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Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome | Clinical Trials | Clareo Health

COMPLETEDPHASE3

Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

A 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

NCT04279314•ACADIA Pharmaceuticals Inc.

View on ClinicalTrials.gov

Summary

To investigate the safety and tolerability of 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 Week 12/End-of-treatment visit of the antecedent study, Study ACP-2566-003
•2. Met all entry criteria for the antecedent study
•3. May benefit from long-term treatment with open-label trofinetide in the judgment of the Investigator
•4. Can still swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
•5. The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
•6. Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 3 months prior to Baseline

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) or has major surgery planned during the study
•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 a QTcF interval of \>450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present 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, Colorado, United States

Children Medical Services

Tampa, Florida, United States

Emory Genetics Clinical Trial Center

Atlanta, Georgia, United States

Rush University Medical Center

Chicago, Illinois, United States

Kennedy Krieger Institute - Clinical Trials Unit

Baltimore, Maryland, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Key Dates

Start Date

January 29, 2020

Primary Completion Date

August 19, 2022

Completion Date

August 19, 2022

Last Updated

April 11, 2024

Enrollment

154

ACTUAL participants

Conditions

Rett Syndrome

Interventions

Trofinetide

DRUG

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

NCT06338267

Rett Syndrome

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RECRUITINGEARLY_PHASE1

Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome

NCT06856759

Rett Syndrome

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RECRUITING

GROWing Up With Rare GENEtic Syndromes

NCT04463316

Prader-Willi Syndrome

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: April 11, 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 the Treatment of Girls and Women With Rett Syndrome

Inclusion Criteria

Exclusion Criteria

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome

GROWing Up With Rare GENEtic Syndromes

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)

Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome