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TERMINATEDPHASE3

Open-Label Extension Study of Trofinetide for Rett Syndrome

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

NCT04776746•ACADIA Pharmaceuticals Inc.

Summary

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

Eligibility

Age

5 - 21 years

Sex

FEMALE

Healthy Volunteers

Inclusion Criteria

•1. Has completed the EOT visit of the antecedent trofinetide Study ACP-2566-004 (i.e., has completed 40 weeks)
•2. May benefit from continued treatment with open-label trofinetide in the judgment of the Investigator
•3. Can still swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
•4. The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
•Childbearing Potential
•5. Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter. If a subject is sexually active or becomes sexually active during the study, she must use 2 clinically acceptable methods of contraception (e.g., oral, intrauterine device \[IUD\], diaphragm plus spermicide, injectable, transdermal or implantable contraception) for the duration of the study and for at least 30 days thereafter. Subject must not be pregnant or breastfeeding.

Exclusion Criteria

•1. Began treatment with growth hormone during the antecedent study
•2. Began treatment with IGF-1 during the antecedent study
•3. Began treatment with insulin during the antecedent study
•4. Has developed a clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, Type 1 diabetes mellitus, or uncontrolled Type 2 diabetes mellitus), renal, hepatic, respiratory, or gastrointestinal disease (such as celiac disease or inflammatory bowel disease)
•5. Subject is judged by the Investigator or the Medical Monitor to be inappropriate for the study due to AEs, medical condition, or noncompliance with investigational product or study procedures in the antecedent study
•6. Has a clinically significant abnormality in vital signs at Baseline
•7. Has an average QTcF interval of \>450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present study (i.e., the ECG performed at the EOT visit of the antecedent study)
•8. Has developed a clinically significant ECG finding during the antecedent study

Locations (21)

University of Alabama at Birmingham

Birmingham, Alabama, United States

Translational Genomics Research Institute (TGen)

Phoenix, Arizona, United States

University of California, San Diego

La Jolla, California, United States

UC Davis MIND Institute

Sacramento, California, United States

Children's Hospital Colorado Aurora

Aurora, Colorado, United States

University of South Florida

Tampa, Florida, United States

Emory Genetics Clinical Trial Center

Atlanta, Georgia, United States

Rush University Medical Center

Chicago, Illinois, United States

Kennedy Krieger Institute, John Hopkins School of Medicine

Baltimore, Maryland, United States

Boston Children's Hospital Harvard Medical School

Boston, Massachusetts, United States

Key Dates

Start Date

November 8, 2020

Primary Completion Date

June 30, 2023

Completion Date

June 30, 2023

Last Updated

September 27, 2024

Enrollment

ACTUAL participants

Conditions

Rett Syndrome

Interventions

trofinetide

DRUG

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 27, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Open-Label Extension Study of Trofinetide for Rett Syndrome

Inclusion Criteria

Exclusion Criteria

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

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GROWing Up With Rare GENEtic Syndromes

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