A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome
- 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
- 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
- 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)
Emory Children's Center
🇺🇸Atlanta, Georgia, United States
Research Institute of Dallas
🇺🇸Dallas, Texas, United States
Children's Minnesota
🇺🇸Saint Paul, Minnesota, United States
Alder Hey Children's Hospital NHS Foundation Trust
🇬🇧Liverpool, United Kingdom
St. Joseph's University Medical Center
🇺🇸Paterson, New Jersey, United States
Hull and East Yorkshire Hospitals NHS Trust
🇬🇧Hull, Yorkshire, United Kingdom
Fulbourn Hospital
🇬🇧Cambridge, United Kingdom
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
The Research Institute at Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Stanford University
🇺🇸Palo Alto, California, United States
Sparrow Clinical Research Institute
🇺🇸Lansing, Michigan, United States
Aintree University Hospital NHS Foundation Trust
🇬🇧Liverpool, United Kingdom
Kansas University Medical Center
🇺🇸Kansas City, Kansas, United States
University of California, Irvine
🇺🇸Orange, California, United States
NYU Winthrop Hospital
🇺🇸Mineola, New York, United States
University Hospitals Cleveland Medical Center
🇺🇸Cleveland, Ohio, United States
The Queen Elizabeth University
🇬🇧Glasgow, Scottland, United Kingdom
Birmingham Women's and Children's Hospital
🇬🇧Birmingham, United Kingdom
Royal London Hospital
🇬🇧London, United Kingdom
Chelsea and Westminster Hospital
🇬🇧London, United Kingdom
Hammersmith Hospital
🇬🇧London, United Kingdom
University of Florida Gainesville
🇺🇸Gainesville, Florida, United States
Vanderbilt University
🇺🇸Nashville, Tennessee, United States
Rady Children's Hospital San Diego
🇺🇸San Diego, California, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Indiana University School of Medicine
🇺🇸Indianapolis, Indiana, United States
National Institutes of Health Hatfield Clinical Research Center
🇺🇸Bethesda, Maryland, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States