Skip to content

Study · Randomized controlled trial · 2019

5 results

Double-blind, randomized, placebo-controlled study of trofinetide in pediatric Rett syndrome.

Glaze DG, Neul JL, Kaufmann WE, Berry-Kravis E, Condon S, Stoms G, Oosterholt S, Della Pasqua O, Glass L, Jones NE, Percy AK

Neurology27 Mar 2019Cited by 113 (Europe PMC)

PMID30918097 (opens in a new tab)PMCIDPMC6550498 (opens in a new tab)DOI10.1212/wnl.0000000000007316 (opens in a new tab)PepBankPUB-30918097
Read at sourcePubMedAsk about this study
Jump to 5 results

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 study5

5 results from studies that gave the peptide. 5 results from people.

  1. 01HumanRandomized controlled trialEfficacy outcome

    In children and adolescents with Rett syndrome, trofinetide improved RSBQ, RTT-Clinician Domain Specific Concerns-Visual Analog Scale, and CGI-I versus placebo.

    In plain wordsIn children and adolescents with Rett syndrome, trofinetide improved three named symptom and clinical rating measures more than placebo.

    Peptide
    Trofinetide
    Outcome
    Not applicableRSBQ, RTT-DSC-VAS, and CGI-I
    Study

    n: not reported in source

    Comparator
    Placebo
    Outcome group
    Rett Syndrome Symptoms · Nervous system and cognition
    Studied dose
    200 mg/kg twice daily · 42 daysAs given in this study (human).

    Source sentences

    Summary
    Trofinetide at 200 mg/kg bid showed statistically significant and clinically relevant improvements relative to placebo on the Rett Syndrome Behaviour Questionnaire, RTT-Clinician Domain Specific Concerns-Visual Analog Scale, and Clinical Global Impression Scale-Improvement.
    3 context sentences
    Summary · context
    This was a phase 2, multicenter, double-blind, placebo-controlled, parallel-group study, in which safety/tolerability, pharmacokinetics, and clinical response to trofinetide were characterized in 82 children/adolescents with RTT, aged 5 to 15 years.
    Summary · context
    Sixty-two participants were randomized 1:1:1:1 to receive placebo twice a day (bid) for 14 days, followed by placebo, 50, 100, or 200 mg/kg bid of trofinetide for 42 days.
    Summary · context
    Following blinded safety data review, 20 additional participants were randomized 1:1 to the 200 mg/kg or placebo bid groups.
  2. 02HumanNon-randomized trialSafety outcome

    In children and adolescents with Rett syndrome, trofinetide at 50 mg/kg twice daily was well tolerated and generally safe.

    In plain wordsIn children and adolescents with Rett syndrome, trofinetide at 50 mg/kg twice daily was well tolerated and generally safe.

    Peptide
    Trofinetide
    Outcome
    Not applicableSafety and tolerability at 50 mg/kg
    Study

    n: not reported in source

    Outcome group
    Adverse events · Whole body
    Studied dose
    50 mg/kg twice daily · 42 daysAs given in this study (human).

    Source sentences

    Summary
    All dose levels were well tolerated and generally safe.
    3 context sentences
    Summary · context
    This was a phase 2, multicenter, double-blind, placebo-controlled, parallel-group study, in which safety/tolerability, pharmacokinetics, and clinical response to trofinetide were characterized in 82 children/adolescents with RTT, aged 5 to 15 years.
    Summary · context
    Sixty-two participants were randomized 1:1:1:1 to receive placebo twice a day (bid) for 14 days, followed by placebo, 50, 100, or 200 mg/kg bid of trofinetide for 42 days.
    Summary · context
    Following blinded safety data review, 20 additional participants were randomized 1:1 to the 200 mg/kg or placebo bid groups.
  3. 03HumanNon-randomized trialSafety outcome

    In children and adolescents with Rett syndrome, trofinetide at 100 mg/kg twice daily was well tolerated and generally safe.

    In plain wordsIn children and adolescents with Rett syndrome, trofinetide at 100 mg/kg twice daily was well tolerated and generally safe.

    Peptide
    Trofinetide
    Outcome
    Not applicableSafety and tolerability at 100 mg/kg
    Study

    n: not reported in source

    Outcome group
    Adverse events · Whole body
    Studied dose
    100 mg/kg twice daily · 42 daysAs given in this study (human).

    Source sentences

    Summary
    All dose levels were well tolerated and generally safe.
    3 context sentences
    Summary · context
    This was a phase 2, multicenter, double-blind, placebo-controlled, parallel-group study, in which safety/tolerability, pharmacokinetics, and clinical response to trofinetide were characterized in 82 children/adolescents with RTT, aged 5 to 15 years.
    Summary · context
    Sixty-two participants were randomized 1:1:1:1 to receive placebo twice a day (bid) for 14 days, followed by placebo, 50, 100, or 200 mg/kg bid of trofinetide for 42 days.
    Summary · context
    Following blinded safety data review, 20 additional participants were randomized 1:1 to the 200 mg/kg or placebo bid groups.
  4. 04HumanNon-randomized trialSafety outcome

    In children and adolescents with Rett syndrome, trofinetide at 200 mg/kg twice daily was well tolerated and generally safe.

    In plain wordsIn children and adolescents with Rett syndrome, trofinetide at 200 mg/kg twice daily was well tolerated and generally safe.

    Peptide
    Trofinetide
    Outcome
    Not applicableSafety and tolerability at 200 mg/kg
    Study

    n: not reported in source

    Outcome group
    Adverse events · Whole body
    Studied dose
    200 mg/kg twice daily · 42 daysAs given in this study (human).

    Source sentences

    Summary
    All dose levels were well tolerated and generally safe.
    3 context sentences
    Summary · context
    This was a phase 2, multicenter, double-blind, placebo-controlled, parallel-group study, in which safety/tolerability, pharmacokinetics, and clinical response to trofinetide were characterized in 82 children/adolescents with RTT, aged 5 to 15 years.
    Summary · context
    Sixty-two participants were randomized 1:1:1:1 to receive placebo twice a day (bid) for 14 days, followed by placebo, 50, 100, or 200 mg/kg bid of trofinetide for 42 days.
    Summary · context
    Following blinded safety data review, 20 additional participants were randomized 1:1 to the 200 mg/kg or placebo bid groups.
  5. 05HumanObservational studyEfficacy outcome

    In children and adolescents with Rett syndrome, exploratory analyses suggested that observed changes correlated with trofinetide exposure.

    In plain wordsIn children and adolescents with Rett syndrome, exploratory analyses suggested a link between trofinetide exposure and observed changes.

    Peptide
    Trofinetide
    Outcome
    Direction not reportedExposure-response association
    Study

    n: not reported in source

    Analysis
    Exploratory
    Outcome group
    Rett Syndrome Symptoms · Nervous system and cognition
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Exploratory analyses suggested that observed changes correlated with trofinetide exposure.
    3 context sentences
    Summary · context
    This was a phase 2, multicenter, double-blind, placebo-controlled, parallel-group study, in which safety/tolerability, pharmacokinetics, and clinical response to trofinetide were characterized in 82 children/adolescents with RTT, aged 5 to 15 years.
    Summary · context
    Sixty-two participants were randomized 1:1:1:1 to receive placebo twice a day (bid) for 14 days, followed by placebo, 50, 100, or 200 mg/kg bid of trofinetide for 42 days.
    Summary · context
    Following blinded safety data review, 20 additional participants were randomized 1:1 to the 200 mg/kg or placebo bid groups.

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.

PepBank is a research reference. Talk to a doctor before you use any peptide.