Phase 3
N=154
Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Rett Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT04279314 ↗Enrolled (actual)
154
Serious AEs
12.3%
Results posted
Apr 2024
Primary outcome: Primary: 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 — 132; 19; 48 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Trofinetide (Drug)
- Age
- Pediatric, Adult · 5+ yrs
- Sex
- Female
- Sponsor
- ACADIA Pharmaceuticals Inc.
- Primary completion
- Aug 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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 |
132; 19; 48 | — |
| PRIMARY Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in ECG |
2 | — |
| PRIMARY Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs |
0; 11; 0; 13; 14; 0 | — |
| PRIMARY Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Body Weight |
44; 20 | — |
| PRIMARY Subjects (N, %) With Post-baseline Potentially Clinically Important Changes |
0; 1; 0; 0; 0; 1 | — |
| SECONDARY Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score Change From Baseline to Week 40 |
-2.9 | — |
| SECONDARY Clinical Global Impression-Improvement (CGI-I) Score at Week 40 |
3.2; 3.1 | — |
| SECONDARY Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) Change From Baseline to Week 40 |
0.5 | — |
| SECONDARY Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND) Change From Baseline to Week 40 |
0.2 | — |
| SECONDARY Rett Syndrome Clinician Rating of Hand Function (RTT-HF) Change From Baseline to Week 40 |
-0.1 | — |
| SECONDARY Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB) Change From Baseline to Week 40 |
-0.2 | — |
| SECONDARY Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) Change From Baseline to Week 40 |
-0.4 | — |
| SECONDARY Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM) Change From Baseline to Week 40 |
-0.2 | — |
| SECONDARY Change From Baseline to Week 40 in Clinical Global Impression-Severity (CGI-S) |
-0.1 | — |
| SECONDARY Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24) Change From Baseline to Week 40 |
-1.9 | — |
| SECONDARY Impact of Childhood Neurologic Disability Scale (ICND) Total Score Change From Baseline to Week 40 |
-5.8 | — |
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
- Has completed the Week 12/End-of-treatment visit of the antecedent study, Study ACP-2566-003
- Met all entry criteria for the antecedent study
- May benefit from long-term 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
- 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
- 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) or has major surgery planned during the study
- 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 a QTcF interval of >450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present study
- 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).
Data sourced from ClinicalTrials.gov (NCT04279314). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.