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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
CSTI-500 given orally in an open-label, dose-escalation design with individualized dosing.
Vanderbilt University Medical Center
Nashville, Tennessee, United States
RECRUITINGIncidence of treatment-emergent adverse events (TEAEs)
Number of participants with TEAEs, defined as an adverse event (AE) that is new or worsened in severity after the dose of study drug (coded using MedDRA).
Time frame: 14 weeks
Proportion achieving target CSTI-500 steady-state Cmax with PK-guided dose individualization
Proportion of participants whose observed CSTI-500 steady-state Cmax is within the protocol-defined target range for the assigned target concentration level, using plasma concentrations measured from scheduled PK sampling to guide dose adjustments.
Time frame: 12 weeks
Incidence of clinically significant findings in laboratory values
Laboratory evaluations include hematology, blood chemistry, and urinalysis parameters.
Time frame: 12 weeks
Incidence of clinically significant findings in 12-lead electrocardiograms (ECGs)
QT interval, corrected QT interval (QTc), PR interval, QRS duration, and heart rate will be measured by 12-lead electrocardiogram.
Time frame: 12 weeks
Incidence of clinically significant findings in vital signs
Participants will be assessed for any clinically significant changes in vital parameters (systolic blood pressure, diastolic blood pressure, heart rate, respiratory rate, and body temperature).
Time frame: 12 weeks
Change from baseline in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score
Change from baseline in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score. The HQ-CT total score ranges from 0 to 36, with higher scores indicating greater hyperphagia.
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Time frame: 12 weeks
Change from baseline in Aberrant Behavior Checklist - Community (ABC-C) total score and subscale scores
Change from baseline in Aberrant Behavior Checklist - Community (ABC-C) total score and subscale scores. The ABC-C is a 58-item caregiver-reported questionnaire, with higher scores indicating more severe aberrant behavior.
Time frame: 12 weeks
Clinical Global Impression-Improvement (CGI-I) score
Clinical Global Impression-Improvement (CGI-I) score assessed at Week 12. The CGI-I is a 7-point ordinal scale, with lower scores indicating greater improvement.
Time frame: 12 weeks
Change from baseline in Clinical Global Impression-Severity (CGI-S) score
Change from baseline in Clinical Global Impression-Severity (CGI-S) score. The CGI-S is a 7-point ordinal scale, with lower scores indicating less severe illness (better outcome).
Time frame: 12 weeks
Change from baseline in Prader-Willi Syndrome Questionnaire (PADQ) score
Change from baseline in the Prader-Willi Syndrome Questionnaire (PADQ) score. The PADQ is a caregiver-reported questionnaire, with higher scores indicating greater symptom severity (worse outcome).
Time frame: 12 weeks
Change from baseline in Caregiver Global Impression-Severity (careGI-S) score
Change from baseline in Caregiver Global Impression-Severity (careGI-S) score. The careGI-S is a caregiver-reported ordinal scale, with lower scores indicating less severe symptoms (better outcome).
Time frame: 12 weeks
Caregiver Global Impression-Change (careGI-C) score
Caregiver Global Impression-Change (careGI-C) score assessed at Week 12. The careGI-C is a caregiver-reported ordinal scale, with lower scores indicating greater improvement.
Time frame: 12 weeks