Human study PLS-00110

Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome.

Retrospective cohort · 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.

Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome.primary · Orphanet journal of rare diseases · 2022-09-02 · PMID 36056411 · DOI

Design and participants

Design: Retrospective cohort
Population: Patients with Barth syndrome: 8 elamipretide-treated patients from the TAZPOWER open-label extension and 19 untreated natural history controls, 12 of whom had serial echocardiographic assessments.
Conditions: Barth syndrome
Planned enrollment (registry): Not reported
Enrolled: Not reported
Randomized: Not reported
Completed follow-up: Not reported
Funding: Not stated in the abstract. PubMed publication type lists Research Support, Non-U.S. Gov't.
Authors’ conflicts: Not stated in the abstract.

Intervention and comparator

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

Elamipretide (TAZPOWER OLE) (intervention) · elamipretide · Elamipretide given daily in the open-label extension of the TAZPOWER trial · Assessed at weeks 64 and 76 of continuous treatment · 8 assigned
Natural history controls (comparator) · Untreated patients with Barth syndrome forming a natural history control cohort, compared by propensity score model · historical · 19 assigned

What the authors reported

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

6-minute walk test distance

primary outcome · patient-important · Weeks 64 and 76 · 6-minute walk test distance in metres, least squares mean difference between groups

Elamipretide (TAZPOWER OLE) vs Natural history controls: mean difference 79.7, p = 0.0004 — LS mean difference of 79.7 m at week 64 in favor of elamipretide
Elamipretide (TAZPOWER OLE) vs Natural history controls: mean difference 91, p = 0.0005 — LS mean difference of 91.0 m at week 76 in favor of elamipretide

Muscle strength by handheld dynamometry

secondary outcome · patient-important · Weeks 64 and 76 · Handheld dynamometry in Newtons, least squares mean difference between groups

Elamipretide (TAZPOWER OLE) vs Natural history controls: mean difference 40.8, p = 0.0002 — LS mean difference of 40.8 N at 64 weeks favoring elamipretide
Elamipretide (TAZPOWER OLE) vs Natural history controls: mean difference 56.7, p = 0.0005 — LS mean difference of 56.7 N at 76 weeks favoring elamipretide

5 times sit-to-stand

secondary outcome · patient-important · 5 times sit-to-stand test

Elamipretide (TAZPOWER OLE) vs Natural history controls: narrative — Statistically significant improvement with continuous elamipretide treatment

Multi-domain responder index

secondary outcome · patient-important · Multi-domain responder index (MDRI)

Elamipretide (TAZPOWER OLE) vs Natural history controls: narrative — Statistically significant improvement with continuous elamipretide treatment

Left ventricular stroke volume

unclear outcome · surrogate · Echocardiographic left ventricular stroke volume change from baseline

Elamipretide (TAZPOWER OLE) vs Natural history controls: narrative — Stroke volume increased from baseline with elamipretide but decreased in natural history controls

Adverse events

None reported. That is not evidence of safety.

Monitoring: Not reported in the abstract.

The authors concluded (paraphrased): The study established a natural history control cohort for Barth syndrome. Results suggest elamipretide may improve the natural history of the disease, partly by attenuating the decline in heart function, and may give meaningful gains in heart function and functional capacity versus controls.

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

confounding: Nonrandomized comparison with a retrospective natural history cohort. A propensity score model cannot remove unmeasured confounding.
selection bias: Treated patients came from a trial open-label extension, while controls were assembled retrospectively. The groups may differ systematically.
small or imprecise: Only 8 treated patients and 19 controls were analyzed, and only 12 controls had serial echocardiograms.
inadequate blinding: Treatment was open-label, and effort-dependent tests such as the 6MWT and dynamometry are prone to expectation effects.
inappropriate endpoint: The cardiac finding relies on stroke volume, an imaging surrogate, and is reported only narratively.
abstract only: Only the abstract was available. Methods, missing data, adverse events and conflicts could not be assessed.

6-minute walk test distance: No information (ROBINS-I)