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Study · Randomized controlled trial · n = 12 · 2021

7 results

A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism.

Reid Thompson W, Hornby B, Manuel R, Bradley E, Laux J, Carr J, Vernon HJ

Genetics in medicine : official journal of the American College of Medical Genetics20 Oct 2020Cited by 112 (Europe PMC)

PMID33077895 (opens in a new tab)PMCIDPMC7935714 (opens in a new tab)DOI10.1038/s41436-020-01006-8 (opens in a new tab)PepBankPUB-33077895
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Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: CC BY-NC-ND. Results still quote short attributed sentences.

Results from this study7

7 results from studies that gave the peptide. 7 results from people.

  1. 01HumanRandomized controlled trialEfficacy outcome

    In people with Barth syndrome, elamipretide did not meet the primary 6-minute walk test endpoint versus placebo in the crossover part 1.

    In plain wordsIn people with Barth syndrome, elamipretide did not improve the 6-minute walk more than placebo in the first part of the trial.

    Peptide
    Elamipretide (SS-31)
    Outcome
    No significant change6-minute walk test in part 1
    Study

    n = 12

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Exercise and physical function · Musculoskeletal
    Studied dose
    40 mg per day · 12 weeksAs given in this study (human).

    Source sentences

    Summary
    In part 1 neither primary endpoint was met.
    1 context sentence
    Summary · context
    In part 1, 12 subjects were randomized to 40 mg per day of elamipretide or placebo for 12 weeks, followed by a 4-week washout and then 12 weeks on the opposite arm.
  2. 02HumanRandomized controlled trialEfficacy outcome

    In people with Barth syndrome, elamipretide did not meet the primary BTHS Symptom Assessment endpoint versus placebo in the crossover part 1.

    In plain wordsIn people with Barth syndrome, elamipretide did not improve symptom scores more than placebo in the first part of the trial.

    Peptide
    Elamipretide (SS-31)
    Outcome
    No significant changeBTHS Symptom Assessment scale in part 1
    Study

    n = 12

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Patient-reported outcomes · Musculoskeletal
    Studied dose
    40 mg per day · 12 weeksAs given in this study (human).

    Source sentences

    Summary
    In part 1 neither primary endpoint was met.
    1 context sentence
    Summary · context
    In part 1, 12 subjects were randomized to 40 mg per day of elamipretide or placebo for 12 weeks, followed by a 4-week washout and then 12 weeks on the opposite arm.
  3. 03HumanNon-randomized trialEfficacy outcome

    In people with Barth syndrome, 6-minute walk distance improved significantly at 36 weeks of open-label elamipretide.

    In plain wordsIn people with Barth syndrome, walking distance improved after 36 weeks of open-label elamipretide, with no placebo comparison.

    Peptide
    Elamipretide (SS-31)
    Outcome
    Increase6-minute walk test at 36 weeks (part 2)
    Study

    n = 8

    Analysis
    Primary
    Outcome group
    Exercise and physical function · Musculoskeletal
    Studied dose
    40 mg per day · 36 weeksAs given in this study (human).

    Measurements

    6MWT change
    95.9 m
    36 weeks, part 2

    Source sentences

    Summary
    At 36 weeks in part 2, there were significant improvements in 6MWT (+95.9 m, p = 0.024) and BTHS-SA (-2.1 points, p = 0.031).
    1 context sentence
    Summary · context
    Ten subjects continued on the open-label extension (part 2) of 40 mg per day of elamipretide, with eight subjects reaching 36 weeks.
  4. 04HumanNon-randomized trialEfficacy outcome

    In people with Barth syndrome, BTHS Symptom Assessment scores improved significantly at 36 weeks of open-label elamipretide.

    In plain wordsIn people with Barth syndrome, symptom scores improved after 36 weeks of open-label elamipretide, with no placebo comparison.

    Peptide
    Elamipretide (SS-31)
    Outcome
    DecreaseBTHS Symptom Assessment at 36 weeks (part 2)
    Study

    n = 8

    Analysis
    Primary
    Outcome group
    Patient-reported outcomes · Musculoskeletal
    Studied dose
    40 mg per day · 36 weeksAs given in this study (human).

    Measurements

    BTHS-SA change
    −2.1 points
    36 weeks, part 2

    Source sentences

    Summary
    At 36 weeks in part 2, there were significant improvements in 6MWT (+95.9 m, p = 0.024) and BTHS-SA (-2.1 points, p = 0.031).
    1 context sentence
    Summary · context
    Ten subjects continued on the open-label extension (part 2) of 40 mg per day of elamipretide, with eight subjects reaching 36 weeks.
  5. 05HumanNon-randomized trialEfficacy outcome

    In people with Barth syndrome, open-label elamipretide was followed by significant improvement in knee extensor strength.

    In plain wordsIn people with Barth syndrome, knee muscle strength improved during open-label elamipretide, with no placebo comparison.

    Peptide
    Elamipretide (SS-31)
    Outcome
    IncreaseKnee extensor strength
    Study

    n: not reported in source

    Analysis
    Secondary
    Outcome group
    Exercise and physical function · Musculoskeletal
    Studied dose
    40 mg per dayAs given in this study (human).

    Source sentences

    Summary
    There were also significant improvements in secondary endpoints including knee extensor strength, patient global impression of symptoms, and some cardiac parameters.
    1 context sentence
    Summary · context
    Ten subjects continued on the open-label extension (part 2) of 40 mg per day of elamipretide, with eight subjects reaching 36 weeks.
  6. 06HumanNon-randomized trialEfficacy outcome

    In people with Barth syndrome, open-label elamipretide was followed by significant improvement in patient global impression of symptoms.

    In plain wordsIn people with Barth syndrome, patients rated their overall symptoms as improved during open-label elamipretide, with no placebo comparison.

    Peptide
    Elamipretide (SS-31)
    Outcome
    Not applicablePatient global impression of symptoms
    Study

    n: not reported in source

    Analysis
    Secondary
    Outcome group
    Patient-reported outcomes · Musculoskeletal
    Studied dose
    40 mg per dayAs given in this study (human).

    Source sentences

    Summary
    There were also significant improvements in secondary endpoints including knee extensor strength, patient global impression of symptoms, and some cardiac parameters.
    1 context sentence
    Summary · context
    Ten subjects continued on the open-label extension (part 2) of 40 mg per day of elamipretide, with eight subjects reaching 36 weeks.
  7. 07HumanNon-randomized trialEfficacy outcome

    In people with Barth syndrome, open-label elamipretide was followed by significant improvement in some cardiac parameters.

    In plain wordsIn people with Barth syndrome, some heart measures improved during open-label elamipretide, with no placebo comparison.

    Peptide
    Elamipretide (SS-31)
    Outcome
    MixedCardiac parameters
    Study

    n: not reported in source

    Analysis
    Secondary
    Outcome group
    Heart function · Cardiovascular
    Studied dose
    40 mg per dayAs given in this study (human).

    Source sentences

    Summary
    There were also significant improvements in secondary endpoints including knee extensor strength, patient global impression of symptoms, and some cardiac parameters.
    1 context sentence
    Summary · context
    Ten subjects continued on the open-label extension (part 2) of 40 mg per day of elamipretide, with eight subjects reaching 36 weeks.

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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