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Study · Randomized controlled trial · 2017

3 results

A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Rett Syndrome.

Glaze DG, Neul JL, Percy A, Feyma T, Beisang A, Yaroshinsky A, Stoms G, Zuchero D, Horrigan J, Glass L, Jones NE

Pediatric neurology8 Jul 2017Cited by 81 (Europe PMC)

PMID28964591 (opens in a new tab)DOI10.1016/j.pediatrneurol.2017.07.002 (opens in a new tab)PepBankPUB-28964591
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: Publisher copyright. 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 at 70 mg/kg demonstrated efficacy versus placebo based on prespecified criteria.

    In plain wordsIn females with Rett syndrome, trofinetide at 70 mg/kg met prespecified benefit criteria compared with placebo.

    Peptide
    Trofinetide
    Outcome
    Not applicablePrespecified efficacy criteria
    Study

    n: not reported in source

    Comparator
    Placebo
    Outcome group
    Rett Syndrome Symptoms · Nervous system and cognition
    Studied dose
    70 mg/kg twice daily · 28 daysAs given in this study (human).

    Source sentences

    Summary
    Trofinetide at 70 mg/kg demonstrated efficacy compared with placebo based on prespecified criteria.
    2 context sentences
    Summary · context
    This was an exploratory, phase 2, multicenter, double-blind, placebo-controlled, dose-escalation study of the safety and tolerability of trofinetide in 56 adolescent and adult females with Rett syndrome.
    Summary · context
    Subjects were randomly assigned in a 2:1 ratio to 35 mg/kg twice daily of trofinetide or placebo for 14 days; 35 mg/kg twice daily or placebo for 28 days; or 70 mg/kg twice daily or placebo for 28 days.
  2. 02HumanNon-randomized trialSafety outcome

    In females with Rett syndrome, trofinetide at 35 mg/kg was well tolerated and generally safe.

    In plain wordsIn females with Rett syndrome, trofinetide at 35 mg/kg was well tolerated and generally safe.

    Peptide
    Trofinetide
    Outcome
    Not applicableSafety and tolerability at 35 mg/kg
    Study

    n: not reported in source

    Outcome group
    Adverse events · Whole body
    Studied dose
    35 mg/kg twice daily · 14 or 28 daysAs given in this study (human).

    Source sentences

    Summary
    Both 35 mg/kg and 70 mg/kg dose levels of trofinetide were well tolerated and generally safe.
    2 context sentences
    Summary · context
    This was an exploratory, phase 2, multicenter, double-blind, placebo-controlled, dose-escalation study of the safety and tolerability of trofinetide in 56 adolescent and adult females with Rett syndrome.
    Summary · context
    Subjects were randomly assigned in a 2:1 ratio to 35 mg/kg twice daily of trofinetide or placebo for 14 days; 35 mg/kg twice daily or placebo for 28 days; or 70 mg/kg twice daily or placebo for 28 days.
  3. 03HumanNon-randomized trialSafety outcome

    In females with Rett syndrome, trofinetide at 70 mg/kg was well tolerated and generally safe.

    In plain wordsIn females with Rett syndrome, trofinetide at 70 mg/kg was well tolerated and generally safe.

    Peptide
    Trofinetide
    Outcome
    Not applicableSafety and tolerability at 70 mg/kg
    Study

    n: not reported in source

    Outcome group
    Adverse events · Whole body
    Studied dose
    70 mg/kg twice daily · 28 daysAs given in this study (human).

    Source sentences

    Summary
    Both 35 mg/kg and 70 mg/kg dose levels of trofinetide were well tolerated and generally safe.
    2 context sentences
    Summary · context
    This was an exploratory, phase 2, multicenter, double-blind, placebo-controlled, dose-escalation study of the safety and tolerability of trofinetide in 56 adolescent and adult females with Rett syndrome.
    Summary · context
    Subjects were randomly assigned in a 2:1 ratio to 35 mg/kg twice daily of trofinetide or placebo for 14 days; 35 mg/kg twice daily or placebo for 28 days; or 70 mg/kg twice daily or placebo for 28 days.

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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