Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
NCT ID: NCT04279314
Last Updated: 2024-04-11
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE3
154 participants
INTERVENTIONAL
2020-01-29
2022-08-19
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Open-Label Extension Study of Trofinetide for Rett Syndrome
NCT04776746
Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
NCT04181723
Open-Label Study of Uridine Triacetate in Pediatric Patients With Hereditary Orotic Aciduria
NCT02110147
Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome
NCT06856759
Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome
NCT00069550
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Trofinetide
Trofinetide
Trofinetide solution of 30-60 mL based on subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Trofinetide
Trofinetide solution of 30-60 mL based on subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
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
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
5 Years
21 Years
FEMALE
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
ACADIA Pharmaceuticals Inc.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
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
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Percy AK, Neul JL, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, An D, Bishop KM, Youakim JM. Trofinetide for the treatment of Rett syndrome: Results from the open-label extension LILAC study. Med. 2024 Sep 13;5(9):1178-1189.e3. doi: 10.1016/j.medj.2024.05.018. Epub 2024 Jun 24.
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.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
ACP-2566-004
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.