Open-Label Phase 2 Study of CSTI-500 to Assess Safety and Effectiveness in People Aged 13 to 50 With Genetically Confirmed Prader-Willi Syndrome

What this Trial Is About

Researchers are studying CSTI-500 in people aged 13 to 50 years who have genetically confirmed Prader-Willi Syndrome PWS. This phase 2, open-label, dose-escalation study aims to evaluate the safety, tolerability, how the drug moves through the body, and its effects. The study focuses on monitoring treatment-emergent adverse events and other clinical parameters to better understand CSTI-500s impact in this population. Participants will receive CSTI-500 taken by mouth in increasing doses guided by blood level measurements to individualize treatment. There are three dose groups targeting low, medium, and high exposure levels of CSTI-500. The study does not use a placebo and all participants know the treatment they receive. The dosing and monitoring occur over a planned 12 to 14 week period. During the trial, participants will have regular safety evaluations including laboratory tests, ECGs, vital signs, and assessments of behavior and hyperphagia symptoms using standardized questionnaires. Caregivers are involved in providing information and supporting adherence. The primary outcomes include adverse event incidence and target drug levels achieved, while secondary outcomes measure changes in clinical global impression and behavior scales over 12 weeks. Overall participation lasts several months with close monitoring throughout.

A Study of CSTI-500 in Patients With Prader-Willi Syndrome