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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04776746
Other study ID # ACP-2566-005
Secondary ID
Status Completed
Phase Phase 3
First received
Last updated
Start date November 8, 2020
Est. completion date June 30, 2023

Study information

Verified date July 2023
Source ACADIA Pharmaceuticals Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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


Recruitment information / eligibility

Status Completed
Enrollment 78
Est. completion date June 30, 2023
Est. primary completion date June 30, 2023
Accepts healthy volunteers No
Gender Female
Age group 5 Years to 22 Years
Eligibility Inclusion Criteria: 1. Has completed the EOT visit of the antecedent trofinetide Study ACP-2566-004 (i.e., has completed 40 weeks) 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: 1. Began treatment with growth hormone during the antecedent study 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 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).

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
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).

Locations

Country Name City State
United States Emory Genetics Clinical Trial Center Atlanta Georgia
United States Children's Hospital Colorado Aurora Aurora Colorado
United States Kennedy Krieger Institute, John Hopkins School of Medicine Baltimore Maryland
United States University of Alabama at Birmingham Birmingham Alabama
United States Boston Children's Hospital Harvard Medical School Boston Massachusetts
United States Montefiore Medical Center Bronx New York
United States The University of North Carolina at Chapel Hill Chapel Hill North Carolina
United States Rush University Medical Center Chicago Illinois
United States Cincinnati Children's Hospital Medical Center Cincinnati Ohio
United States Cleveland Clinic Cleveland Ohio
United States Greenwood Genetic Center Greenwood South Carolina
United States Texas Children's Hospital Houston Texas
United States University of California, San Diego La Jolla California
United States Vanderbilt University Medical Center Nashville Tennessee
United States Children's Hospital of Philadelphia Philadelphia Pennsylvania
United States Translational Genomics Research Institute (TGen) Phoenix Arizona
United States UC Davis MIND Institute Sacramento California
United States Washington University School of Medicine Saint Louis Missouri
United States Gillette Children's Specialty Healthcare Saint Paul Minnesota
United States Seattle Children's Hospital Seattle Washington
United States University of South Florida Tampa Florida

Sponsors (1)

Lead Sponsor Collaborator
ACADIA Pharmaceuticals Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Percentage of subjects with treatment-emergent adverse events (TAEs), percentage of subjects with serious adverse events (SAEs), and percentage of subjects withdrawals due to adverse events (AEs) 32 months
Primary Number of subjects with post-Baseline potentially clinically important changes in ECG 32 months
Primary Percentage of subjects with post-Baseline potentially clinically important changes in ECG 32 months
Primary Number of subjects with post-Baseline potentially clinically important changes in vital signs 32 months
Primary Percentage of subjects with post-Baseline potentially clinically important changes in vital signs 32 months
Primary Number of subjects with post-Baseline potentially clinically important changes in body weight 32 months
Primary Percentage of subjects with post-Baseline potentially clinically important changes in body weight 32 months
Primary Number of subjects with post-Baseline potentially clinically important changes in laboratory parameters 32 months
Primary Percentage of subjects with post-Baseline potentially clinically important changes in laboratory parameters 32 months
See also
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