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

Phase 3

Completed

• Conditions: Prader-Willi Syndrome
• Interventions: Drug: Placebo for DCCR; Drug: DCCR

• Registration Number: NCT03440814
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-02-13
• Study First Submitted QC: 2018-02-19
• Study First Posted: 2018-02-22
• Study Start: 2018-05-09
• Primary Completion: 2020-05-01
• Study Completion: 2020-05-01
• Status Verified: 2023-06
• Results First Submitted: 2023-06-30
• Results First Submitted QC: 2023-08-25
• Last Update Submitted: 2023-08-25
• Results First Posted: 2023-09-21
• Last Update Posted: 2023-09-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 127

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo for DCCR	75 - 450 mg placebo for DCCR
DCCR	DCCR	75 - 450 mg DCCR

Primary Outcome Measures

Name	Time	Method
Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13)	Baseline to 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

Name	Time	Method
Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13)	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)	Baseline to 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).
Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13)	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.

Trial Locations

Locations (29)

Emory Children's Center; 🇺🇸 Atlanta, Georgia, United States

Research Institute of Dallas; 🇺🇸 Dallas, Texas, United States

Children's Minnesota; 🇺🇸 Saint Paul, Minnesota, United States

Alder Hey Children's Hospital NHS Foundation Trust; 🇬🇧 Liverpool, United Kingdom

St. Joseph's University Medical Center; 🇺🇸 Paterson, New Jersey, United States

Hull and East Yorkshire Hospitals NHS Trust; 🇬🇧 Hull, Yorkshire, United Kingdom

Fulbourn Hospital; 🇬🇧 Cambridge, United Kingdom

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

The Research Institute at Nationwide Children's Hospital; 🇺🇸 Columbus, Ohio, United States

Seattle Children's Hospital; 🇺🇸 Seattle, Washington, United States

Stanford University; 🇺🇸 Palo Alto, California, United States

Sparrow Clinical Research Institute; 🇺🇸 Lansing, Michigan, United States

Aintree University Hospital NHS Foundation Trust; 🇬🇧 Liverpool, United Kingdom

Kansas University Medical Center; 🇺🇸 Kansas City, Kansas, United States

University of California, Irvine; 🇺🇸 Orange, California, United States

NYU Winthrop Hospital; 🇺🇸 Mineola, New York, United States

University Hospitals Cleveland Medical Center; 🇺🇸 Cleveland, Ohio, United States

The Queen Elizabeth University; 🇬🇧 Glasgow, Scottland, United Kingdom

Birmingham Women's and Children's Hospital; 🇬🇧 Birmingham, United Kingdom

Royal London Hospital; 🇬🇧 London, United Kingdom

Chelsea and Westminster Hospital; 🇬🇧 London, United Kingdom

Hammersmith Hospital; 🇬🇧 London, United Kingdom

University of Florida Gainesville; 🇺🇸 Gainesville, Florida, United States

Vanderbilt University; 🇺🇸 Nashville, Tennessee, United States

Rady Children's Hospital San Diego; 🇺🇸 San Diego, California, United States

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

Indiana University School of Medicine; 🇺🇸 Indianapolis, Indiana, United States

National Institutes of Health Hatfield Clinical Research Center; 🇺🇸 Bethesda, Maryland, United States

University of Utah; 🇺🇸 Salt Lake City, Utah, United States