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Study · Observational study · 2024

4 results

Exposure-Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome.

Darwish M, Passarell J, Youakim JM, Bradley H, Bishop KM

Advances in therapy16 Feb 2024Cited by 3 (Europe PMC)

PMID38363467 (opens in a new tab)PMCIDPMC10960884 (opens in a new tab)DOI10.1007/s12325-024-02796-y (opens in a new tab)PepBankPUB-38363467
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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. Results still quote short attributed sentences.

Results from this study4

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

  1. 01HumanObservational studyEfficacy outcome

    In individuals with Rett syndrome, higher trofinetide exposure was associated with improved RSBQ scores.

    In plain wordsIn people with Rett syndrome, higher trofinetide exposure was linked to better symptom scores.

    Peptide
    Trofinetide
    Outcome
    DecreaseRSBQ score exposure association
    Study

    n: not reported in source

    Comparator
    Lower trofinetide exposure
    Outcome group
    Rett Syndrome Symptoms · Nervous system and cognition
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Higher trofinetide exposure was associated with improvements in RSBQ, CSBS-DP-IT Social Composite, and RTT-COMC scores.
    2 context sentences
    Summary · context
    Efficacy endpoints were modeled using trofinetide exposure measures predicted from the population pharmacokinetic model and Bayesian estimates.
    Summary · context
    The analysis population for each E-R model comprised individuals receiving placebo or trofinetide who had available trofinetide exposure measures.
  2. 02HumanObservational studyEfficacy outcome

    In individuals with Rett syndrome, higher trofinetide exposure was associated with improved CSBS-DP-IT Social Composite scores.

    In plain wordsIn people with Rett syndrome, higher trofinetide exposure was linked to better social communication scores.

    Peptide
    Trofinetide
    Outcome
    Direction not reportedCSBS-DP-IT Social Composite exposure association
    Study

    n: not reported in source

    Comparator
    Lower trofinetide exposure
    Outcome group
    Communication · Nervous system and cognition
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Higher trofinetide exposure was associated with improvements in RSBQ, CSBS-DP-IT Social Composite, and RTT-COMC scores.
    3 context sentences
    Summary · context
    Significant E-R relationships were also found for the CSBS-DP-IT Social Composite and RTT-COMC scores.
    Summary · context
    Efficacy endpoints were modeled using trofinetide exposure measures predicted from the population pharmacokinetic model and Bayesian estimates.
    Summary · context
    The analysis population for each E-R model comprised individuals receiving placebo or trofinetide who had available trofinetide exposure measures.
  3. 03HumanObservational studyEfficacy outcome

    In individuals with Rett syndrome, higher trofinetide exposure was associated with improved RTT-COMC scores.

    In plain wordsIn people with Rett syndrome, higher trofinetide exposure was linked to better nonverbal communication scores.

    Peptide
    Trofinetide
    Outcome
    Direction not reportedRTT-COMC exposure association
    Study

    n: not reported in source

    Comparator
    Lower trofinetide exposure
    Outcome group
    Communication · Nervous system and cognition
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Higher trofinetide exposure was associated with improvements in RSBQ, CSBS-DP-IT Social Composite, and RTT-COMC scores.
    3 context sentences
    Summary · context
    Significant E-R relationships were also found for the CSBS-DP-IT Social Composite and RTT-COMC scores.
    Summary · context
    Efficacy endpoints were modeled using trofinetide exposure measures predicted from the population pharmacokinetic model and Bayesian estimates.
    Summary · context
    The analysis population for each E-R model comprised individuals receiving placebo or trofinetide who had available trofinetide exposure measures.
  4. 04Computer modelIn silicoEfficacy outcome

    In an exposure-response model of Rett syndrome clinical data, assumed target trofinetide exposure was estimated to give RSBQ reductions at week 12 approximately five- to seven-fold greater than placebo.

    In plain wordsIn a computer model of Rett syndrome trial data, trofinetide at target exposure was estimated to cut symptom scores more than placebo.

    Peptide
    Trofinetide
    Outcome
    DecreaseModel-derived RSBQ reduction at target exposure
    Study

    n: not reported in source

    exposure-response efficacy model fitted to clinical study data
    Comparator
    Placebo
    Outcome group
    Rett Syndrome Symptoms · Nervous system and cognition
    Studied dose
    Dose not stated

    Measurements

    Modeled RSBQ reduction
    3.55 to 4.94 points
    trofinetide at assumed target AUC0-12 800-1200 ug.h/mL, week 12
    Modeled RSBQ reduction
    0.76 points
    placebo, week 12
    Fold difference in RSBQ reduction
    5 to 7 fold
    approximately, trofinetide versus placebo

    Source sentences

    Summary
    Assuming target trofinetide AUC0-12 values of 800-1200 μg·h/mL, the reductions in RSBQ total scores at week 12 were approximately five- to seven-fold greater with trofinetide (range 3.55-4.94) versus placebo (0.76).
    2 context sentences
    Summary · context
    Efficacy endpoints were modeled using trofinetide exposure measures predicted from the population pharmacokinetic model and Bayesian estimates.
    Summary · context
    The analysis population for each E-R model comprised individuals receiving placebo or trofinetide who had available trofinetide exposure measures.

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

Registered trials3

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

3 trials · 3 completed · 1 with posted results · source ClinicalTrials.gov (opens in a new tab)

CompletedPhase 3Results postedNCT04181723

Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)

ACADIA Pharmaceuticals Inc.n = 1876 Nov 2019 to 28 Oct 2021

Rett Syndrome

Trial pageClinicalTrials.govUpdated 8 Apr 2024
CompletedPhase 2NCT02715115

A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

Neuren Pharmaceuticals Limitedn = 82Mar 2016 to 5 Jan 2017

Rett Syndrome

Trial pageClinicalTrials.govUpdated 14 Aug 2020
CompletedPhase 2NCT01703533

A Safety Study of NNZ-2566 in Patients With Rett Syndrome

Neuren Pharmaceuticals Limitedn = 67Mar 2013 to Sept 2014

Rett Syndrome

Trial pageClinicalTrials.govUpdated 5 Feb 2018

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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