Trofinetide for the treatment of Rett syndrome: Results from the open-label extension LILAC study.

Percy AK, Neul JL, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, An D, Bishop KM, Youakim JM

Med (New York, N.Y.)24 Jun 2024Cited by 25 (Europe PMC)

PMID38917793DOI10.1016/j.medj.2024.05.018PepBankPUB-38917793

Publisher page PubMedAsk about this study

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 study8

8 results from studies that gave the peptide. 8 results from people.

01HumanNon-randomized trialSafety outcome
In females with Rett syndrome receiving trofinetide in LILAC, diarrhea occurred in 74.7%.
In plain wordsIn females with Rett syndrome receiving trofinetide, 74.7% had diarrhea.
Peptide
Trofinetide
Outcome
Not applicableDiarrhea
Study
n = 154
Analysis
Primary
Outcome group
Gastrointestinal adverse events · Gastrointestinal
Studied dose
Dose not stated · 40 weeksAs given in this study (human).
Measurements
Diarrhea
74.7%
LILAC
Source sentences
Summary
The most common adverse events in LILAC were diarrhea (74.7%), vomiting (28.6%), and COVID-19 (11.0%).
2 context sentences(design, population, supporting numbers)
Summary · context
Overall, 154 participants were enrolled and treated with trofinetide in LILAC.
Summary · context
Females with Rett syndrome aged 5-21 years received open-label treatment with trofinetide for 40 weeks.
Report a problemFND-3891779301
02HumanNon-randomized trialSafety outcome
In females with Rett syndrome receiving trofinetide in LILAC, vomiting occurred in 28.6%.
In plain wordsIn females with Rett syndrome receiving trofinetide, 28.6% had vomiting.
Peptide
Trofinetide
Outcome
Not applicableVomiting
Study
n = 154
Analysis
Primary
Outcome group
Gastrointestinal adverse events · Gastrointestinal
Studied dose
Dose not stated · 40 weeksAs given in this study (human).
Measurements
Vomiting
28.6%
LILAC
Source sentences
Summary
The most common adverse events in LILAC were diarrhea (74.7%), vomiting (28.6%), and COVID-19 (11.0%).
2 context sentences(design, population, supporting numbers)
Summary · context
Overall, 154 participants were enrolled and treated with trofinetide in LILAC.
Summary · context
Females with Rett syndrome aged 5-21 years received open-label treatment with trofinetide for 40 weeks.
Report a problemFND-3891779302
03HumanNon-randomized trialSafety outcome
In females with Rett syndrome receiving trofinetide in LILAC, COVID-19 occurred in 11.0%.
In plain wordsIn females with Rett syndrome receiving trofinetide, 11.0% had COVID-19.
Peptide
Trofinetide
Outcome
Not applicableCOVID-19
Study
n = 154
Analysis
Primary
Outcome group
Adverse events · Infection
Studied dose
Dose not stated · 40 weeksAs given in this study (human).
Measurements
COVID-19
11%
LILAC
Source sentences
Summary
The most common adverse events in LILAC were diarrhea (74.7%), vomiting (28.6%), and COVID-19 (11.0%).
2 context sentences(design, population, supporting numbers)
Summary · context
Overall, 154 participants were enrolled and treated with trofinetide in LILAC.
Summary · context
Females with Rett syndrome aged 5-21 years received open-label treatment with trofinetide for 40 weeks.
Report a problemFND-3891779303
04HumanNon-randomized trialSafety outcome
In females with Rett syndrome receiving trofinetide in LILAC, diarrhea led to treatment withdrawal in 21.4%.
In plain wordsIn females with Rett syndrome receiving trofinetide, 21.4% stopped treatment because of diarrhea.
Peptide
Trofinetide
Outcome
Not applicableWithdrawal due to diarrhea
Study
n = 154
Analysis
Primary
Outcome group
Gastrointestinal adverse events · Gastrointestinal
Studied dose
Dose not stated · 40 weeksAs given in this study (human).
Measurements
Withdrawal due to diarrhea
21.4%
LILAC
Source sentences
Summary
Diarrhea was the most common adverse event leading to treatment withdrawal (21.4%).
2 context sentences(design, population, supporting numbers)
Summary · context
Overall, 154 participants were enrolled and treated with trofinetide in LILAC.
Summary · context
Females with Rett syndrome aged 5-21 years received open-label treatment with trofinetide for 40 weeks.
Report a problemFND-3891779304
05HumanNon-randomized trialEfficacy outcome
In females with Rett syndrome previously receiving trofinetide, mean RSBQ change from LAVENDER baseline was -7.3 at LILAC week 40.
In plain wordsIn females with Rett syndrome previously receiving trofinetide, symptom scores fell by 7.3 from LAVENDER baseline at LILAC week 40.
Peptide
Trofinetide
Outcome
DecreaseRSBQ change at week 40, prior trofinetide
Study
n: not reported in source
Comparator
LAVENDER baseline
Analysis
Secondary
Outcome group
Rett Syndrome Symptoms · Nervous system and cognition
Studied dose
Dose not stated · 12 weeks in LAVENDER, then 40 weeks open-label in LILACAs given in this study (human).
Measurements
RSBQ change
−7.3 points
prior trofinetide
SE
1.62 points
prior trofinetide
Source sentences
Summary
The Rett Syndrome Behaviour Questionnaire mean score (standard error) improvement from the LAVENDER baseline to week 40 in LILAC was -7.3 (1.62) and -7.0 (1.61) for participants treated with trofinetide and placebo in LAVENDER, respectively.
1 context sentence(design, population, supporting numbers)
Summary · context
Females with Rett syndrome aged 5-21 years received open-label treatment with trofinetide for 40 weeks.
Report a problemFND-3891779305
06HumanNon-randomized trialEfficacy outcome
In females with Rett syndrome who received placebo in LAVENDER and then open-label trofinetide in LILAC, mean RSBQ change from LAVENDER baseline to LILAC week 40 was -7.0.
In plain wordsIn females with Rett syndrome who received placebo first, then trofinetide, symptom scores fell by 7.0 from the LAVENDER baseline to LILAC week 40.
Peptide
Trofinetide
Outcome
DecreaseRSBQ change at week 40, prior placebo
Study
n: not reported in source
Comparator
LAVENDER baseline
Analysis
Secondary
Outcome group
Rett Syndrome Symptoms · Nervous system and cognition
Studied dose
Dose not stated · 40 weeksAs given in this study (human).
Measurements
RSBQ change
−7 points
prior placebo
SE
1.61 points
prior placebo
Source sentences
Summary
The Rett Syndrome Behaviour Questionnaire mean score (standard error) improvement from the LAVENDER baseline to week 40 in LILAC was -7.3 (1.62) and -7.0 (1.61) for participants treated with trofinetide and placebo in LAVENDER, respectively.
1 context sentence(design, population, supporting numbers)
Summary · context
Females with Rett syndrome aged 5-21 years received open-label treatment with trofinetide for 40 weeks.
Report a problemFND-3891779306
07HumanNon-randomized trialEfficacy outcome
In females with Rett syndrome previously receiving trofinetide, mean CGI-I at LILAC week 40 was 3.1, rated from LILAC baseline.
In plain wordsIn females with Rett syndrome previously receiving trofinetide, mean improvement rating was 3.1 at LILAC week 40, relative to LILAC baseline.
Peptide
Trofinetide
Outcome
Not applicableCGI-I at week 40, prior trofinetide
Study
n: not reported in source
Analysis
Secondary
Outcome group
Rett Syndrome Symptoms · Nervous system and cognition
Studied dose
Dose not stated · 40 weeksAs given in this study (human).
Measurements
CGI-I
3.1 points
prior trofinetide
SE
0.11 points
prior trofinetide
Source sentences
Summary
Mean Clinical Global Impression-Improvement scores (standard error) at week 40 rated from the LILAC baseline were 3.1 (0.11) and 3.2 (0.14) for participants treated with trofinetide and placebo in LAVENDER, respectively.
1 context sentence(design, population, supporting numbers)
Summary · context
Females with Rett syndrome aged 5-21 years received open-label treatment with trofinetide for 40 weeks.
Report a problemFND-3891779307
08HumanNon-randomized trialEfficacy outcome
In females with Rett syndrome previously receiving placebo, mean CGI-I with trofinetide at LILAC week 40 was 3.2, rated from LILAC baseline.
In plain wordsIn females with Rett syndrome previously receiving placebo, mean improvement rating with trofinetide was 3.2 at LILAC week 40, relative to LILAC baseline.
Peptide
Trofinetide
Outcome
Not applicableCGI-I at week 40, prior placebo
Study
n: not reported in source
Analysis
Secondary
Outcome group
Rett Syndrome Symptoms · Nervous system and cognition
Studied dose
Dose not stated · 40 weeksAs given in this study (human).
Measurements
CGI-I
3.2 points
prior placebo
SE
0.14 points
prior placebo
Source sentences
Summary
Mean Clinical Global Impression-Improvement scores (standard error) at week 40 rated from the LILAC baseline were 3.1 (0.11) and 3.2 (0.14) for participants treated with trofinetide and placebo in LAVENDER, respectively.
1 context sentence(design, population, supporting numbers)
Summary · context
Females with Rett syndrome aged 5-21 years received open-label treatment with trofinetide for 40 weeks.
Report a problemFND-3891779308

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

CompletedPhase 3Results postedNCT04279314

Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

ACADIA Pharmaceuticals Inc.n = 15429 Jan 2020 to 19 Aug 2022

Rett Syndrome

Trial page ClinicalTrials.govUpdated 11 Apr 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.

Version 2Current
28 Oct 2026, 02:03 UTC
Study results updated
Version 1
27 Oct 2026, 16:37 UTC
First added · source update from Europe PMC

Subjects

Terms as the source lists them.

Publication types

MeSH terms

Keywords

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