Health TrialsbyHealthPanel

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

This trial does not accept healthy volunteers

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

Design model

Sequential

Treatments tested in this trial

  • CSTI-500

    Drug

    CSTI-500 given orally in an open-label, dose-escalation design with individualized dosing.

Treatment groups

12 Participants
are divided into 3 treatment groups
Group A: CSTI-500 (Open-label) Low Target ExposureExperimental treatment 1 intervention
Group B: CSTI-500 (Open-Label) Medium Target ExposureExperimental treatment 1 intervention
Group C: CSTI-500 (Open-label) High Target ExposureExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence 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
2

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
3

Incidence of clinically significant findings in laboratory values

Laboratory evaluations include hematology, blood chemistry, and urinalysis parameters.

Time frame
12 weeks
4

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

Secondary outcomes

1

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.

Time frame
12 weeks
2

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
3

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
4

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

ConSynance Therapeutics

Lead sponsor

Source ClinicalTrials.gov
Apply now