Trofinetide for the treatment of Rett syndrome: Long-term safety and efficacy results of the 32-month, open-label LILAC-2 study.

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

Med (New York, N.Y.)17 Jul 2024Cited by 20 (Europe PMC)

PMID39025065DOI10.1016/j.medj.2024.06.007PepBankPUB-39025065

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 study6

6 results from studies that gave the peptide. 6 results from people.

01HumanNon-randomized trialSafety outcome
In females with Rett syndrome receiving trofinetide in LILAC-2, diarrhea occurred in 53.2%.
In plain wordsIn females with Rett syndrome receiving trofinetide, 53.2% had diarrhea in LILAC-2.
Peptide
Trofinetide
Outcome
Not applicableDiarrhea
Study
n = 77
Outcome group
Gastrointestinal adverse events · Gastrointestinal
Studied dose
Dose not stated
Measurements
Diarrhea
53.2%
LILAC-2
Source sentences
Summary
The most common AEs were diarrhea (53.2%), COVID-19 (27.3%), and vomiting (19.5%).
3 context sentences(design, population, supporting numbers)
Summary · context
In total, 77 participants were enrolled in LILAC-2.
Summary · context
Here, we report long-term safety and efficacy results of LILAC-2, a 32-month, open-label extension study.
Summary · context
Females aged 5-22 years who completed LILAC were eligible to enter LILAC-2.
Report a problemFND-3902506501
02HumanNon-randomized trialSafety outcome
In females with Rett syndrome receiving trofinetide in LILAC-2, COVID-19 occurred in 27.3%.
In plain wordsIn females with Rett syndrome receiving trofinetide, 27.3% had COVID-19 in LILAC-2.
Peptide
Trofinetide
Outcome
Not applicableCOVID-19
Study
n = 77
Outcome group
Adverse events · Infection
Studied dose
Dose not stated
Measurements
COVID-19
27.3%
LILAC-2
Source sentences
Summary
The most common AEs were diarrhea (53.2%), COVID-19 (27.3%), and vomiting (19.5%).
3 context sentences(design, population, supporting numbers)
Summary · context
In total, 77 participants were enrolled in LILAC-2.
Summary · context
Here, we report long-term safety and efficacy results of LILAC-2, a 32-month, open-label extension study.
Summary · context
Females aged 5-22 years who completed LILAC were eligible to enter LILAC-2.
Report a problemFND-3902506502
03HumanNon-randomized trialSafety outcome
In females with Rett syndrome receiving trofinetide in LILAC-2, vomiting occurred in 19.5%.
In plain wordsIn females with Rett syndrome receiving trofinetide, 19.5% had vomiting in LILAC-2.
Peptide
Trofinetide
Outcome
Not applicableVomiting
Study
n = 77
Outcome group
Gastrointestinal adverse events · Gastrointestinal
Studied dose
Dose not stated
Measurements
Vomiting
19.5%
LILAC-2
Source sentences
Summary
The most common AEs were diarrhea (53.2%), COVID-19 (27.3%), and vomiting (19.5%).
3 context sentences(design, population, supporting numbers)
Summary · context
In total, 77 participants were enrolled in LILAC-2.
Summary · context
Here, we report long-term safety and efficacy results of LILAC-2, a 32-month, open-label extension study.
Summary · context
Females aged 5-22 years who completed LILAC were eligible to enter LILAC-2.
Report a problemFND-3902506503
04HumanNon-randomized trialEfficacy outcome
In females with Rett syndrome receiving trofinetide, mean RSBQ change from LAVENDER baseline was -11.8 at LILAC-2 week 104.
In plain wordsIn females with Rett syndrome receiving trofinetide, symptom scores fell by 11.8 from LAVENDER baseline at LILAC-2 week 104.
Peptide
Trofinetide
Outcome
DecreaseRSBQ change at week 104
Study
n: not reported in source
Comparator
LAVENDER baseline
Outcome group
Rett Syndrome Symptoms · Nervous system and cognition
Studied dose
Dose not stated
Measurements
RSBQ change
−11.8 points
week 104
SE
2.45 points
week 104
Source sentences
Summary
The mean (standard error [SE]) change in RSBQ score from LAVENDER baseline to week 104 of LILAC-2 was -11.8 (2.45).
2 context sentences(design, population, supporting numbers)
Summary · context
Here, we report long-term safety and efficacy results of LILAC-2, a 32-month, open-label extension study.
Summary · context
Females aged 5-22 years who completed LILAC were eligible to enter LILAC-2.
Report a problemFND-3902506504
05HumanNon-randomized trialEfficacy outcome
In females with Rett syndrome receiving trofinetide, mean CGI-I was 3.1 at LILAC-2 week 12, relative to LILAC baseline.
In plain wordsIn females with Rett syndrome receiving trofinetide, mean improvement rating was 3.1 at LILAC-2 week 12, relative to LILAC baseline.
Peptide
Trofinetide
Outcome
Not applicableCGI-I at week 12
Study
n: not reported in source
Outcome group
Rett Syndrome Symptoms · Nervous system and cognition
Studied dose
Dose not stated
Measurements
CGI-I
3.1 points
week 12
SE
0.1 points
week 12
Source sentences
Summary
The mean (SE) CGI-I score from LILAC baseline to week 12 of LILAC-2 was 3.1 (0.10).
2 context sentences(design, population, supporting numbers)
Summary · context
Here, we report long-term safety and efficacy results of LILAC-2, a 32-month, open-label extension study.
Summary · context
Females aged 5-22 years who completed LILAC were eligible to enter LILAC-2.
Report a problemFND-3902506505
06HumanObservational studyEfficacy outcome
Among caregivers of females with Rett syndrome receiving trofinetide, 24 of 25 were satisfied or very satisfied with its benefits.
In plain wordsAmong caregivers of females with Rett syndrome receiving trofinetide, 96% were satisfied or very satisfied with its benefits.
Peptide
Trofinetide
Outcome
Not applicableCaregiver satisfaction
Study
n = 25
Outcome group
Patient-reported outcomes · Nervous system and cognition
Studied dose
Dose not stated
Measurements
Satisfied or very satisfied
96%
caregivers
Satisfied caregivers
24 caregivers
25 interviewed
Source sentences
Summary
Most caregivers (96%; n = 24/25) were satisfied or very satisfied with the benefits of trofinetide.
3 context sentences(design, population, supporting numbers)
Summary · context
Caregiver interviews explored the patient's experience with RTT and the efficacy of trofinetide during study participation.
Summary · context
Here, we report long-term safety and efficacy results of LILAC-2, a 32-month, open-label extension study.
Summary · context
Females aged 5-22 years who completed LILAC were eligible to enter LILAC-2.
Report a problemFND-3902506506

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 3Results postedNCT04776746

Open-Label Extension Study of Trofinetide for Rett Syndrome

ACADIA Pharmaceuticals Inc.n = 778 Nov 2020 to 30 Jun 2023

Rett Syndrome

Trial page ClinicalTrials.govUpdated 27 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.

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

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