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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 up to 5 years
Awards & highlights

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 ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

1Treatment groups
Experimental Treatment
Group I: Drug - trofinetideExperimental Treatment1 Intervention
trofinetide oral solution
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
trofinetide
2020
Completed Phase 3
~80

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
~16 spots leftby Sep 2025
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