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Trofinetide for Rett Syndrome (LILAC™ Trial)

Phase 3
Waitlist Available
Research Sponsored by ACADIA Pharmaceuticals Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 40 weeks treatment duration
Awards & highlights

LILAC™ Trial Summary

This trial will study the safety of a drug called trofinetide in girls and women with Rett syndrome.

Eligible Conditions
  • Rett Syndrome

LILAC™ Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~40 weeks treatment duration
This trial's timeline: 3 weeks for screening, Varies for treatment, and 40 weeks treatment duration for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Percentage of Subjects With Treatment-emergent Adverse Events (TEAEs), Percentage of Subjects With Serious Adverse Events (SAEs), and Percentage of Subjects With Withdrawals Due to AEs
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Body Weight
+2 more
Secondary outcome measures
Change From Baseline to Week 40 in Clinical Global Impression-Severity (CGI-S)
Clinical Global Impression-Improvement (CGI-I) Score at Week 40
Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) Change From Baseline to Week 40
+8 more

Side effects data

From 2021 Phase 3 trial • 187 Patients • NCT04181723
81%
Diarrhoea
27%
Vomiting
9%
Pyrexia
8%
Seizure
6%
Irritability
5%
Decreased appetite
1%
Bronchiolitis
1%
Bacteraemia
1%
Urinary tract infection
1%
COVID-19 pneumonia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Drug - Trofinetide
Placebo

LILAC™ Trial Design

1Treatment groups
Experimental Treatment
Group I: TrofinetideExperimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Trofinetide
2020
Completed Phase 3
~360

Find a Location

Who is running the clinical trial?

ACADIA Pharmaceuticals Inc.Lead Sponsor
46 Previous Clinical Trials
10,786 Total Patients Enrolled
3 Trials studying Rett Syndrome
280 Patients Enrolled for Rett Syndrome

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
2020. "Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04279314.
~30 spots leftby Apr 2025
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