Clinical trial NCT04279314: Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Official title: A 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Rett Syndrome
Lead sponsor: ACADIA Pharmaceuticals Inc.
Sponsor class: INDUSTRY
First posted: 2020-02-21
Enrollment: 154
Sex: FEMALE
Minimum age (years): 5
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: Inclusion Criteria:

1. Has completed the Week 12/End-of-treatment visit of the antecedent study, Study ACP-2566-003
2. Met all entry criteria for the antecedent study
3. May benefit from long-term treatment with open-label trofinetide in the judgment of the Investigator
4. Can still swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
5. The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
6. Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 3 months prior to Baseline

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) or has major surgery planned during the study
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 a QTcF interval of \>450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present study
8. Has developed a clinically significant ECG finding during the antecedent study

Additional inclusion/exclusion criteria apply. Patients will be evaluated at baseline 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 prespecified entry criteria).

Primary outcomes:
measure: Percentage of Subjects With Treatment-emergent Adverse Events (TEAEs), Percentage of Subjects With Serious Adverse Events (SAEs), and Percentage of Subjects With Withdrawals Due to AEs
timeFrame: 40 Weeks Treatment Duration
measure: Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in ECG
timeFrame: 40 Weeks Treatment Duration
measure: Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs
timeFrame: 40 Weeks Treatment Duration
measure: Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Body Weight
timeFrame: 40 Weeks Treatment Duration
measure: Subjects (N, %) With Post-baseline Potentially Clinically Important Changes
timeFrame: 40 Weeks Treatment Duration
Secondary outcomes:
measure: Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score Change From Baseline to Week 40
timeFrame: 40 Weeks Treatment Duration
measure: Clinical Global Impression-Improvement (CGI-I) Score at Week 40
timeFrame: 40 Weeks Treatment Duration
measure: Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) Change From Baseline to Week 40
timeFrame: 40 Weeks Treatment Duration
measure: Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND) Change From Baseline to Week 40
timeFrame: 40 Weeks Treatment Duration
measure: Rett Syndrome Clinician Rating of Hand Function (RTT-HF) Change From Baseline to Week 40
timeFrame: 40 Weeks Treatment Duration
measure: Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB) Change From Baseline to Week 40
timeFrame: 40 Weeks Treatment Duration
measure: Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) Change From Baseline to Week 40
timeFrame: 40 Weeks Treatment Duration
measure: Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM) Change From Baseline to Week 40
timeFrame: 40 Weeks Treatment Duration
measure: Change From Baseline to Week 40 in Clinical Global Impression-Severity (CGI-S)
timeFrame: 40 Weeks Treatment Duration
measure: Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24) Change From Baseline to Week 40
timeFrame: 40 Weeks Treatment Duration
measure: Impact of Childhood Neurologic Disability Scale (ICND) Total Score Change From Baseline to Week 40
timeFrame: 40 Weeks Treatment Duration
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Translational Genomics Research Institute (TGen)
state: Arizona
city: La Jolla
country: United States
facility: University of California, San Diego
state: California
city: Sacramento
country: United States
facility: UC Davis MIND Institute
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Tampa
country: United States
facility: Children Medical Services
state: Florida
city: Atlanta
country: United States
facility: Emory Genetics Clinical Trial Center
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Baltimore
country: United States
facility: Kennedy Krieger Institute - Clinical Trials Unit
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Saint Paul
country: United States
facility: Gillette Children's Specialty Healthcare
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine, St. Louis Children's Hospital
state: Missouri
city: Bronx
country: United States
facility: Montefiore Medical Center, Children's Hospital at Montefiore
state: New York
city: Chapel Hill
country: United States
facility: The University of North Carolina at Chapel Hill
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Greenwood
country: United States
facility: Greenwood Genetic Center
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Seattle
country: United States
facility: Seattle Children's
state: Washington
Study officials:
References: