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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03440814
Recruitment Status : Recruiting
First Posted : February 21, 2018
Last Update Posted : June 11, 2019
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)
Estimated Enrollment : 105 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
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : June 2019

Resource links provided by the National Library of Medicine

Drug Information available for: Diazoxide

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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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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

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 identifier (NCT number): NCT03440814

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Contact: C601 Project Manager 650-353-2051

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United States, California
University of California, Irvine Recruiting
Orange, California, United States, 92868
Contact: Margaret Knight    949-824-0521   
Principal Investigator: Virginia Kimonis, MD         
Stanford University Recruiting
Palo Alto, California, United States, 94305
Contact: Alicia Harnett    650-723-0441   
Principal Investigator: David Stevenson, MD         
Rady Children's Hospital San Diego Recruiting
San Diego, California, United States, 92123
Contact: Rachel Winograd, R.N.    858-966-8453   
Principal Investigator: Lynne M. Bird, MD         
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Marcie Terry    720-777-8439   
Principal Investigator: Shawn McCandless, MD         
United States, Florida
University of Florida Gainesville Recruiting
Gainesville, Florida, United States, 32608
Contact: Beverly P Giordano, MS, PNP    352-294-5280   
Principal Investigator: Jennifer L Miller, MD         
United States, Kansas
Kansas University Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Yolanda Harness    913-574-0302   
Principal Investigator: Merlin Butler, MD         
United States, Maryland
National Institutes of Health Hatfield Clinical Research Center Recruiting
Bethesda, Maryland, United States, 20892
Contact: Elisabeth Davis, BA    301-496-6726   
Contact: Shelia Brady, MSN    301-451-3783   
Principal Investigator: Jack A Yanovski, MD, Ph.D         
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Andrea Hale, RN, BSN, MPH, CCRP    617-918-2867   
Principal Investigator: Amy Fleischman, MD         
United States, Michigan
Sparrow Clinical Research Institute Recruiting
Lansing, Michigan, United States, 48912
Contact: Jennifer Boak    517-364-5733   
Principal Investigator: Elizabeth Littlejohn, MD         
United States, Minnesota
Children's Minnesota Recruiting
Saint Paul, Minnesota, United States, 55102
Contact: Brittany Machus, MA, CCRP    651-220-5730   
Principal Investigator: Jennifer Abuzzahab, MD         
United States, New York
NYU Winthrop Hospital Recruiting
Mineola, New York, United States, 11501
Contact: Marilyn Richardson    516-663-9582   
Principal Investigator: Moris Angulo, MD         
United States, Ohio
University Hospitals Cleveland Medical Center Recruiting
Cleveland, Ohio, United States, 44106
Contact: Audrey Lynn, Ph.D    216-844-7124   
Principal Investigator: Lori-Anne Schillaci, MD, FAAP         
The Research Institute at Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Emily Viall    614-722-2630   
Principal Investigator: Kathryn Obrynba, MD         
United States, Tennessee
Vanderbilt University Recruiting
Nashville, Tennessee, United States, 37232
Contact: Sarah N McKeel, R.N., BSN    615-875-3772   
Principal Investigator: Ashley Hall Shoemaker, MD         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Sue Kearns, BSN    206-987-2640   
Principal Investigator: Parisa Salehi, MD         
United Kingdom
Chelsea and Westminster Hospital Recruiting
London, United Kingdom, SW10 9NH
Contact: Rhian Bull   
Principal Investigator: Nicola Bridges         
Sponsors and Collaborators
Soleno Therapeutics, Inc.

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Responsible Party: Soleno Therapeutics, Inc. Identifier: NCT03440814     History of Changes
Other Study ID Numbers: C601
First Posted: February 21, 2018    Key Record Dates
Last Update Posted: June 11, 2019
Last Verified: June 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.:
Prader-Willi Syndrome

Additional relevant MeSH terms:
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Prader-Willi Syndrome
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Nutrition Disorders
Lipotropic Agents
Hypolipidemic Agents
Molecular Mechanisms of Pharmacological Action
Gastrointestinal Agents
Lipid Regulating Agents
Nootropic Agents
Antihypertensive Agents
Vasodilator Agents