RecruitingPrader-Willi Syndrome

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

Eligible age

13–50 yrs

Accepts

All genders

Locations

1 state

Healthy volunteers

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About this study

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.

Sponsor: ConSynance Therapeutics

You may qualify if…

✓ 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 has been discontinued \>6 months prior to Screening.
✓ 6. 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.
✓ 7. 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.
✓ 8. Caregivers must agree to complete all study required assessments.

You may not qualify if…

✕ 1. Participation in any clinical study with an investigational drug/device within 3 months prior to screening or during the study
✕ 2. PWS diagnosis of UPD (maternal uniparental disomy).
✕ 3. Current use of DCCR or if used previously, must be off at least 4 weeks before screening.
✕ 4. Recent use (within 3 months) of weight loss agents including prescription, herbal medications, and weight loss supplements. Ozempic, for diabetes, would be exclusionary due to its effect on weight loss.
✕ 5. History of bariatric surgery or major surgery within 6 months of screening or planned during the study.
✕ 6. Any malignancy in the 2 years prior to screening (excluding basal cell carcinoma or squamous cell carcinoma of the skin or cervical carcinoma in situ that have been successfully treated).
✕ 7. Current liver, pulmonary, cardiac, or GI disease that would be expected to adversely affect study participation. Stable disease, e.g., asthma or controlled hypertension is not excluded. Liver disease or liver injury as indicated by abnormal liver function tests, ALT, AST, alkaline phosphatase, or serum bilirubin (≥3X ULN for any of these tests).
✕ a. Participants with impaired liver function (Child-Pugh Scores A, B, C)

Where it's recruiting

Tennessee

Nashville

Source: ClinicalTrials.gov · NCT07348601 · last updated 2026-10-21