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

Primary Purpose

Prader-Willi Syndrome

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
DCCR
Placebo for DCCR
Sponsored by
Soleno Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Prader-Willi Syndrome focused on measuring PWS, Prader-Willi Syndrome

Eligibility Criteria

4 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of California, Irvine
  • Stanford University
  • Rady Children's Hospital San Diego
  • Children's Hospital Colorado
  • University of Florida Gainesville
  • Emory Children's Center
  • Indiana University School of Medicine
  • Kansas University Medical Center
  • National Institutes of Health Hatfield Clinical Research Center
  • Boston Children's Hospital
  • Sparrow Clinical Research Institute
  • Children's Minnesota
  • St. Joseph's University Medical Center
  • NYU Winthrop Hospital
  • University Hospitals Cleveland Medical Center
  • The Research Institute at Nationwide Children's Hospital
  • Vanderbilt University
  • Research Institute of Dallas
  • University of Utah
  • Seattle Children's Hospital
  • The Queen Elizabeth University
  • Hull and East Yorkshire Hospitals NHS Trust
  • Birmingham Women's and Children's Hospital
  • Fulbourn Hospital
  • Alder Hey Children's Hospital NHS Foundation Trust
  • Aintree University Hospital NHS Foundation Trust
  • Royal London Hospital
  • Chelsea and Westminster Hospital
  • Hammersmith Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

DCCR

Placebo

Arm Description

75 - 450 mg DCCR

75 - 450 mg placebo for DCCR

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.
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.
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).

Full Information

First Posted
February 13, 2018
Last Updated
August 25, 2023
Sponsor
Soleno Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03440814
Brief Title
A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome
Official Title
A Randomized, Double-Blind, Placebo-Controlled Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Completed
Study Start Date
May 9, 2018 (Actual)
Primary Completion Date
May 1, 2020 (Actual)
Study Completion Date
May 1, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Soleno Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome
Keywords
PWS, Prader-Willi Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
127 (Actual)

8. Arms, Groups, and Interventions

Arm Title
DCCR
Arm Type
Experimental
Arm Description
75 - 450 mg DCCR
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
75 - 450 mg placebo for DCCR
Intervention Type
Drug
Intervention Name(s)
DCCR
Intervention Description
Once daily oral administration
Intervention Type
Drug
Intervention Name(s)
Placebo for DCCR
Intervention Description
Once daily oral administration
Primary Outcome Measure Information:
Title
Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13)
Description
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)
Secondary Outcome Measure Information:
Title
Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13)
Description
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)
Title
Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13)
Description
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)
Title
Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13)
Description
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)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
4 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Evelien Gevers, MD, PhD
Organizational Affiliation
Queen Mary University of London, Barts Health NHS Trust
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Jennifer L. Miller, MD
Organizational Affiliation
University of Florida
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of California, Irvine
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Rady Children's Hospital San Diego
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Florida Gainesville
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Facility Name
Emory Children's Center
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Indiana University School of Medicine
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Kansas University Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
National Institutes of Health Hatfield Clinical Research Center
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Sparrow Clinical Research Institute
City
Lansing
State/Province
Michigan
ZIP/Postal Code
48912
Country
United States
Facility Name
Children's Minnesota
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55102
Country
United States
Facility Name
St. Joseph's University Medical Center
City
Paterson
State/Province
New Jersey
ZIP/Postal Code
07503
Country
United States
Facility Name
NYU Winthrop Hospital
City
Mineola
State/Province
New York
ZIP/Postal Code
11501
Country
United States
Facility Name
University Hospitals Cleveland Medical Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
The Research Institute at Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
Vanderbilt University
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Research Institute of Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84113
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
The Queen Elizabeth University
City
Glasgow
State/Province
Scottland
ZIP/Postal Code
G51 4TF
Country
United Kingdom
Facility Name
Hull and East Yorkshire Hospitals NHS Trust
City
Hull
State/Province
Yorkshire
ZIP/Postal Code
HU3 2JZ
Country
United Kingdom
Facility Name
Birmingham Women's and Children's Hospital
City
Birmingham
ZIP/Postal Code
B4 6NH
Country
United Kingdom
Facility Name
Fulbourn Hospital
City
Cambridge
ZIP/Postal Code
CB21 5ER
Country
United Kingdom
Facility Name
Alder Hey Children's Hospital NHS Foundation Trust
City
Liverpool
ZIP/Postal Code
L12 2AP
Country
United Kingdom
Facility Name
Aintree University Hospital NHS Foundation Trust
City
Liverpool
ZIP/Postal Code
L9 7AL
Country
United Kingdom
Facility Name
Royal London Hospital
City
London
ZIP/Postal Code
E1 1BB
Country
United Kingdom
Facility Name
Chelsea and Westminster Hospital
City
London
ZIP/Postal Code
SW10 9NH
Country
United Kingdom
Facility Name
Hammersmith Hospital
City
London
ZIP/Postal Code
W12 OHS
Country
United Kingdom

12. IPD Sharing Statement

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

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