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

Phase 3

Completed

Conditions: Prader-Willi Syndrome

Interventions: Drug: Placebo for DCCR
Drug: DCCR

Registration Number: NCT03440814

Lead Sponsor: Soleno Therapeutics, Inc.

Brief Summary: 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.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 127

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo for DCCR	75 - 450 mg placebo for DCCR
DCCR	DCCR	75 - 450 mg DCCR

Primary Outcome Measures

Name	Time	Method
Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13)	Baseline to 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.

Secondary Outcome Measures

Name	Time	Method
Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13)	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.
Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13)	Baseline to 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).
Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13)	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.

Trial Locations

Locations (29): University of California, Irvine
🇺🇸
Orange, California, United States
Stanford University
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Palo Alto, California, United States
Rady Children's Hospital San Diego
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San Diego, California, United States
Children's Hospital Colorado
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Aurora, Colorado, United States
University of Florida Gainesville
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Gainesville, Florida, United States
Emory Children's Center
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Atlanta, Georgia, United States
Indiana University School of Medicine
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Indianapolis, Indiana, United States
Kansas University Medical Center
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Kansas City, Kansas, United States
National Institutes of Health Hatfield Clinical Research Center
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Bethesda, Maryland, United States
Boston Children's Hospital
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Boston, Massachusetts, United States
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University of California, Irvine
🇺🇸Orange, California, United States