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An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome (DAFFODIL™ Trial)

Phase 2 & 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 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up mean study drug exposure 434 days, corresponding to 1.2 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a medicine called trofinetide, which is taken by mouth, to see if it is safe for extended use in girls with Rett syndrome. The medicine aims to help the brain work better and reduce the symptoms of the disorder.

Eligible Conditions
  • Rett Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~mean study drug exposure 434 days, corresponding to 1.2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and mean study drug exposure 434 days, corresponding to 1.2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
AUC0-12,ss (Area Under the Concentration-time Curve From Time 0 to 12 h at Steady State)
Cmax,ss (Maximum Observed Drug Concentration at Steady State)
Cmin,ss (Minimum Observed Drug Concentration at Steady State of Oral Trofinetide)
+2 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%
Bacteraemia
1%
Bronchiolitis
1%
COVID-19 pneumonia
1%
Urinary tract infection
100%
80%
60%
40%
20%
0%
Study treatment Arm
Drug - Trofinetide
Placebo

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Drug - trofinetideExperimental Treatment1 Intervention
Oral dose of trofinetide
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Trofinetide
2020
Completed Phase 3
~350

Find a Location

Who is running the clinical trial?

ACADIA Pharmaceuticals Inc.Lead Sponsor
47 Previous Clinical Trials
11,589 Total Patients Enrolled
3 Trials studying Rett Syndrome
418 Patients Enrolled for Rett Syndrome
~4 spots leftby Dec 2025
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