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Study · Observational study · n = 32 · 2025

9 results

Assessing Experiences With Trofinetide for Rett Syndrome: Interviews With Caregivers of Participants in Clinical Trials.

Barrett AM, Olayinka-Amao O, Martin S, Doshi D, Bishop KM, Youakim JM

Clinical therapeutics16 Jan 2025Cited by 3 (Europe PMC)

PMID39824747 (opens in a new tab)DOI10.1016/j.clinthera.2024.12.012 (opens in a new tab)PepBankPUB-39824747
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 study9

9 results from studies that gave the peptide. 9 results from people.

  1. 01HumanObservational studyOther

    Among caregivers of trofinetide trial participants, 31/32 were satisfied or very satisfied with treatment.

    In plain wordsAmong caregivers of trofinetide trial participants, 31/32 were satisfied or very satisfied with treatment.

    Peptide
    Trofinetide
    Outcome
    Not applicableCaregiver satisfaction
    Study

    n = 32

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

    Measurements

    Satisfied or very satisfied
    31 caregivers
    31/32

    Source sentences

    Summary
    Nearly all (31/32) caregivers were very satisfied or satisfied with trofinetide.
    2 context sentences
    Summary · context
    To gain insight into experiences with RTT and effects of trofinetide treatment at different stages of RTT, interviews with caregivers of individuals with RTT were conducted upon their exit from the open-label trofinetide trials.
    Summary · context
    Interviews were conducted with caregivers of participants in the LILAC/LILAC-2 open-label extension trials of the phase 3 LAVENDER trial in participants aged 5 to 20 years, and in DAFFODIL, an open-label trial in participants aged 2 to 4 years.
  2. 02HumanObservational studyEfficacy outcome

    Among LILAC/LILAC-2 trofinetide participants, caregivers reported improved hand use in 11/26 individuals, or 42.3%.

    In plain wordsCaregivers reported improved hand use in 42.3% of LILAC/LILAC-2 trofinetide participants.

    Peptide
    Trofinetide
    Outcome
    IncreaseHand use in LILAC/LILAC-2
    Study

    n = 26

    Outcome group
    Exercise and physical function · Nervous system and cognition
    Studied dose
    Dose not stated

    Measurements

    Improved hand use
    11 participants
    11/26; 42.3%
    Improved hand use
    42.3%
    11/26 participants

    Source sentences

    Summary
    Caregivers most commonly reported improvements in hand use (11/26 [42.3%]), engagement with others (11/26 [42.3%]), eye gaze (8/26 [30.8%]), use of the Tobii eye tracking device (7/26 [26.9%]), and attention/focus/concentration (7/26 [26.9%]) in LILAC/LILAC-2.
    2 context sentences
    Summary · context
    To gain insight into experiences with RTT and effects of trofinetide treatment at different stages of RTT, interviews with caregivers of individuals with RTT were conducted upon their exit from the open-label trofinetide trials.
    Summary · context
    Interviews were conducted with caregivers of participants in the LILAC/LILAC-2 open-label extension trials of the phase 3 LAVENDER trial in participants aged 5 to 20 years, and in DAFFODIL, an open-label trial in participants aged 2 to 4 years.
  3. 03HumanObservational studyEfficacy outcome

    Among LILAC/LILAC-2 trofinetide participants, caregivers reported improved engagement with others in 11/26 individuals, or 42.3%.

    In plain wordsCaregivers reported improved engagement with others in 42.3% of LILAC/LILAC-2 trofinetide participants.

    Peptide
    Trofinetide
    Outcome
    IncreaseEngagement in LILAC/LILAC-2
    Study

    n = 26

    Outcome group
    Social engagement · Nervous system and cognition
    Studied dose
    Dose not stated

    Measurements

    Improved engagement
    11 participants
    11/26; 42.3%
    Improved engagement
    42.3%
    11/26 participants

    Source sentences

    Summary
    Caregivers most commonly reported improvements in hand use (11/26 [42.3%]), engagement with others (11/26 [42.3%]), eye gaze (8/26 [30.8%]), use of the Tobii eye tracking device (7/26 [26.9%]), and attention/focus/concentration (7/26 [26.9%]) in LILAC/LILAC-2.
    2 context sentences
    Summary · context
    To gain insight into experiences with RTT and effects of trofinetide treatment at different stages of RTT, interviews with caregivers of individuals with RTT were conducted upon their exit from the open-label trofinetide trials.
    Summary · context
    Interviews were conducted with caregivers of participants in the LILAC/LILAC-2 open-label extension trials of the phase 3 LAVENDER trial in participants aged 5 to 20 years, and in DAFFODIL, an open-label trial in participants aged 2 to 4 years.
  4. 04HumanObservational studyEfficacy outcome

    Among LILAC/LILAC-2 trofinetide participants, caregivers reported improved eye gaze in 8/26 individuals, or 30.8%.

    In plain wordsCaregivers reported improved eye gaze in 30.8% of LILAC/LILAC-2 trofinetide participants.

    Peptide
    Trofinetide
    Outcome
    IncreaseEye gaze in LILAC/LILAC-2
    Study

    n = 26

    Outcome group
    Communication · Nervous system and cognition
    Studied dose
    Dose not stated

    Measurements

    Improved eye gaze
    8 participants
    8/26; 30.8%
    Improved eye gaze
    30.8%
    8/26 participants

    Source sentences

    Summary
    Caregivers most commonly reported improvements in hand use (11/26 [42.3%]), engagement with others (11/26 [42.3%]), eye gaze (8/26 [30.8%]), use of the Tobii eye tracking device (7/26 [26.9%]), and attention/focus/concentration (7/26 [26.9%]) in LILAC/LILAC-2.
    2 context sentences
    Summary · context
    To gain insight into experiences with RTT and effects of trofinetide treatment at different stages of RTT, interviews with caregivers of individuals with RTT were conducted upon their exit from the open-label trofinetide trials.
    Summary · context
    Interviews were conducted with caregivers of participants in the LILAC/LILAC-2 open-label extension trials of the phase 3 LAVENDER trial in participants aged 5 to 20 years, and in DAFFODIL, an open-label trial in participants aged 2 to 4 years.
  5. 05HumanObservational studyEfficacy outcome

    Among LILAC/LILAC-2 trofinetide participants, caregivers reported improved Tobii eye tracking device use in 7/26 individuals, or 26.9%.

    In plain wordsCaregivers reported improved eye tracking device use in 26.9% of LILAC/LILAC-2 trofinetide participants.

    Peptide
    Trofinetide
    Outcome
    IncreaseEye tracking device use in LILAC/LILAC-2
    Study

    n = 26

    Outcome group
    Communication · Nervous system and cognition
    Studied dose
    Dose not stated

    Measurements

    Improved device use
    7 participants
    7/26; 26.9%
    Improved device use
    26.9%
    7/26 participants

    Source sentences

    Summary
    Caregivers most commonly reported improvements in hand use (11/26 [42.3%]), engagement with others (11/26 [42.3%]), eye gaze (8/26 [30.8%]), use of the Tobii eye tracking device (7/26 [26.9%]), and attention/focus/concentration (7/26 [26.9%]) in LILAC/LILAC-2.
    2 context sentences
    Summary · context
    To gain insight into experiences with RTT and effects of trofinetide treatment at different stages of RTT, interviews with caregivers of individuals with RTT were conducted upon their exit from the open-label trofinetide trials.
    Summary · context
    Interviews were conducted with caregivers of participants in the LILAC/LILAC-2 open-label extension trials of the phase 3 LAVENDER trial in participants aged 5 to 20 years, and in DAFFODIL, an open-label trial in participants aged 2 to 4 years.
  6. 06HumanObservational studyEfficacy outcome

    Among LILAC/LILAC-2 trofinetide participants, caregivers reported improved attention/focus/concentration in 7/26 individuals, or 26.9%.

    In plain wordsCaregivers reported improved attention, focus, or concentration in 26.9% of LILAC/LILAC-2 trofinetide participants.

    Peptide
    Trofinetide
    Outcome
    IncreaseAttention/focus/concentration in LILAC/LILAC-2
    Study

    n = 26

    Outcome group
    Cognition and memory · Nervous system and cognition
    Studied dose
    Dose not stated

    Measurements

    Improved attention/focus/concentration
    7 participants
    7/26; 26.9%
    Improved attention/focus/concentration
    26.9%
    7/26 participants

    Source sentences

    Summary
    Caregivers most commonly reported improvements in hand use (11/26 [42.3%]), engagement with others (11/26 [42.3%]), eye gaze (8/26 [30.8%]), use of the Tobii eye tracking device (7/26 [26.9%]), and attention/focus/concentration (7/26 [26.9%]) in LILAC/LILAC-2.
    2 context sentences
    Summary · context
    To gain insight into experiences with RTT and effects of trofinetide treatment at different stages of RTT, interviews with caregivers of individuals with RTT were conducted upon their exit from the open-label trofinetide trials.
    Summary · context
    Interviews were conducted with caregivers of participants in the LILAC/LILAC-2 open-label extension trials of the phase 3 LAVENDER trial in participants aged 5 to 20 years, and in DAFFODIL, an open-label trial in participants aged 2 to 4 years.
  7. 07HumanObservational studyEfficacy outcome

    Among DAFFODIL trofinetide participants, caregivers reported improvements in new words in 5/7 individuals, or 71.4%.

    In plain wordsCaregivers reported improvements in new words in 71.4% of DAFFODIL trofinetide participants.

    Peptide
    Trofinetide
    Outcome
    IncreaseNew words in DAFFODIL
    Study

    n = 7

    Outcome group
    Communication · Nervous system and cognition
    Studied dose
    Dose not stated

    Measurements

    Improved new words
    5 participants
    5/7; 71.4%
    Improved new words
    71.4%
    5/7 participants

    Source sentences

    Summary
    In DAFFODIL, caregivers reported improvements in new words (5/7 [71.4%]), hand use (4/7 [57.1%]), and eye contact (4/7 [57.1%]).
    2 context sentences
    Summary · context
    To gain insight into experiences with RTT and effects of trofinetide treatment at different stages of RTT, interviews with caregivers of individuals with RTT were conducted upon their exit from the open-label trofinetide trials.
    Summary · context
    Interviews were conducted with caregivers of participants in the LILAC/LILAC-2 open-label extension trials of the phase 3 LAVENDER trial in participants aged 5 to 20 years, and in DAFFODIL, an open-label trial in participants aged 2 to 4 years.
  8. 08HumanObservational studyEfficacy outcome

    Among DAFFODIL trofinetide participants, caregivers reported improved hand use in 4/7 individuals, or 57.1%.

    In plain wordsCaregivers reported improved hand use in 57.1% of DAFFODIL trofinetide participants.

    Peptide
    Trofinetide
    Outcome
    IncreaseHand use in DAFFODIL
    Study

    n = 7

    Outcome group
    Exercise and physical function · Nervous system and cognition
    Studied dose
    Dose not stated

    Measurements

    Improved hand use
    4 participants
    4/7; 57.1%
    Improved hand use
    57.1%
    4/7 participants

    Source sentences

    Summary
    In DAFFODIL, caregivers reported improvements in new words (5/7 [71.4%]), hand use (4/7 [57.1%]), and eye contact (4/7 [57.1%]).
    2 context sentences
    Summary · context
    To gain insight into experiences with RTT and effects of trofinetide treatment at different stages of RTT, interviews with caregivers of individuals with RTT were conducted upon their exit from the open-label trofinetide trials.
    Summary · context
    Interviews were conducted with caregivers of participants in the LILAC/LILAC-2 open-label extension trials of the phase 3 LAVENDER trial in participants aged 5 to 20 years, and in DAFFODIL, an open-label trial in participants aged 2 to 4 years.
  9. 09HumanObservational studyEfficacy outcome

    Among DAFFODIL trofinetide participants, caregivers reported improved eye contact in 4/7 individuals, or 57.1%.

    In plain wordsCaregivers reported improved eye contact in 57.1% of DAFFODIL trofinetide participants.

    Peptide
    Trofinetide
    Outcome
    IncreaseEye contact in DAFFODIL
    Study

    n = 7

    Outcome group
    Communication · Nervous system and cognition
    Studied dose
    Dose not stated

    Measurements

    Improved eye contact
    4 participants
    4/7; 57.1%
    Improved eye contact
    57.1%
    4/7 participants

    Source sentences

    Summary
    In DAFFODIL, caregivers reported improvements in new words (5/7 [71.4%]), hand use (4/7 [57.1%]), and eye contact (4/7 [57.1%]).
    2 context sentences
    Summary · context
    To gain insight into experiences with RTT and effects of trofinetide treatment at different stages of RTT, interviews with caregivers of individuals with RTT were conducted upon their exit from the open-label trofinetide trials.
    Summary · context
    Interviews were conducted with caregivers of participants in the LILAC/LILAC-2 open-label extension trials of the phase 3 LAVENDER trial in participants aged 5 to 20 years, and in DAFFODIL, an open-label trial in participants aged 2 to 4 years.

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

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