Open-Label Extension Study of Trofinetide for Rett Syndrome
NCT ID: NCT04776746
Last Updated: 2024-09-27
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE3
77 participants
INTERVENTIONAL
2020-11-08
2023-06-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Drug - trofinetide
trofinetide oral solution
trofinetide
Study drug is administered twice a day for up to approximately 32 months. Doses may be taken orally or administered by gastrostomy (G) tube. The subject's assigned dose for this study will be the final dose from the antecedent study (ACP-2566-004).
Interventions
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trofinetide
Study drug is administered twice a day for up to approximately 32 months. Doses may be taken orally or administered by gastrostomy (G) tube. The subject's assigned dose for this study will be the final dose from the antecedent study (ACP-2566-004).
Eligibility Criteria
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Inclusion Criteria
2. May benefit from continued treatment with open-label trofinetide in the judgment of the Investigator
3. Can still swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
4. The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
Childbearing Potential
5. Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter. If a subject is sexually active or becomes sexually active during the study, she must use 2 clinically acceptable methods of contraception (e.g., oral, intrauterine device \[IUD\], diaphragm plus spermicide, injectable, transdermal or implantable contraception) for the duration of the study and for at least 30 days thereafter. Subject must not be pregnant or breastfeeding.
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)
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 an average QTcF interval of \>450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present study (i.e., the ECG performed at the EOT visit of the antecedent study)
8. Has developed a clinically significant ECG finding during the antecedent study
5 Years
21 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 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
Aurora, Colorado, United States
University of South Florida
Tampa, Florida, United States
Emory Genetics Clinical Trial Center
Atlanta, Georgia, United States
Rush University Medical Center
Chicago, Illinois, United States
Kennedy Krieger Institute, John Hopkins School of Medicine
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
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 Hospital
Seattle, Washington, United States
Countries
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References
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Percy AK, Neul JL, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, Barrett AM, An D, Bishop KM, Youakim JM. Trofinetide for the treatment of Rett syndrome: Long-term safety and efficacy results of the 32-month, open-label LILAC-2 study. Med. 2024 Oct 11;5(10):1275-1281.e2. doi: 10.1016/j.medj.2024.06.007. Epub 2024 Jul 17.
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
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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ACP-2566-005
Identifier Type: -
Identifier Source: org_study_id
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