate-to-severe to mild), with no quantitative measures. |
| Precision | Serious concern | Single patient; precision cannot be assessed. |
| Funding and conflicts of interest | Not reported | Funding and conflicts of interest are not stated in the record. |
Uncontrolled single case with confounding and unclear measurement; serious concern.
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.
Ross heart failure class: 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-patient case report with no control group or comparator. |
|---|---|---|
| Selection of participants | Serious concern | Single compassionate-use case likely published because of a good response. |
| Confounding | Serious concern | Concomitant maximal heart failure therapy is a strong confounder of symptom class. |
| Follow-up and attrition | Some concern | Only a six-month period is reported in the abstract. |
| Outcome measurement | Serious concern | Ross class is a clinician-rated subjective scale, unblinded in an open case report. |
| Completeness of reporting | Some concern | Only start and end classes are reported in the abstract. |
| Precision | Serious concern | Single patient; no estimate of precision possible. |
| Funding and conflicts of interest | Not reported | Funding and conflicts of interest are not stated in the record. |
Subjective, unblinded rating in an uncontrolled single case with confounding; serious concern.
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.
Functional capacity / exercise tolerance: 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-patient case report with no control group or comparator. |
|---|---|---|
| Selection of participants | Serious concern | Single compassionate-use case selected for reporting. |
| Confounding | Serious concern | Concurrent heart failure therapy, nutrition support and growth could explain the functional gains. |
| Follow-up and attrition | Some concern | Only six months of follow-up are described in the abstract. |
| Outcome measurement | Not reported | The abstract names no test or instrument for functional capacity. |
| Completeness of reporting | Serious concern | The result is described only as substantial gains, without values. |
| Precision | Serious concern | Single patient with no quantitative result. |
| Funding and conflicts of interest | Not reported | Funding and conflicts of interest are not stated in the record. |
Narrative-only result in an uncontrolled, confounded single case; serious concern.
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.
Nutritional 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-patient case report with no control group or comparator. |
|---|---|---|
| Selection of participants | Serious concern | Single compassionate-use case selected for reporting. |
| Confounding | Serious concern | Gastrostomy feeding and heart failure therapy are concurrent confounders of nutrition. |
| Follow-up and attrition | Some concern | Only six months of follow-up are described in the abstract. |
| Outcome measurement | Not reported | The abstract does not specify how nutritional status was measured. |
| Completeness of reporting | Serious concern | Only narrative substantial gains are reported, with no values. |
| Precision | Serious concern | Single patient with no quantitative result. |
| Funding and conflicts of interest | Not reported | Funding and conflicts of interest are not stated in the record. |
Narrative result with strong confounding in a single case; serious concern.
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.
Health-related 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.
| Control group | Serious concern | Single-patient case report with no control group or comparator. |
|---|---|---|
| Selection of participants | Serious concern | Single compassionate-use case selected for reporting. |
| Confounding | Serious concern | Concurrent therapies and changes in transplant status may affect quality of life. |
| Follow-up and attrition | Some concern | Only six months of follow-up are described in the abstract. |
| Outcome measurement | Serious concern | Subjective outcome, unblinded; the instrument is not named in the abstract. |
| Completeness of reporting | Serious concern | Only narrative substantial gains are reported, without scores. |
| Precision | Serious concern | Single patient with no quantitative result. |
| Funding and conflicts of interest | Not reported | Funding and conflicts of interest are not stated in the record. |
Subjective, unblinded, narrative outcome in a single uncontrolled case; serious concern.
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.
Return to school: 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-patient case report with no control group or comparator. |
|---|---|---|
| Selection of participants | Serious concern | Single compassionate-use case selected for reporting. |
| Confounding | Serious concern | Clinical improvement from concurrent heart failure therapy could explain the school return. |
| Follow-up and attrition | Some concern | Sustained attendance beyond the reported period is unknown. |
| Outcome measurement | Some concern | School return is an objective event, but its timing is not detailed in the abstract. |
| Completeness of reporting | Some concern | Reported narratively in a single abstract sentence. |
| Precision | Serious concern | Single patient; no precision assessable. |
| Funding and conflicts of interest | Not reported | Funding and conflicts of interest are not stated in the record. |
Uncontrolled single case with confounding; serious concern.
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.
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-patient case report with no control group or comparator. |
|---|---|---|
| Selection of participants | Serious concern | Single compassionate-use case selected for reporting. |
| Confounding | Serious concern | Concurrent maximal heart failure therapy could contribute to the delisting decision. |
| Follow-up and attrition | Some concern | Suspension is described as temporary; the longer-term course is unknown. |
| Outcome measurement | Some concern | The listing decision is clinician-driven and its criteria are not described in the abstract. |
| Completeness of reporting | Some concern | Reported narratively with no details on the criteria. |
| Precision | Serious concern | Single patient; no precision assessable. |
| Funding and conflicts of interest | Not reported | Funding and conflicts of interest are not stated in the record. |
Uncontrolled single case with confounding and clinician-driven outcome; serious concern.
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.
Adverse events / 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.
| Control group | Serious concern | No comparator, so adverse events cannot be attributed to elamipretide versus background therapy. |
|---|---|---|
| Selection of participants | Serious concern | Single compassionate-use case; a favorable report may be selectively published. |
| Confounding | Serious concern | Multiple concurrent heart failure drugs could cause abdominal pain or enzyme changes. |
| Follow-up and attrition | Some concern | Six months of exposure only; later adverse events are unknown. |
| Outcome measurement | Not reported | Adverse event ascertainment methods are not described in the abstract. |
| Completeness of reporting | Some concern | Tolerability is summarized as generally well tolerated without a full event listing. |
| Precision | Serious concern | One patient cannot estimate adverse event frequency. |
| Funding and conflicts of interest | Not reported | Funding and conflicts of interest are not stated in the record. |
Single uncontrolled exposure with unclear ascertainment; serious concern for safety inference.
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 comparator) 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: 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
- adverse_events: PMID 42415111, abstract (sentences 10-11) · extracted by AI (automated, not verified by a person) · abstract only
- arms: PMID 42415111, abstract (sentence 7) · extracted by AI (automated, not verified by a person) · abstract only
- author_conclusion: PMID 42415111, abstract (Conclusions, sentences 1-3) · extracted by AI (automated, not verified by a person) · abstract only
- design: PMID 42415111, abstract (sentence 4 (Case presentation)) · extracted by AI (automated, not verified by a person) · abstract only
- limitations: PMID 42415111, abstract (sentences 4-8) · extracted by AI (automated, not verified by a person) · abstract only
- outcomes: PMID 42415111, abstract (sentences 8-11) · extracted by AI (automated, not verified by a person) · abstract only
- participants: PMID 42415111, abstract (sentences 4-6) · extracted by AI (automated, not verified by a person) · abstract only
- population: PMID 42415111, abstract (sentences 4-6) · extracted by AI (automated, not verified by a person) · abstract only
- results: PMID 42415111, abstract (sentences 8-9) · 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).