A Study of CSTI-500 in Patients With Prader-Willi Syndrome
ConditionPrader-Willi Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age13-50
SponsorConSynance Therapeutics
About this trial
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 criteria
Qualifiers
Generally healthy male and female individuals between the ages of 13 and 50, inclusive
Documented medical record history of PWS confirmed by genetic testing and PWS Nutritional Phase 3
CGI-S score ≥4 at Screening and Baseline (behavioral)
Screening HQ CT total scores ≥ 13
Disqualifiers
Participation in any clinical study with an investigational drug/device within 3 months prior to screening or during the study
PWS diagnosis of UPD (maternal uniparental disomy).
Current use of DCCR or if used previously, must be off at least 4 weeks before screening.
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.
Trial design
Treatments tested in this trial
- CSTI-500
Treatment groups
12 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov