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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 Sep 4, 2026, 8:10 AM 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 7, 2020
Primary completion
Jun 29, 2023
Completion
Jun 29, 2023
Last update posted
Sep 26, 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 26, 2024 · Synced Sep 4, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT04776746 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →