Clinical trial NCT03440814: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Prader-Willi Syndrome
Keywords: PWS
Prader-Willi Syndrome
Lead sponsor: Soleno Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2018-02-22
Enrollment: 127
Sex: ALL
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13)
timeFrame: Baseline to Visit 7 (Week 13)
Secondary outcomes:
measure: Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13)
timeFrame: at Visit 7 (Week 13)
measure: Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13)
timeFrame: at Visit 7 (Week 13)
measure: Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13)
timeFrame: Baseline to Visit 7 (Week 13)
Other outcomes:
Locations:
city: Orange
country: United States
facility: University of California, Irvine
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital San Diego
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Gainesville
country: United States
facility: University of Florida Gainesville
state: Florida
city: Atlanta
country: United States
facility: Emory Children's Center
state: Georgia
city: Indianapolis
country: United States
facility: Indiana University School of Medicine
state: Indiana
city: Kansas City
country: United States
facility: Kansas University Medical Center
state: Kansas
city: Bethesda
country: United States
facility: National Institutes of Health Hatfield Clinical Research Center
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Lansing
country: United States
facility: Sparrow Clinical Research Institute
state: Michigan
city: Saint Paul
country: United States
facility: Children's Minnesota
state: Minnesota
city: Paterson
country: United States
facility: St. Joseph's University Medical Center
state: New Jersey
city: Mineola
country: United States
facility: NYU Winthrop Hospital
state: New York
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center
state: Ohio
city: Columbus
country: United States
facility: The Research Institute at Nationwide Children's Hospital
state: Ohio
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
city: Dallas
country: United States
facility: Research Institute of Dallas
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Glasgow
country: United Kingdom
facility: The Queen Elizabeth University
state: Scottland
city: Hull
country: United Kingdom
facility: Hull and East Yorkshire Hospitals NHS Trust
state: Yorkshire
city: Birmingham
country: United Kingdom
facility: Birmingham Women's and Children's Hospital
city: Cambridge
country: United Kingdom
facility: Fulbourn Hospital
city: Liverpool
country: United Kingdom
facility: Alder Hey Children's Hospital NHS Foundation Trust
city: Liverpool
country: United Kingdom
facility: Aintree University Hospital NHS Foundation Trust
city: London
country: United Kingdom
facility: Royal London Hospital
city: London
country: United Kingdom
facility: Chelsea and Westminster Hospital
city: London
country: United Kingdom
facility: Hammersmith Hospital
Study officials:
affiliation: Queen Mary University of London, Barts Health NHS Trust
name: Evelien Gevers, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Florida
name: Jennifer L. Miller, MD
role: PRINCIPAL_INVESTIGATOR
References: