Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
NCT ID: NCT04181723
Last Updated: 2024-04-08
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
187 participants
INTERVENTIONAL
2019-11-06
2021-10-28
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Drug - Trofinetide
Trofinetide solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
Trofinetide
Trofinetide solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Placebo
Trofinetide placebo solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
Placebo
Trofinetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Interventions
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Trofinetide
Trofinetide solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Placebo
Trofinetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Eligibility Criteria
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Inclusion Criteria
2. Body weight ≥12 kg at Screening
3. Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
4. Has classic/typical Rett syndrome (RTT)
5. Has a documented disease-causing mutation in the MECP2 gene
6. Has a stable pattern of seizures, or has had no seizures, within 8 weeks of Screening
7. 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.
8. The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
9. 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
Exclusion Criteria
2. 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
3. Has a history of, or current, cerebrovascular disease or brain trauma
4. Has significant, uncorrected visual or uncorrected hearing impairment
5. Has a history of, or current, malignancy
6. Has a known history or symptoms of long QT syndrome
5 Years
20 Years
FEMALE
No
Sponsors
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ACADIA Pharmaceuticals Inc.
INDUSTRY
Responsible Party
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Locations
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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
Countries
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References
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Darwish M, Passarell J, Youakim JM, Bradley H, Bishop KM. Exposure-Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome. Adv Ther. 2024 Apr;41(4):1462-1480. doi: 10.1007/s12325-024-02796-y. Epub 2024 Feb 16.
Parent H, Ferranti A, Niswender C. Trofinetide: a pioneering treatment for Rett syndrome. Trends Pharmacol Sci. 2023 Oct;44(10):740-741. doi: 10.1016/j.tips.2023.06.008. Epub 2023 Jul 16.
Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, Lin T, Stankovic S, Bishop KM, Youakim JM. Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study. Nat Med. 2023 Jun;29(6):1468-1475. doi: 10.1038/s41591-023-02398-1. Epub 2023 Jun 8.
Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Peters SU, Jones NE, Youakim JM. Design and outcome measures of LAVENDER, a phase 3 study of trofinetide for Rett syndrome. Contemp Clin Trials. 2022 Mar;114:106704. doi: 10.1016/j.cct.2022.106704. Epub 2022 Feb 8.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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ACP-2566-003
Identifier Type: -
Identifier Source: org_study_id
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