Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)

Completed · Phase 3 · Has a placebo group

Conditions studied: Rett Syndrome

In brief

To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome

Key facts

Study ID

NCT04181723

Run by

ACADIA Pharmaceuticals Inc.

People needed

187

Starts

2019-11-06

Expected to finish

2021-10-28

Last updated by the study team

2024-04-08

Who can join

Age: 5 and older, up to 20. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• Female subjects 5 to 20 years of age, inclusive, at Screening
• Body weight ≥12 kg at Screening
• Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
• Has classic/typical Rett syndrome (RTT)
• Has a documented disease-causing mutation in the MECP2 gene
• Has a stable pattern of seizures, or has had no seizures, within 8 weeks of Screening
• Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter or must agree to use acceptable methods of contraception. Subject must not be pregnant or breastfeeding.
• 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 Screening

You may not qualify if…

• Has been treated with insulin within 12 weeks of Baseline
• Has current 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
• Has a history of, or current, cerebrovascular disease or brain trauma
• Has significant, uncorrected visual or uncorrected hearing impairment
• Has a history of, or current, malignancy
• Has a known history or symptoms of long QT syndrome
• Additional inclusion/exclusion criteria apply. Patients will be evaluated at screening 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 pre-specified entry criteria).

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• Translational Gemomics 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 Children's Hospital — Chicago, Illinois, United States
• Kennedy Krieger Institute - Clinical Trials Unit — Baltimore, Maryland, United States
• Boston Children's Hospital Harvard Medical School — Boston, Massachusetts, United States
• Gillette Children's Specialty Healthcare — Saint Paul, Minnesota, United States
• Washington University School of Medicine — 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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