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

Completed · Phase 3

Conditions studied: Rett Syndrome

In brief

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

Key facts

Study ID

NCT04279314

Run by

ACADIA Pharmaceuticals Inc.

People needed

154

Starts

2020-01-29

Expected to finish

2022-08-19

Last updated by the study team

2024-04-11

Who can join

Age: 5 and older, up to 21. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• Has completed the Week 12/End-of-treatment visit of the antecedent study, Study ACP-2566-003
• Met all entry criteria for the antecedent study
• May benefit from long-term treatment with open-label trofinetide in the judgment of the Investigator
• Can still swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
• The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
• 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

You may not qualify if…

• Began treatment with growth hormone during the antecedent study
• Began treatment with IGF-1 during the antecedent study
• Began treatment with insulin during the antecedent study
• 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
• 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
• Has a clinically significant abnormality in vital signs at Baseline
• Has a QTcF interval of >450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present study
• Has developed a clinically significant ECG finding during the antecedent study
• Additional inclusion/exclusion criteria apply. Patients will be evaluated at baseline to ensure that all criteria for study participation are met. Patients may be excluded from the study based on these assessments (and specifically, if it is determined that their baseline health and condition do not meet all prespecified entry criteria).

Where it is running

• 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
• Gillette Children's Specialty Healthcare — Saint Paul, Minnesota, United States
• Washington University School of Medicine, St. Louis Children's Hospital — St Louis, Missouri, United States
• Montefiore Medical Center, Children's Hospital at Montefiore — The Bronx, New York, United States
• The University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Cleveland Clinic — Cleveland, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Greenwood Genetic Center — Greenwood, South Carolina, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• Texas Children's Hospital — Houston, Texas, United States
• Seattle Children's — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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