← Back to Search

Other

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 mean study drug exposure was 426 days, corresponding to 1.2 years
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial is studying the safety of an oral medication called trofinetide in girls and women with Rett syndrome. The goal is to see if it can help improve brain function and reduce symptoms over time.

Eligible Conditions
  • Rett Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~mean study drug exposure was 426 days, corresponding to 1.2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and mean study drug exposure was 426 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
Number (%) of Patients With Potentially Clinically Important Changes in Body Weight Post-baseline
Number (%) of Patients With Potentially Clinically Important Changes in ECG Post-baseline
Number (%) of Patients With Potentially Clinically Important Changes in Laboratory Parameters Post-baseline
+2 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Drug - trofinetideExperimental Treatment1 Intervention
trofinetide oral solution

Find a Location

Who is running the clinical trial?

ACADIA Pharmaceuticals Inc.Lead Sponsor
47 Previous Clinical Trials
11,518 Total Patients Enrolled
3 Trials studying Rett Syndrome
356 Patients Enrolled for Rett Syndrome
~15 spots leftby Dec 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top