Clinical trial
A 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Name
ACP-2566-004
Description
To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls and women with Rett syndrome
Trial arms
Trial start
2020-01-29
Estimated PCD
2022-08-19
Trial end
2022-08-19
Status
Completed
Phase
Early phase I
Treatment
Trofinetide
Trofinetide solution of 30-60 mL based on subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
Arms:
Trofinetide
Size
154
Primary endpoint
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
40 Weeks Treatment Duration
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in ECG
40 Weeks Treatment Duration
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs
40 Weeks Treatment Duration
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Body Weight
40 Weeks Treatment Duration
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes
40 Weeks Treatment Duration
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).
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 154, 'type': 'ACTUAL'}}
Updated at
2024-04-11
1 organization
Organization
Acadia Pharmaceuticals Inc.