Stopped: As a result of marketing approval of trofinetide on 10 March 2023, the study was terminated by the Sponsor with the intent of switching patients to commercially available product.
To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls with Rett syndrome
Age range
2 Years – 5 Years
Sex
FEMALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Safety and Tolerability of Treatment With Oral Trofinetide
Timeframe: Mean study drug exposure 434 days, corresponding to 1.2 years
AUC0-12,ss (Area Under the Concentration-time Curve From Time 0 to 12 h at Steady State)
Timeframe: PK samples were taken predose and at Weeks 2, 4, 8, and 12
Cmax,ss (Maximum Observed Drug Concentration at Steady State)
Timeframe: PK samples were taken predose and at Weeks 2, 4, 8, and 12
Cmin,ss (Minimum Observed Drug Concentration at Steady State of Oral Trofinetide)
Timeframe: PK samples were taken predose and at Weeks 2, 4, 8, and 12
Tmax (Time of the Maximum Observed Drug Concentration at Steady State)
Timeframe: PK samples were taken predose and at Weeks 2, 4, 8, and 12