Study of CSTI-500 for Prader-Willi Syndrome
This study is testing CSTI-500, given by mouth, for people with Prader-Willi Syndrome (PWS). Researchers want to see if CSTI-500 is safe and how well your body handles it. They will also look at how effective it is. You might be able to join if you are between 13 and 50 years old, have a confirmed diagnosis of PWS, and meet certain behavioral scores. The study will measure side effects and how much CSTI-500 is in your blood to help guide your individual dose. Success will be measured by how many side effects occur and if target drug levels are reached.
- Study design
- This is an open-label study, meaning you and the researchers will know you are receiving CSTI-500. It will involve about 12 participants and uses increasing doses of the medication.
- What's involved
- You will receive CSTI-500 orally, with individualized dosing based on blood levels. Safety will be checked for 14 weeks, and laboratory values for 12 weeks.
- Compensation
- Not stated in the trial record.
- Follow-up
- Safety will be monitored for 14 weeks, and laboratory values for 12 weeks.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
A Study of CSTI-500 in Patients With Prader-Willi Syndrome
At a glance
Conditions
NCT07348601
Where you'd take part
This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Vanderbilt University Medical Center
Nashville, Tennesseestudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Incidence of treatment-emergent adverse events (TEAEs)14 weeks
Number of participants with TEAEs, defined as an adverse event (AE) that is new or worsened in severity after the dose of study drug (coded using MedDRA).
- Proportion achieving target CSTI-500 steady-state Cmax with PK-guided dose individualization12 weeks
Proportion of participants whose observed CSTI-500 steady-state Cmax is within the protocol-defined target range for the assigned target concentration level, using plasma concentrations measured from scheduled PK sampling to guide dose adjustments.
- Incidence of clinically significant findings in laboratory values12 weeks
Laboratory evaluations include hematology, blood chemistry, and urinalysis parameters.
- Incidence of clinically significant findings in 12-lead electrocardiograms (ECGs)12 weeks
QT interval, corrected QT interval (QTc), PR interval, QRS duration, and heart rate will be measured by 12-lead electrocardiogram.
- Incidence of clinically significant findings in vital signs12 weeks
Participants will be assessed for any clinically significant changes in vital parameters (systolic blood pressure, diastolic blood pressure, heart rate, respiratory rate, and body temperature).