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

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

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.

Study Overview

• Status: Completed
• Conditions: Prader-Willi Syndrome
• Intervention / Treatment: Drug: DCCR; Drug: Placebo for DCCR

• Study Type: Interventional
• Enrollment (Actual): 127
• Phase: Phase 3

Contacts and Locations

Study Locations

• United Kingdom
  • Birmingham, United Kingdom, B4 6NH
    • Birmingham Women's and Children's Hospital
  • Cambridge, United Kingdom, CB21 5ER
    • Fulbourn Hospital
  • Liverpool, United Kingdom, L9 7AL
    • Aintree University Hospital NHS Foundation Trust
  • Liverpool, United Kingdom, L12 2AP
    • Alder Hey Children's Hospital NHS Foundation Trust
  • London, United Kingdom, SW10 9NH
    • Chelsea and Westminster Hospital
  • London, United Kingdom, E1 1BB
    • Royal London Hospital
  • London, United Kingdom, W12 OHS
    • Hammersmith Hospital
  • Scottland
    • Glasgow, Scottland, United Kingdom, G51 4TF
      • The Queen Elizabeth University
  • Yorkshire
    • Hull, Yorkshire, United Kingdom, HU3 2JZ
      • Hull and East Yorkshire Hospitals NHS Trust
• United States
  • California
    • Orange, California, United States, 92868
      • University of California, Irvine
    • Palo Alto, California, United States, 94305
      • Stanford University
    • San Diego, California, United States, 92123
      • Rady Children's Hospital San Diego
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Children's Hospital Colorado
  • Florida
    • Gainesville, Florida, United States, 32608
      • University of Florida Gainesville
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory Children's Center
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Indiana University School of Medicine
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • Kansas University Medical Center
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • National Institutes of Health Hatfield Clinical Research Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
  • Michigan
    • Lansing, Michigan, United States, 48912
      • Sparrow Clinical Research Institute
  • Minnesota
    • Saint Paul, Minnesota, United States, 55102
      • Children's Minnesota
  • New Jersey
    • Paterson, New Jersey, United States, 07503
      • St. Joseph's University Medical Center
  • New York
    • Mineola, New York, United States, 11501
      • NYU Winthrop Hospital
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • University Hospitals Cleveland Medical Center
    • Columbus, Ohio, United States, 43205
      • The Research Institute at Nationwide Children's Hospital
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt University
  • Texas
    • Dallas, Texas, United States, 75231
      • Research Institute of Dallas
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • University of Utah
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: DCCR; 75 - 450 mg DCCR	Drug: DCCR; Once daily oral administration
Placebo Comparator: Placebo; 75 - 450 mg placebo for DCCR	Drug: Placebo for DCCR; Once daily oral administration

What is the study measuring?

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)

Collaborators and Investigators

• Sponsor: Soleno Therapeutics, Inc.
• Investigators: Principal Investigator: Evelien Gevers, MD, PhD, Queen Mary University of London, Barts Health NHS Trust; Principal Investigator: Jennifer L. Miller, MD, University of Florida

Study record dates

Study Major Dates

• Study Start (Actual): May 9, 2018
• Primary Completion (Actual): May 1, 2020
• Study Completion (Actual): May 1, 2020

Study Registration Dates

• First Submitted: February 13, 2018
• First Submitted That Met QC Criteria: February 19, 2018
• First Posted (Actual): February 22, 2018

Study Record Updates

• Last Update Posted (Actual): September 21, 2023
• Last Update Submitted That Met QC Criteria: August 25, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Keywords: Prader-Willi Syndrome; PWS
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Overnutrition; Nutrition Disorders; Overweight; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Syndrome; Prader-Willi Syndrome
• Other Study ID Numbers: C601

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No