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Study · Not a test of the peptide alone · 2023

1 result

Identifying responders to elamipretide in Barth syndrome: Hierarchical clustering for time series data.

Van den Eynde J, Chinni B, Vernon H, Thompson WR, Hornby B, Kutty S, Manlhiot C

Orphanet journal of rare diseases11 Apr 2023Cited by 3 (Europe PMC)

PMID37041653 (opens in a new tab)PMCIDPMC10088720 (opens in a new tab)DOI10.1186/s13023-023-02676-8 (opens in a new tab)PepBankPUB-37041653
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Summary

BackgroundBarth syndrome (BTHS) is a rare genetic disease that is characterized by cardiomyopathy, skeletal myopathy, neutropenia, and growth abnormalities and often leads to death in childhood. Recently, elamipretide has been tested as a potential first disease-modifying drug. This study aimed to identify patients with BTHS who may respond to elamipretide, based on continuous physiological measurements acquired through wearable devices.ResultsData from a randomized, double-blind, placebo-controlled crossover trial of 12 patients with BTHS were used, including physiological time series data measured using a wearable device (heart rate, respiratory rate, activity, and posture) and functional scores. The latter included the 6-minute walk test (6MWT), Patient-Reported Outcomes Measurement Information System (PROMIS) fatigue score, SWAY Balance Mobile Application score (SWAY balance score), BTHS Symptom Assessment (BTHS-SA) Total Fatigue score, muscle strength by handheld dynamometry, 5 times sit-and-stand test (5XSST), and monolysocardiolipin to cardiolipin ratio (MLCL:CL). Groups were created through median split of the functional scores into "highest score" and "lowest score", and "best response to elamipretide" and "worst response to elamipretide". Agglomerative hierarchical clustering (AHC) models were implemented to assess whether physiological data could classify patients according to functional status and distinguish non-responders from responders to elamipretide. AHC models clustered patients according to their functional status with accuracies of 60-93%, with the greatest accuracies for 6MWT (93%), PROMIS (87%), and SWAY balance score (80%). Another set of AHC models clustered patients with respect to their response to treatment with elamipretide with perfect accuracy (all 100%).ConclusionsIn this proof-of-concept study, we demonstrated that continuously acquired physiological measurements from wearable devices can be used to predict functional status and response to treatment among patients with BTHS.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study1

No result from this study comes from a test of the peptide.

Studies that did not test the peptide alone1

In this study, Elamipretide (SS-31) was a lab tool or a carrier, part of a mix, or the subject of a lab method. These results are not evidence about taking Elamipretide (SS-31).

  1. 01HumanNot a test of Elamipretide (SS-31) aloneRandomized controlled trialOther

    In patients with Barth syndrome from a crossover trial, clustering models using wearable-device data classified response to elamipretide with perfect accuracy.

    In plain wordsIn people with Barth syndrome, a computer model using wearable-device data separated responders from non-responders to elamipretide with perfect accuracy in a small study.

    Peptide
    Elamipretide (SS-31)
    Outcome
    Not applicableClassification of responders to elamipretide from wearable data
    Study

    n = 12

    Outcome group
    Overall treatment effect · Whole body

    Measurements

    Classification accuracy
    100%
    response to elamipretide, AHC models

    Source sentences

    Summary
    Another set of AHC models clustered patients with respect to their response to treatment with elamipretide with perfect accuracy (all 100%).
    1 context sentence
    Summary · context
    Data from a randomized, double-blind, placebo-controlled crossover trial of 12 patients with BTHS were used, including physiological time series data measured using a wearable device (heart rate, respiratory rate, activity, and posture) and functional scores.

    In this study, Elamipretide (SS-31) was a lab tool or a carrier, part of a mix, or the subject of a lab method. This result is not evidence about taking Elamipretide (SS-31).

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

  1. Version 2Current

    9 Oct 2026, 18:17 UTC

    Study results updated

  2. Version 1

    9 Oct 2026, 02:47 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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