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Study · Randomized controlled trial · 2024

3 results

Trofinetide Treatment Demonstrates a Benefit Over Placebo for the Ability to Communicate in Rett Syndrome.

Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Peters SU, Marsh ED, An D, Bishop KM, Youakim JM

Pediatric neurology23 Nov 2023Cited by 22 (Europe PMC)

PMID38232652 (opens in a new tab)DOI10.1016/j.pediatrneurol.2023.11.005 (opens in a new tab)PepBankPUB-38232652
Publisher pagePubMedAsk about this study
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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 study3

3 results from studies that gave the peptide. 3 results from people.

  1. 01HumanRandomized controlled trialEfficacy outcome

    In females with Rett syndrome, trofinetide increased CSBS-DP-IT Social Composite scores versus placebo.

    In plain wordsIn females with Rett syndrome, trofinetide improved social communication scores more than placebo.

    Peptide
    Trofinetide
    Outcome
    IncreaseCSBS-DP-IT Social Composite score
    Study

    n: not reported in source

    Comparator
    Placebo
    Analysis
    Secondary
    Outcome group
    Communication · Nervous system and cognition
    Studied dose
    Dose not stated · 12 weeksAs given in this study (human).

    Measurements

    LSM difference
    1 points
    trofinetide versus placebo
    95% CI lower
    0.3 points
    difference
    95% CI upper
    1.7 points
    difference
    P
    0.0064
    difference
    Cohen's d
    0.43
    difference

    Source sentences

    Summary
    Trofinetide demonstrated a statistically significant difference versus placebo for the CSBS-DP-IT Social Composite score (least squares mean [LSM] difference = 1.0; 95% confidence interval [CI], 0.3 to 1.7; P = 0.0064; Cohen's d effect size = 0.43)
    1 context sentence
    Summary · context
    Females with RTT, aged five to 20 years, were randomized 1:1 to trofinetide or placebo for 12 weeks.
  2. 02HumanRandomized controlled trialEfficacy outcome

    In females with Rett syndrome, trofinetide reduced RTT-COMC scores versus placebo with nominal significance.

    In plain wordsIn females with Rett syndrome, trofinetide improved nonverbal communication scores more than placebo, with nominal significance.

    Peptide
    Trofinetide
    Outcome
    DecreaseRTT-COMC score
    Study

    n: not reported in source

    Comparator
    Placebo
    Analysis
    Secondary
    Outcome group
    Communication · Nervous system and cognition
    Studied dose
    Dose not stated · 12 weeksAs given in this study (human).

    Measurements

    LSM difference
    −0.3 points
    trofinetide versus placebo
    95% CI lower
    −0.6 points
    difference
    95% CI upper
    0 points
    difference
    P
    0.0257
    difference
    Cohen's d
    0.36
    difference

    Source sentences

    Summary
    and a nominally significant difference for the RTT-COMC (LSM difference: -0.3; 95% CI, -0.6 to -0.0; P = 0.0257; Cohen's d effect size = 0.36).
    1 context sentence
    Summary · context
    Females with RTT, aged five to 20 years, were randomized 1:1 to trofinetide or placebo for 12 weeks.
  3. 03HumanRandomized controlled trialEfficacy outcome

    In females with Rett syndrome, trofinetide did not differ from placebo for RTT-VCOM scores.

    In plain wordsIn females with Rett syndrome, verbal communication scores did not differ between trofinetide and placebo.

    Peptide
    Trofinetide
    Outcome
    No significant changeRTT-VCOM score
    Study

    n: not reported in source

    Comparator
    Placebo
    Analysis
    Secondary
    Outcome group
    Communication · Nervous system and cognition
    Studied dose
    Dose not stated · 12 weeksAs given in this study (human).

    Source sentences

    Summary
    As expected, there was no difference for the RTT-VCOM.
    1 context sentence
    Summary · context
    Females with RTT, aged five to 20 years, were randomized 1:1 to trofinetide or placebo for 12 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

    28 Oct 2026, 02:03 UTC

    Study results updated

  2. Version 1

    27 Oct 2026, 16:37 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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