Open-Label Extension Study of Trofinetide for Rett Syndrome

Rett Syndrome Clinical Trial

Official title:

An Open-Label Extension Study of Continuing Treatment With Trofinetide for Rett Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04776746
• Other study ID #: ACP-2566-005
• Status: Completed
• Phase: Phase 3
• 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
• 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).

Related Conditions & MeSH terms

• Rett Syndrome
• Syndrome

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