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Drug - Trofinetide for Rett Syndrome

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 baseline and week 12
Awards & highlights
Pivotal Trial

Summary

This trial is testing a possible new treatment for Rett syndrome, which is a disorder that affects mostly girls.

Eligible Conditions
  • Rett Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline and week 12
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline and week 12 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Clinical Global Impression-Improvement (CGI-I) Score at Week 12
Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
Secondary study objectives
Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S)
Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social)
Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score
+6 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
Placebo
Drug - Trofinetide

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Drug - TrofinetideExperimental Treatment1 Intervention
Trofinetide solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
Group II: PlaceboPlacebo Group1 Intervention
Trofinetide placebo solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
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
11,408 Total Patients Enrolled
3 Trials studying Rett Syndrome
246 Patients Enrolled for Rett Syndrome
~31 spots leftby Nov 2025
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