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Study · Non-randomized trial · n = 15 · 2025

5 results

Results from the phase 2/3 DAFFODIL study of trofinetide in girls aged 2-4 years with Rett syndrome.

Percy AK, Ryther R, Marsh ED, Neul JL, Benke TA, Berry-Kravis EM, Feyma T, Lieberman DN, Ananth AL, Fu C, Buhrfiend C, Barrett A and 5 more

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Doshi D, Darwish M, An D, Bishop KM, Youakim JM

Med (New York, N.Y.)4 Mar 2025Cited by 7 (Europe PMC)

PMID40043705 (opens in a new tab)DOI10.1016/j.medj.2025.100608 (opens in a new tab)PepBankPUB-40043705
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. Results still quote short attributed sentences.

Results from this study5

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

  1. 01HumanNon-randomized trialEfficacy outcome

    In young girls with RTT receiving trofinetide, symptoms improved throughout the study on CGI-I, CaGI-I and ICND-QoL change from baseline.

    In plain wordsYoung girls with RTT receiving trofinetide showed symptom improvement on clinician, caregiver, and quality-of-life measures during the study.

    Peptide
    Trofinetide
    Outcome
    Direction not reportedRTT symptoms across CGI-I, CaGI-I and ICND-QoL
    Study

    n = 15

    Analysis
    Exploratory
    Outcome group
    RTT symptoms · Nervous system and cognition
    Studied dose
    Dose not stated

    Source sentences

    Summary
    RTT symptoms improved throughout the study as measured by the CGI-I, CaGI-I, and change from baseline in the ICND-QoL.
    3 context sentences
    Summary · context
    Assessments included treatment-emergent adverse events (TEAEs) and exploratory efficacy (Clinical Global Impressions-Improvement [CGI-I], CGI-Severity, caregiver GI-I [CaGI-I], and overall quality of life rating of the Impact of Childhood Neurologic Disability Scale [ICND-QoL]).
    Summary · context
    The DAFFODIL study was conducted in girls aged 2-4 years with RTT to examine the safety, tolerability, and efficacy of trofinetide and to validate that the recommended dosage, according to body weight, achieved target exposure.
    Summary · context
    DAFFODIL was a phase 2/3, open-label study of trofinetide consisting of two treatment periods (12 weeks [period A] and ∼21 months [period B]).
  2. 02HumanNon-randomized trialSafety outcome

    In young girls with RTT receiving trofinetide, diarrhea occurred in 80.0% and was mild or moderate.

    In plain wordsIn young girls with RTT receiving trofinetide, 80.0% had mild or moderate diarrhea.

    Peptide
    Trofinetide
    Outcome
    Not applicableDiarrhea
    Study

    n = 15

    Outcome group
    Gastrointestinal adverse events · Gastrointestinal
    Studied dose
    Dose not stated

    Measurements

    Diarrhea
    80%
    reported result

    Source sentences

    Summary
    Overall, the most common TEAEs were diarrhea (80.0%) and vomiting (53.3%), which were mild or moderate in severity.
    2 context sentences
    Summary · context
    The DAFFODIL study was conducted in girls aged 2-4 years with RTT to examine the safety, tolerability, and efficacy of trofinetide and to validate that the recommended dosage, according to body weight, achieved target exposure.
    Summary · context
    DAFFODIL was a phase 2/3, open-label study of trofinetide consisting of two treatment periods (12 weeks [period A] and ∼21 months [period B]).
  3. 03HumanNon-randomized trialSafety outcome

    In young girls with RTT receiving trofinetide, vomiting occurred in 53.3% and was mild or moderate.

    In plain wordsIn young girls with RTT receiving trofinetide, 53.3% had mild or moderate vomiting.

    Peptide
    Trofinetide
    Outcome
    Not applicableVomiting
    Study

    n = 15

    Outcome group
    Gastrointestinal adverse events · Gastrointestinal
    Studied dose
    Dose not stated

    Measurements

    Vomiting
    53.3%
    reported result

    Source sentences

    Summary
    Overall, the most common TEAEs were diarrhea (80.0%) and vomiting (53.3%), which were mild or moderate in severity.
    2 context sentences
    Summary · context
    The DAFFODIL study was conducted in girls aged 2-4 years with RTT to examine the safety, tolerability, and efficacy of trofinetide and to validate that the recommended dosage, according to body weight, achieved target exposure.
    Summary · context
    DAFFODIL was a phase 2/3, open-label study of trofinetide consisting of two treatment periods (12 weeks [period A] and ∼21 months [period B]).
  4. 04HumanNon-randomized trialPharmacokinetics

    In young girls with RTT, trofinetide steady-state exposure at clinical doses fell within the target exposure range.

    In plain wordsIn young girls with RTT, trofinetide exposure at clinical doses fell within the target range.

    Peptide
    Trofinetide
    Outcome
    Not applicableSteady-state exposure
    Study

    n = 15

    Outcome group
    Pharmacokinetics · Pharmacology
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Steady-state exposure at clinical doses fell within the target exposure range.
    3 context sentences
    Summary · context
    Pharmacokinetic samples were collected at regular intervals during period A.
    Summary · context
    The DAFFODIL study was conducted in girls aged 2-4 years with RTT to examine the safety, tolerability, and efficacy of trofinetide and to validate that the recommended dosage, according to body weight, achieved target exposure.
    Summary · context
    DAFFODIL was a phase 2/3, open-label study of trofinetide consisting of two treatment periods (12 weeks [period A] and ∼21 months [period B]).
  5. 05HumanNon-randomized trialOther

    All seven interviewed caregivers of young girls with RTT reported satisfaction or high satisfaction with trofinetide benefits.

    In plain wordsAll interviewed caregivers of young girls with RTT were satisfied or very satisfied with trofinetide benefits.

    Peptide
    Trofinetide
    Outcome
    Not applicableCaregiver satisfaction
    Study

    n = 7

    Outcome group
    Patient-reported outcomes · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    In caregiver interviews (n = 7), all caregivers reported they were "very satisfied" or "satisfied" with trofinetide benefits.
    2 context sentences
    Summary · context
    The DAFFODIL study was conducted in girls aged 2-4 years with RTT to examine the safety, tolerability, and efficacy of trofinetide and to validate that the recommended dosage, according to body weight, achieved target exposure.
    Summary · context
    DAFFODIL was a phase 2/3, open-label study of trofinetide consisting of two treatment periods (12 weeks [period A] and ∼21 months [period B]).

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

Registered trials1

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

1 trial · 1 stopped early · 1 with posted results · source ClinicalTrials.gov (opens in a new tab)

TerminatedPhase 2/3Results postedNCT04988867

An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

ACADIA Pharmaceuticals Inc.n = 1522 Sept 2021 to 31 May 2023

Rett Syndrome

Trial pageClinicalTrials.govUpdated 24 Sept 2024

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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