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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03440814
Recruitment Status : Completed
First Posted : February 22, 2018
Last Update Posted : June 26, 2020
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Drug: DCCR Drug: Placebo for DCCR Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 127 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome
Actual Study Start Date : May 9, 2018
Actual Primary Completion Date : May 1, 2020
Actual Study Completion Date : May 1, 2020

Resource links provided by the National Library of Medicine


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




Primary Outcome Measures :
  1. Hyperphagia Score [ Time Frame: Baseline to Week 13 ]
    Change in hyperphagia-related behavior as measured by total score of a Hyperphagia Questionnaire


Secondary Outcome Measures :
  1. Body fat mass (DXA) [ Time Frame: Baseline to Week 13 ]
    Change in Body Fat Mass from Baseline to Week 13

  2. CGI-I [ Time Frame: at Week 13 ]
    Clinical Global Impression of Improvement

  3. Caregiver GI-C [ Time Frame: at Week 13 ]
    Caregiver Global Impression of Change



Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03440814


Locations
Hide Hide 29 study locations
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United States, California
University of California, Irvine
Orange, California, United States, 92868
Stanford University
Palo Alto, California, United States, 94305
Rady Children's Hospital San Diego
San Diego, California, United States, 92123
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, Florida
University of Florida Gainesville
Gainesville, Florida, United States, 32608
United States, Georgia
Emory Children's Center
Atlanta, Georgia, United States, 30322
United States, Indiana
Indiana University School of Medicine
Indianapolis, Indiana, United States, 46202
United States, Kansas
Kansas University Medical Center
Kansas City, Kansas, United States, 66160
United States, Maryland
National Institutes of Health Hatfield Clinical Research Center
Bethesda, Maryland, United States, 20892
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Michigan
Sparrow Clinical Research Institute
Lansing, Michigan, United States, 48912
United States, Minnesota
Children's Minnesota
Saint Paul, Minnesota, United States, 55102
United States, New Jersey
St. Joseph's University Medical Center
Paterson, New Jersey, United States, 07503
United States, New York
NYU Winthrop Hospital
Mineola, New York, United States, 11501
United States, Ohio
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States, 44106
The Research Institute at Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Tennessee
Vanderbilt University
Nashville, Tennessee, United States, 37232
United States, Texas
Research Institute of Dallas
Dallas, Texas, United States, 75231
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84113
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
United Kingdom
The Queen Elizabeth University
Glasgow, Scottland, United Kingdom, G51 4TF
Hull and East Yorkshire Hospitals NHS Trust
Hull, Yorkshire, United Kingdom, HU3 2JZ
Birmingham Women's and Children's Hospital
Birmingham, United Kingdom, B4 6NH
Fulbourn Hospital
Cambridge, United Kingdom, CB21 5ER
Alder Hey Children's Hospital NHS Foundation Trust
Liverpool, United Kingdom, L12 2AP
Aintree University Hospital NHS Foundation Trust
Liverpool, United Kingdom, L9 7AL
Royal London Hospital
London, United Kingdom, E1 1BB
Chelsea and Westminster Hospital
London, United Kingdom, SW10 9NH
Hammersmith Hospital
London, United Kingdom, W12 OHS
Sponsors and Collaborators
Soleno Therapeutics, Inc.
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Responsible Party: Soleno Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03440814    
Other Study ID Numbers: C601
First Posted: February 22, 2018    Key Record Dates
Last Update Posted: June 26, 2020
Last Verified: December 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Soleno Therapeutics, Inc.:
PWS
Prader-Willi Syndrome
Additional relevant MeSH terms:
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Prader-Willi Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders