A Study of CSTI-500 in Patients With Prader-Willi Syndrome (NCT07348601) | Clinical Trial Compass
Not Yet RecruitingPhase 2
A Study of CSTI-500 in Patients With Prader-Willi Syndrome
United States12 participantsStarted 2026-03
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 range13 Years – 50 Years
SexALL
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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.
✕. 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)
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
✕. 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.