A new phase 2 study is evaluating CSTI-500, an investigational medication being developed to address some of the most challenging symptoms of PWS, including hyperphagia and behavioral dysregulation. Researchers will study the safety, tolerability, and potential effectiveness of the medication in adolescents and adults with PWS. Participants will receive increasing doses of CSTI-500, and blood levels will be measured to guide individualized dosing.
About CSTI-500
CSTI-500 is an oral investigational medication designed to affect serotonin, dopamine, and norepinephrine signaling in the brain — neurotransmitters that play important roles in mood regulation, impulse control, emotional regulation, and hunger. Researchers hope the treatment may help improve hyperphagia-related behaviors, emotional outbursts, and behavioral symptoms commonly experienced by individuals with PWS.
An earlier Phase 1 study involving people with PWS showed encouraging safety and pharmacokinetic results, supporting advancement to this next phase of testing.
Who May Be Eligible?
The trial is seeking individuals who:
- Are between 13 and 50 years old
- Have a genetically confirmed diagnosis of PWS
- Have a caregiver or parent able to participate in study visits and assessments
- Meet additional medical and medication eligibility requirements
The study team will review the full eligibility criteria with interested families during the screening process.
What Is Involved in Participating?
This is an open-label clinical trial, meaning all participants enrolled in the study will receive CSTI-500.
Participation includes:
- A screening period to determine eligibility
- Approximately 12 weeks of treatment
- Regular clinic visits and study assessments
- Monitoring for safety, side effects, and symptom changes
- Blood draws and other evaluations to help researchers understand how the medication is processed by the body
- Participation from a caregiver to help complete study-related assessments and questionnaires
Researchers will study different dosing exposure levels as part of the trial.
Study Locations
This trial is taking place at Vanderbilt University Medical Center
Contact
For questions about participation or study eligibility, please contact:
Elizabeth Roof
elizabeth.roof@vanderbilt.edu
615-343-3330
To learn more about the CSTI-500 study, visit ClinicalTrials.gov Study Listing for NCT07348601
