A Study of CSTI-500 in Patients With Prader-Willi Syndrome (NCT07348601) | Clinical Trial Compass
RecruitingPhase 2
A Study of CSTI-500 in Patients With Prader-Willi Syndrome
United States12 participantsStarted 2026-05
Plain-language summary
This is a proof-of-concept, open-label, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of CSTI-500 in participants with genetically confirmed Prader-Willi Syndrome (PWS) who are 13 to 50 years of age. Participants will receive increasing doses of CSTI-500, and blood levels will be measured to guide individualized dosing.
Who can participate
Age range
13 Years – 50 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Generally healthy male and female individuals between the ages of 13 and 50, inclusive
. Documented medical record history of PWS confirmed by genetic testing and PWS Nutritional Phase 3
. CGI-S score ≥4 at Screening and Baseline (behavioral)
. Screening HQ CT total scores ≥ 13
Exclusion criteria
. Caregiver/parent must agree to bring the subject to the site for the visits, remain with the subject during visit times when allowed and respond to any questions.
. Caregiver/parent is willing to provide informed consent and agrees to adhere to required study procedures including telemedicine visits, visit duration requirements, and offer consistent care.
Questions worth asking your doctor
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
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.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of treatment-emergent adverse events (TEAEs)
Timeframe: 14 weeks
2
Proportion achieving target CSTI-500 steady-state Cmax with PK-guided dose individualization
Timeframe: 12 weeks
3
Incidence of clinically significant findings in laboratory values
Timeframe: 12 weeks
4
Incidence of clinically significant findings in 12-lead electrocardiograms (ECGs)
Timeframe: 12 weeks
5
Incidence of clinically significant findings in vital signs
. Caregivers must agree to complete all study required assessments.
. Participants who cannot consent for themselves and are able will provide assent.
0. Female participants must not be pregnant or lactating. Nonpregnancy will be confirmed for all females by a urine pregnancy test conducted at Screening and at the Baseline Visit prior to enrollment into the study. If of childbearing potential, the subject/caregiver agrees to the use of one of the accepted contraceptive regimens from Screening to the first administration of the study medication, during the study, and for at least 30 days after the last dose of the study medication. An acceptable method of contraception includes one of the following:
. Hormonal contraceptives (birth control pills, injectable/implant/insertable hormonal birth control products, transdermal patch)
. Intrauterine device (with or without hormones)
. OR agrees to use a double barrier method (e.g., condom and spermicide) during the study and for at least 30 days after the last dose of the study medication.