A Proof-of-Concept Open-Label Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of CSTI-500 in Participants With Prader-Willi Syndrome
ConSynance Therapeutics
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.
Eligibility
- Age range
- 13–50 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Generally healthy male and female individuals between the ages of 13 and 50, inclusive 2. Documented medical record history of PWS confirmed by genetic testing and PWS Nutritional Phase 3 3. CGI-S score ≥4 at Screening and Baseline (behavioral) 4. Screening HQ CT total scores ≥ 13 5. Participants must not be taking SSRI, SNRI, DNRI (bupropion), tricyclic antidepressants, stimulants, antipsychotic medications, and MAO inhibitors within 30 days of screening and willing to not start taking these medications while on the study. Fluoxetine is exclusionary unless treatment ha…
Interventions
- DrugCSTI-500
CSTI-500 given orally in an open-label, dose-escalation design with individualized dosing.
Location
- Vanderbilt University Medical CenterNashville, Tennessee