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

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ClinicalTrials.gov Identifier: NCT03440814
Recruitment Status : Recruiting
First Posted : February 21, 2018
Last Update Posted : September 26, 2018
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

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 : December 2018
Estimated Study Completion Date : December 2018

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



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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


Contacts
Contact: C601 Project Manager 650-353-2051 C601ProjectManager@soleno.life

Locations
United States, California
Stanford University Recruiting
Palo Alto, California, United States, 94305
Contact: Alicia Harnett    650-723-0441    aharnett@stanford.edu   
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    Rwinograd@rchsd.org   
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    Marcelle.Terry@childrenscolorado.org   
Principal Investigator: Christine L Chan, MD         
United States, Florida
University of Florida Gainesville Recruiting
Gainesville, Florida, United States, 32608
Contact: Beverly P Giordano, MS, PNP    352-294-5280    bgiordano@ufl.edu   
Principal Investigator: Jennifer L Miller, MD         
United States, Minnesota
Children's Minnesota Recruiting
Saint Paul, Minnesota, United States, 55102
Contact: Brittany Machus, MA, CCRP    651-220-5730    brittany.machus@childrensmn.org   
Principal Investigator: Jennifer Abuzzahab, MD         
United States, Ohio
University Hospitals Cleveland Medical Center Recruiting
Cleveland, Ohio, United States, 44106
Contact: Audrey Lynn, Ph.D    216-844-7124    audrey.lynn@uhhospitals.org   
Principal Investigator: Lori-Anne Schillaci, MD, FAAP         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Sue Kearns, BSN    206-987-2640    sue.kearns@seattlechildrens.org   
Principal Investigator: Parisa Salehi, MD         
Sponsors and Collaborators
Soleno Therapeutics, Inc.

Responsible Party: Soleno Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03440814     History of Changes
Other Study ID Numbers: C601
First Posted: February 21, 2018    Key Record Dates
Last Update Posted: September 26, 2018
Last Verified: September 2018

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:
Syndrome
Prader-Willi 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
Choline
Diazoxide
Lipotropic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Gastrointestinal Agents
Lipid Regulating Agents
Nootropic Agents
Antihypertensive Agents
Vasodilator Agents