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Open-Label Extension Study of Trofinetide for Rett Syndrome

Primary Purpose

Rett Syndrome

Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
trofinetide
Sponsored by
ACADIA Pharmaceuticals Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Rett Syndrome

Eligibility Criteria

5 Years - 22 Years (Child, Adult)FemaleDoes not accept healthy volunteers

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).

Sites / Locations

  • University of Alabama at Birmingham
  • Translational Genomics Research Institute (TGen)
  • University of California, San Diego
  • UC Davis MIND Institute
  • Children's Hospital Colorado Aurora
  • University of South Florida
  • Emory Genetics Clinical Trial Center
  • Rush University Medical Center
  • Kennedy Krieger Institute, John Hopkins School of Medicine
  • Boston Children's Hospital Harvard Medical School
  • Gillette Children's Specialty Healthcare
  • Washington University School of Medicine
  • Montefiore Medical Center
  • The University of North Carolina at Chapel Hill
  • Cincinnati Children's Hospital Medical Center
  • Cleveland Clinic
  • Children's Hospital of Philadelphia
  • Greenwood Genetic Center
  • Vanderbilt University Medical Center
  • Texas Children's Hospital
  • Seattle Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Drug - trofinetide

Arm Description

trofinetide oral solution

Outcomes

Primary Outcome Measures

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)
Number of subjects with post-Baseline potentially clinically important changes in ECG
Percentage of subjects with post-Baseline potentially clinically important changes in ECG
Number of subjects with post-Baseline potentially clinically important changes in vital signs
Percentage of subjects with post-Baseline potentially clinically important changes in vital signs
Number of subjects with post-Baseline potentially clinically important changes in body weight
Percentage of subjects with post-Baseline potentially clinically important changes in body weight
Number of subjects with post-Baseline potentially clinically important changes in laboratory parameters
Percentage of subjects with post-Baseline potentially clinically important changes in laboratory parameters

Secondary Outcome Measures

Full Information

First Posted
November 29, 2020
Last Updated
July 28, 2023
Sponsor
ACADIA Pharmaceuticals Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04776746
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Completed
Study Start Date
November 8, 2020 (Actual)
Primary Completion Date
June 30, 2023 (Actual)
Study Completion Date
June 30, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ACADIA Pharmaceuticals Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Rett Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
78 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Drug - trofinetide
Arm Type
Experimental
Arm Description
trofinetide oral solution
Intervention Type
Drug
Intervention Name(s)
trofinetide
Intervention Description
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).
Primary Outcome Measure Information:
Title
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
Title
Number of subjects with post-Baseline potentially clinically important changes in ECG
Time Frame
32 months
Title
Percentage of subjects with post-Baseline potentially clinically important changes in ECG
Time Frame
32 months
Title
Number of subjects with post-Baseline potentially clinically important changes in vital signs
Time Frame
32 months
Title
Percentage of subjects with post-Baseline potentially clinically important changes in vital signs
Time Frame
32 months
Title
Number of subjects with post-Baseline potentially clinically important changes in body weight
Time Frame
32 months
Title
Percentage of subjects with post-Baseline potentially clinically important changes in body weight
Time Frame
32 months
Title
Number of subjects with post-Baseline potentially clinically important changes in laboratory parameters
Time Frame
32 months
Title
Percentage of subjects with post-Baseline potentially clinically important changes in laboratory parameters
Time Frame
32 months

10. Eligibility

Sex
Female
Gender Based
Yes
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
22 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Has completed the EOT visit of the antecedent trofinetide Study ACP-2566-004 (i.e., has completed 40 weeks) May benefit from continued treatment with open-label trofinetide in the judgment of the Investigator Can still swallow the study medication provided as a liquid solution or can take it by gastrostomy tube The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments Childbearing Potential 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: Began treatment with growth hormone during the antecedent study Began treatment with IGF-1 during the antecedent study Began treatment with insulin during the antecedent study 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) 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 Has a clinically significant abnormality in vital signs at Baseline 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) 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).
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Translational Genomics Research Institute (TGen)
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85012
Country
United States
Facility Name
University of California, San Diego
City
La Jolla
State/Province
California
ZIP/Postal Code
92093
Country
United States
Facility Name
UC Davis MIND Institute
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Children's Hospital Colorado Aurora
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Emory Genetics Clinical Trial Center
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Kennedy Krieger Institute, John Hopkins School of Medicine
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Boston Children's Hospital Harvard Medical School
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Gillette Children's Specialty Healthcare
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55101
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Montefiore Medical Center
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Facility Name
The University of North Carolina at Chapel Hill
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Cleveland Clinic
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Greenwood Genetic Center
City
Greenwood
State/Province
South Carolina
ZIP/Postal Code
29646
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Open-Label Extension Study of Trofinetide for Rett Syndrome

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