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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
127
Once daily oral administration
Once daily oral administration
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.
Time frame: Baseline to Visit 7 (Week 13)
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.
Time frame: 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.
Time frame: at Visit 7 (Week 13)
Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13)
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University of California, Irvine
Orange, California, United States
Stanford University
Palo Alto, California, United States
Rady Children's Hospital San Diego
San Diego, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
University of Florida Gainesville
Gainesville, Florida, United States
Emory Children's Center
Atlanta, Georgia, United States
Indiana University School of Medicine
Indianapolis, Indiana, United States
Kansas University Medical Center
Kansas City, Kansas, United States
National Institutes of Health Hatfield Clinical Research Center
Bethesda, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
...and 19 more locations
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).
Time frame: Baseline to Visit 7 (Week 13)