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Terminated Phase 3 Interventional Results available

Open-Label Extension Study of Trofinetide for Rett Syndrome

ClinicalTrials.gov ID: NCT04776746

Public ClinicalTrials.gov record NCT04776746. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Oct 20, 2026, 6:12 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

An Open-Label Extension Study of Continuing Treatment With Trofinetide for Rett Syndrome

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

To investigate the safety and tolerability of continued long-term treatment with oral trofinetide in girls and women with Rett syndrome

Study identification

NCT ID
NCT04776746
Recruitment status
Terminated
Study type
Interventional
Phase
Phase 3
Lead sponsor
ACADIA Pharmaceuticals Inc.
Industry
Enrollment
77 participants

Conditions and interventions

Conditions

Interventions

Drug

Eligibility (public fields only)

Age range
5 Years to 21 Years
Sex
Female
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Nov 8, 2020 (Actual)
Primary completion
Jun 30, 2023 (Actual)
Completion
Jun 30, 2023 (Actual)
Last update posted
Sep 27, 2024

2020 – 2023

United States locations

U.S. sites
21
U.S. states
19
U.S. cities
21
Facility City State ZIP Site status
University of Alabama at Birmingham Birmingham Alabama 35233 —
Translational Genomics Research Institute (TGen) Phoenix Arizona 85012 —
University of California, San Diego La Jolla California 92037 —
UC Davis MIND Institute Sacramento California 95817 —
Children's Hospital Colorado Aurora Aurora Colorado 80045 —
University of South Florida Tampa Florida 33612 —
Emory Genetics Clinical Trial Center Atlanta Georgia 30322 —
Rush University Medical Center Chicago Illinois 60612 —
Kennedy Krieger Institute, John Hopkins School of Medicine Baltimore Maryland 21205 —
Boston Children's Hospital Harvard Medical School Boston Massachusetts 02115 —
Gillette Children's Specialty Healthcare Saint Paul Minnesota 55101 —
Washington University School of Medicine St Louis Missouri 63110 —
Montefiore Medical Center The Bronx New York 10467 —
The University of North Carolina at Chapel Hill Chapel Hill North Carolina 27599 —
Cincinnati Children's Hospital Medical Center Cincinnati Ohio 45229 —
Cleveland Clinic Cleveland Ohio 44195 —
Children's Hospital of Philadelphia Philadelphia Pennsylvania 19104 —
Greenwood Genetic Center Greenwood South Carolina 29646 —
Vanderbilt University Medical Center Nashville Tennessee 37232 —
Texas Children's Hospital Houston Texas 77030 —
Seattle Children's Hospital Seattle Washington 98105 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT04776746, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Sep 27, 2024 · Synced Oct 20, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT04776746 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →