Open-Label Extension Study of Trofinetide for Rett Syndrome

• ClinicalTrials.gov Identifier: NCT04776746
• Recruitment Status: Completed
• First Posted: March 2, 2021
• Last Update Posted: August 1, 2023
• Sponsor: ACADIA Pharmaceuticals Inc.
• Information provided by (Responsible Party): ACADIA Pharmaceuticals Inc.

Tracking Information

• First Submitted Date: November 29, 2020
• First Posted Date: March 2, 2021
• Last Update Posted Date: August 1, 2023
• Actual Study Start Date: November 8, 2020
• Actual Primary Completion Date: June 30, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 25, 2021)

• 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) [ Time Frame: 32 months ]
• Number of subjects with post-Baseline potentially clinically important changes in ECG [ Time Frame: 32 months ]
• Percentage of subjects with post-Baseline potentially clinically important changes in ECG [ Time Frame: 32 months ]
• Number of subjects with post-Baseline potentially clinically important changes in vital signs [ Time Frame: 32 months ]
• Percentage of subjects with post-Baseline potentially clinically important changes in vital signs [ Time Frame: 32 months ]
• Number of subjects with post-Baseline potentially clinically important changes in body weight [ Time Frame: 32 months ]
• Percentage of subjects with post-Baseline potentially clinically important changes in body weight [ Time Frame: 32 months ]
• Number of subjects with post-Baseline potentially clinically important changes in laboratory parameters [ Time Frame: 32 months ]
• Percentage of subjects with post-Baseline potentially clinically important changes in laboratory parameters [ Time Frame: 32 months ]

Original Primary Outcome Measures (submitted: February 26, 2021)

• 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) [ Time Frame: 32 months ]
• Number and percentage of subjects with post-Baseline potentially clinically important changes in ECG [ Time Frame: 32 months ]
• Number and percentage of subjects with post-Baseline potentially clinically important changes in vital signs [ Time Frame: 32 months ]
• Number and percentage of subjects with post-Baseline potentially clinically important changes in body weight [ Time Frame: 32 months ]
• Number and percentage of subjects with post-Baseline potentially clinically important changes in laboratory parameters [ Time Frame: 32 months ]

• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Open-Label Extension Study of Trofinetide for Rett Syndrome
• Official Title: An Open-Label Extension Study of Continuing Treatment With Trofinetide for Rett Syndrome
• Brief Summary: To investigate the safety and tolerability of continued long-term treatment with oral trofinetide in girls and women with Rett syndrome
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Rett 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).

Study Arms

Experimental: Drug - trofinetide

trofinetide oral solution

Intervention: Drug: trofinetide

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: April 2, 2023): 78
• Original Estimated Enrollment (submitted: February 26, 2021): 153
• Actual Study Completion Date: June 30, 2023
• Actual Primary Completion Date: June 30, 2023 (Final data collection date for primary outcome measure)

Eligibility Criteria

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).

Sex/Gender

• Sexes Eligible for Study: Female
• Gender Based Eligibility: Yes

• Ages: 5 Years to 22 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04776746
• Other Study ID Numbers: ACP-2566-005
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: ACADIA Pharmaceuticals Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: ACADIA Pharmaceuticals Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: ACADIA Pharmaceuticals Inc.
• Verification Date: July 2023