A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome

Study Overview
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Brief Summary
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The purpose of this is study is to evaluate the effects of DCCR (diazoxide choline controlled release tablets) in children and adults with Prader-Willi syndrome.

Official Title
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A Randomized, Double-Blind, Placebo-Controlled Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome

Conditions
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Prader-Willi Syndrome

Intervention / Treatment
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* Drug: DCCR
* Drug: Placebo for DCCR


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (patients, as appropriate) Genetically-confirmed Prader-Willi syndrome and hyperphagic In a stable care setting for at least 6 months prior to Visit 1 Caregiver must have been caring for the patient for at least 6 months prior to Visit 1 Exclusion Criteria: Have participated in an interventional clinical study (i.e., investigational drug or device, approved drugs or device evaluated for unapproved use) within prior 3 months Positive urine pregnancy test (in females of child-bearing potential) or females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 30 days after study participation Any other known disease and/or condition, which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol

Ages Eligible for Study
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Minimum Age: 4 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: DCCR<br>75 - 450 mg DCCR | Drug: DCCR<br>* Once daily oral administration<br>|
| Placebo Comparator: Placebo<br>75 - 450 mg placebo for DCCR | Drug: Placebo for DCCR<br>* Once daily oral administration<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) | Hyperphagia-related behaviors were assessed by the validated hyperphagia questionnaire for clinical trials (HQ-CT), an instrument designed to measure symptoms of food related preoccupations and behaviors that was completed by the caregiver. The HQ-CT consists of nine items with responses ranging from 0-4 units each (possible total score range: 0-36). The HQ-CT was assessed at Screening, Baseline (Visit 2), and approximately every 4 weeks post-dose at Week 4, Week 8, and Week 13. A decrease in score from baseline represented improvement. | Baseline to Visit 7 (Week 13) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) | The Clinical Global Impression of Improvement (CGI-I) is a single statement designed to assess the Investigator's overall perception of change in the subject's condition across the course of the clinical trial. The Investigator provided a response to Compared to the subject's condition at enrollment, the subject's condition is: by rating the subject's behavior using a 7-point response scale: Very much improved, Much improved, Minimally improved, No change, Minimally worse, Much worse, and Very much worse. The Investigator only took into account the subject's PWS condition. | at Visit 7 (Week 13) | 
| Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13) | The Caregiver Global Impression of Change (GI-C) is a single statement designed to assess the caregiver's overall perception of change in the subject across the course of the clinical trial. The caregiver provided a response to Please choose the response below that best describes the overall change in the person's PWS since they started taking the study medication using a 7-point graded response scale: Very much better, Moderately better, A little better, No change, A little worse, Moderately worse, and Very much worse. | at Visit 7 (Week 13) | 
| Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13) | Whole body scans were performed. Reports included a breakdown of the following regions: left arm, right arm, trunk, left leg, right leg, and head. Each region was evaluated for body fat mass (g). | Baseline to Visit 7 (Week 13) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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PWS, Prader-Willi Syndrome