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Study · Observational study · 2025

10 results

Safety Profiles of Trofinetide in Pediatric Rett Syndrome Population: A Real-World Postmarketing Pharmacovigilance Analysis.

Li J, Zhang Z, Yan T

Clinical therapeutics22 Aug 2025

PMID40849266 (opens in a new tab)DOI10.1016/j.clinthera.2025.07.022 (opens in a new tab)PepBankPUB-40849266
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 study10

10 results from studies that gave the peptide. 10 results from people.

  1. 01HumanObservational studySafety outcome

    In pediatric RTT pharmacovigilance reports, trofinetide was linked to disproportionate diarrhoea reporting, with 1,528 reports and ROR 38.1.

    In plain wordsTrofinetide was linked to disproportionate diarrhoea reporting compared with the reporting background in pediatric RTT safety data.

    Peptide
    Trofinetide
    Outcome
    IncreaseDiarrhoea reporting signal
    Study

    n: not reported in source

    Comparator
    FAERS reporting background
    Outcome group
    Gastrointestinal adverse events · Gastrointestinal
    Studied dose
    Dose not stated

    Measurements

    Reports
    1528 reports
    diarrhoea
    ROR
    38.1 ratio
    diarrhoea

    Source sentences

    Summary
    Common labeled AEs were confirmed, including diarrhoea (n = 1,528; ROR = 38.1), vomiting (n = 434; ROR = 5.96), and seizures (n = 251; ROR = 5.30).
    3 context sentences
    Summary · context
    This study evaluated the safety profile of trofinetide in pediatric patients with Rett syndrome using real-world pharmacovigilance data.
    Summary · context
    Adverse event (AE) reports were extracted from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) from Q1 2023 to Q4 2024.
    Summary · context
    Disproportionality analyses were conducted using ROR, PRR, BCPNN, and MGPS to detect AE signals associated with trofinetide use in individuals under 18 years.
  2. 02HumanObservational studySafety outcome

    In pediatric RTT pharmacovigilance reports, trofinetide was linked to disproportionate vomiting reporting, with 434 reports and ROR 5.96.

    In plain wordsTrofinetide was linked to disproportionate vomiting reporting compared with the reporting background in pediatric RTT safety data.

    Peptide
    Trofinetide
    Outcome
    IncreaseVomiting reporting signal
    Study

    n: not reported in source

    Comparator
    FAERS reporting background
    Outcome group
    Gastrointestinal adverse events · Gastrointestinal
    Studied dose
    Dose not stated

    Measurements

    Reports
    434 reports
    vomiting
    ROR
    5.96 ratio
    vomiting

    Source sentences

    Summary
    Common labeled AEs were confirmed, including diarrhoea (n = 1,528; ROR = 38.1), vomiting (n = 434; ROR = 5.96), and seizures (n = 251; ROR = 5.30).
    3 context sentences
    Summary · context
    This study evaluated the safety profile of trofinetide in pediatric patients with Rett syndrome using real-world pharmacovigilance data.
    Summary · context
    Adverse event (AE) reports were extracted from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) from Q1 2023 to Q4 2024.
    Summary · context
    Disproportionality analyses were conducted using ROR, PRR, BCPNN, and MGPS to detect AE signals associated with trofinetide use in individuals under 18 years.
  3. 03HumanObservational studySafety outcome

    In pediatric RTT pharmacovigilance reports, trofinetide was linked to disproportionate seizure reporting, with 251 reports and ROR 5.30.

    In plain wordsTrofinetide was linked to disproportionate seizure reporting compared with the reporting background in pediatric RTT safety data.

    Peptide
    Trofinetide
    Outcome
    IncreaseSeizure reporting signal
    Study

    n: not reported in source

    Comparator
    FAERS reporting background
    Outcome group
    Adverse events · Nervous system and cognition
    Studied dose
    Dose not stated

    Measurements

    Reports
    251 reports
    seizures
    ROR
    5.3 ratio
    seizures

    Source sentences

    Summary
    Common labeled AEs were confirmed, including diarrhoea (n = 1,528; ROR = 38.1), vomiting (n = 434; ROR = 5.96), and seizures (n = 251; ROR = 5.30).
    3 context sentences
    Summary · context
    This study evaluated the safety profile of trofinetide in pediatric patients with Rett syndrome using real-world pharmacovigilance data.
    Summary · context
    Adverse event (AE) reports were extracted from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) from Q1 2023 to Q4 2024.
    Summary · context
    Disproportionality analyses were conducted using ROR, PRR, BCPNN, and MGPS to detect AE signals associated with trofinetide use in individuals under 18 years.
  4. 04HumanObservational studySafety outcome

    In pediatric RTT pharmacovigilance reports, trofinetide was linked to disproportionate weight-decrease reporting, with 135 reports and ROR 7.21.

    In plain wordsTrofinetide was linked to disproportionate weight-decrease reporting compared with the reporting background in pediatric RTT safety data.

    Peptide
    Trofinetide
    Outcome
    IncreaseWeight decrease reporting signal
    Study

    n: not reported in source

    Comparator
    FAERS reporting background
    Outcome group
    Adverse events · Metabolic and weight
    Studied dose
    Dose not stated

    Measurements

    Reports
    135 reports
    weight decreased
    ROR
    7.21 ratio
    weight decreased

    Source sentences

    Summary
    Off-label signals included weight decreased (n = 135; ROR = 7.21), tremor (n = 39; ROR = 3.37), dystonia (n = 18; ROR = 4.39), and dyskinesia (n = 24; ROR = 4.53).
    3 context sentences
    Summary · context
    This study evaluated the safety profile of trofinetide in pediatric patients with Rett syndrome using real-world pharmacovigilance data.
    Summary · context
    Adverse event (AE) reports were extracted from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) from Q1 2023 to Q4 2024.
    Summary · context
    Disproportionality analyses were conducted using ROR, PRR, BCPNN, and MGPS to detect AE signals associated with trofinetide use in individuals under 18 years.
  5. 05HumanObservational studySafety outcome

    In pediatric RTT pharmacovigilance reports, trofinetide was linked to disproportionate tremor reporting, with 39 reports and ROR 3.37.

    In plain wordsTrofinetide was linked to disproportionate tremor reporting compared with the reporting background in pediatric RTT safety data.

    Peptide
    Trofinetide
    Outcome
    IncreaseTremor reporting signal
    Study

    n: not reported in source

    Comparator
    FAERS reporting background
    Outcome group
    Adverse events · Nervous system and cognition
    Studied dose
    Dose not stated

    Measurements

    Reports
    39 reports
    tremor
    ROR
    3.37 ratio
    tremor

    Source sentences

    Summary
    Off-label signals included weight decreased (n = 135; ROR = 7.21), tremor (n = 39; ROR = 3.37), dystonia (n = 18; ROR = 4.39), and dyskinesia (n = 24; ROR = 4.53).
    3 context sentences
    Summary · context
    This study evaluated the safety profile of trofinetide in pediatric patients with Rett syndrome using real-world pharmacovigilance data.
    Summary · context
    Adverse event (AE) reports were extracted from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) from Q1 2023 to Q4 2024.
    Summary · context
    Disproportionality analyses were conducted using ROR, PRR, BCPNN, and MGPS to detect AE signals associated with trofinetide use in individuals under 18 years.
  6. 06HumanObservational studySafety outcome

    In pediatric RTT pharmacovigilance reports, trofinetide was linked to disproportionate dystonia reporting, with 18 reports and ROR 4.39.

    In plain wordsTrofinetide was linked to disproportionate dystonia reporting compared with the reporting background in pediatric RTT safety data.

    Peptide
    Trofinetide
    Outcome
    IncreaseDystonia reporting signal
    Study

    n: not reported in source

    Comparator
    FAERS reporting background
    Outcome group
    Adverse events · Nervous system and cognition
    Studied dose
    Dose not stated

    Measurements

    Reports
    18 reports
    dystonia
    ROR
    4.39 ratio
    dystonia

    Source sentences

    Summary
    Off-label signals included weight decreased (n = 135; ROR = 7.21), tremor (n = 39; ROR = 3.37), dystonia (n = 18; ROR = 4.39), and dyskinesia (n = 24; ROR = 4.53).
    3 context sentences
    Summary · context
    This study evaluated the safety profile of trofinetide in pediatric patients with Rett syndrome using real-world pharmacovigilance data.
    Summary · context
    Adverse event (AE) reports were extracted from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) from Q1 2023 to Q4 2024.
    Summary · context
    Disproportionality analyses were conducted using ROR, PRR, BCPNN, and MGPS to detect AE signals associated with trofinetide use in individuals under 18 years.
  7. 07HumanObservational studySafety outcome

    In pediatric RTT pharmacovigilance reports, trofinetide was linked to disproportionate dyskinesia reporting, with 24 reports and ROR 4.53.

    In plain wordsTrofinetide was linked to disproportionate dyskinesia reporting compared with the reporting background in pediatric RTT safety data.

    Peptide
    Trofinetide
    Outcome
    IncreaseDyskinesia reporting signal
    Study

    n: not reported in source

    Comparator
    FAERS reporting background
    Outcome group
    Adverse events · Nervous system and cognition
    Studied dose
    Dose not stated

    Measurements

    Reports
    24 reports
    dyskinesia
    ROR
    4.53 ratio
    dyskinesia

    Source sentences

    Summary
    Off-label signals included weight decreased (n = 135; ROR = 7.21), tremor (n = 39; ROR = 3.37), dystonia (n = 18; ROR = 4.39), and dyskinesia (n = 24; ROR = 4.53).
    3 context sentences
    Summary · context
    This study evaluated the safety profile of trofinetide in pediatric patients with Rett syndrome using real-world pharmacovigilance data.
    Summary · context
    Adverse event (AE) reports were extracted from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) from Q1 2023 to Q4 2024.
    Summary · context
    Disproportionality analyses were conducted using ROR, PRR, BCPNN, and MGPS to detect AE signals associated with trofinetide use in individuals under 18 years.
  8. 08HumanObservational studySafety outcome

    In pediatric RTT trofinetide safety reports, over 70% of adverse events occurred within the first month.

    In plain wordsIn pediatric RTT trofinetide safety reports, over 70% of adverse events occurred within the first month.

    Peptide
    Trofinetide
    Outcome
    Not applicableAdverse event onset
    Study

    n: not reported in source

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated

    Measurements

    Adverse events
    > 70%
    first month

    Source sentences

    Summary
    Over 70% of AEs occurred within the first month.
    3 context sentences
    Summary · context
    This study evaluated the safety profile of trofinetide in pediatric patients with Rett syndrome using real-world pharmacovigilance data.
    Summary · context
    Adverse event (AE) reports were extracted from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) from Q1 2023 to Q4 2024.
    Summary · context
    Disproportionality analyses were conducted using ROR, PRR, BCPNN, and MGPS to detect AE signals associated with trofinetide use in individuals under 18 years.
  9. 09HumanObservational studySafety outcome

    In pediatric RTT trofinetide safety reports, hypersomnia was reported more frequently in younger children, with ROR 12.11.

    In plain wordsIn pediatric RTT trofinetide safety reports, hypersomnia was reported more often in younger children.

    Peptide
    Trofinetide
    Outcome
    IncreaseHypersomnia association with younger age
    Study

    n: not reported in source

    Outcome group
    Sleep · Sleep
    Studied dose
    Dose not stated

    Measurements

    ROR
    12.11 ratio
    hypersomnia in younger children

    Source sentences

    Summary
    Hypersomnia was more frequently reported in younger children (ROR = 12.11), and higher doses were associated with psychiatric symptoms.
    3 context sentences
    Summary · context
    This study evaluated the safety profile of trofinetide in pediatric patients with Rett syndrome using real-world pharmacovigilance data.
    Summary · context
    Adverse event (AE) reports were extracted from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) from Q1 2023 to Q4 2024.
    Summary · context
    Disproportionality analyses were conducted using ROR, PRR, BCPNN, and MGPS to detect AE signals associated with trofinetide use in individuals under 18 years.
  10. 10HumanObservational studySafety outcome

    In pediatric RTT trofinetide safety reports, higher doses were associated with psychiatric symptoms.

    In plain wordsIn pediatric RTT trofinetide safety reports, higher doses were linked to psychiatric symptoms.

    Peptide
    Trofinetide
    Outcome
    Direction not reportedPsychiatric symptom association with dose
    Study

    n: not reported in source

    Outcome group
    Adverse events · Mental health and mood
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Hypersomnia was more frequently reported in younger children (ROR = 12.11), and higher doses were associated with psychiatric symptoms.
    3 context sentences
    Summary · context
    This study evaluated the safety profile of trofinetide in pediatric patients with Rett syndrome using real-world pharmacovigilance data.
    Summary · context
    Adverse event (AE) reports were extracted from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) from Q1 2023 to Q4 2024.
    Summary · context
    Disproportionality analyses were conducted using ROR, PRR, BCPNN, and MGPS to detect AE signals associated with trofinetide use in individuals under 18 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

Subjects

Terms as the source lists them.

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