Actively Recruiting
A Proof-of-Concept Open-Label Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of CSTI-500 in Participants With Prader-Willi Syndrome
Led by ConSynance Therapeutics · Updated on 2026-05-15
12
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
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-500's 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.
CONDITIONS
Brief Title
A Study of CSTI-500 in Patients With Prader-Willi Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Generally healthy male and female individuals between the ages of 13 and 50, inclusive
- Documented medical record history of Prader-Willi Syndrome confirmed by genetic testing and PWS Nutritional Phase 3
- Clinical Global Impression-Severity (CGI-S) score of 4 or higher at Screening and Baseline (behavioral)
- Screening Hyperphagia Questionnaire for Clinical Trials (HQ CT) total score of 13 or higher
- Not taking SSRIs, SNRIs, DNRI (bupropion), tricyclic antidepressants, stimulants, antipsychotic medications, or MAO inhibitors within 30 days of screening and willing to avoid these during the study
- Fluoxetine use exclusion unless discontinued more than 6 months prior to screening
- Caregiver or parent agrees to bring the participant to visits, stay during visit times, and respond to questions
- Caregiver or parent willing to provide informed consent and follow study procedures including telemedicine visits
- Caregivers agree to complete all required assessments
- Participants unable to consent must provide assent if able
- Female participants must not be pregnant or lactating; confirmed by urine pregnancy tests at screening and baseline
- If of childbearing potential, agree to use accepted contraceptive methods during screening, study, and 30 days after last dose
- Females of non-childbearing potential confirmed by surgical sterility or postmenopausal status with FSH levels of 40 mIU/mL or higher
- Male participants not sterile must agree to use double barrier contraception methods
- Blood pressure and heart rate within specified ranges at screening
- All concomitant medications stable for 3 months prior to screening with no planned changes during the study
- Willingness to comply with all study procedures and availability for the study duration
You will not qualify if you...
- Participation in any clinical study with an investigational drug or device within 3 months prior to screening or during the study
- Prader-Willi Syndrome diagnosis of maternal uniparental disomy (UPD)
- Current or recent use (within 4 weeks) of DCCR
- Recent use (within 3 months) of weight loss agents including prescription, herbal medications, and supplements
- History of bariatric or major surgery within 6 months of screening or planned during the study
- Any malignancy within 2 years prior to screening except certain treated skin or cervical cancers
- Current liver, lung, heart, or gastrointestinal disease expected to affect participation, including impaired liver function
- Unexplained symptoms such as dizziness, syncope, fatigue, palpitations, headaches, or exercise intolerance
- Use of prohibited medications including certain antidepressants, stimulants, antipsychotics, mood stabilizers, and GLP-1 agonists
- Postural orthostatic tachycardia syndrome (POTS) as defined by heart rate increase and symptoms
- Clinically significant abnormal ECG findings at screening
- Significant cardiac arrhythmias or heart failure NYHA class II or greater
- Recent myocardial infarction, stroke, or transient ischemic attack within 5 years
- Reduced kidney function measured by eGFR below 90 mL/min/1.73m2
- Uncontrolled type 2 diabetes with HbA1c 9% or higher
- Insulin-dependent type 1 diabetes
- Positive tests for hepatitis B, hepatitis C, or HIV
- Uncontrolled thyroid disease
- Known allergy to components of the investigational product
- History or active severe psychiatric disorders or suicidal behavior
- Known alcohol or substance abuse
- Use of moderate to strong inhibitors or inducers of specific liver enzymes or grapefruit juice
- Any other condition that may interfere with completing the study
- Inability to swallow oral capsules whole with water
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 12 weeks
Participants receive CSTI-500 orally in an open-label, dose-escalation design with individualized dosing to evaluate safety, tolerability, and efficacy.
Weekly visits for up to 12 weeks
Trial Site Locations
Total: 1 location
1
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
Actively Recruiting
Research Team
B
Beth Mack
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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