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

Prader-Willi Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03440814
• Other study ID #: C601
• Status: Completed
• Phase: Phase 3
• Start date: May 9, 2018
• Est. completion date: May 1, 2020

Study information

• Verified date: June 2023
• Source: Soleno Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 127
• Est. completion date: May 1, 2020
• Est. primary completion date: May 1, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• DCCR
Once daily oral administration
• Placebo for DCCR
Once daily oral administration

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Soleno Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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)
Secondary	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)
Secondary	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)