Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome

An Open-Label Study of DCCR (Diazoxide Choline) Extended-Release Tablets in Patients With Prader-Willi Syndrome

The purpose of this is study is to evaluate the long-term safety of DCCR (diazoxide choline) extended-release tablets) in patients with Prader-Willi syndrome.

Study Overview

• Status: Active, not recruiting
• Conditions: Prader-Willi Syndrome
• Intervention / Treatment: Drug: DCCR

• Study Type: Interventional
• Enrollment (Estimated): 83
• Phase: Phase 3

Contacts and Locations

Study Locations

• United Kingdom
  • Cambridge, United Kingdom, CB21 5EF
    • Fulbourn Hospital
  • Glasgow, United Kingdom, G51 4TF
    • The Queen Elizabeth University
  • Hull, United Kingdom, HU3 2JZ
    • Hull and East Yorkshire Hospitals NHS Trust
  • London, United Kingdom, SW10 9NH
    • Chelsea and Westminster Hospital
  • London, United Kingdom, E1 1BB
    • Royal London Hospital
• United States
  • California
    • Orange, California, United States, 92868
      • UC Irvine
    • Palo Alto, California, United States, 94304
      • Stanford University
    • San Diego, California, United States, 92123
      • Rady Children's Hospital of San Diego
  • Florida
    • Gainesville, Florida, United States, 32608
      • U of Florida Gainesville
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Emory Children's Center
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Indiana University School of Medicine
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • National Institutes of Health
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
  • Michigan
    • Lansing, Michigan, United States, 48912
      • Sparrow Clinical Research Institute
  • Minnesota
    • Saint Paul, Minnesota, United States, 55102
      • Children's Hospital and Clinic Minnesota
  • New Jersey
    • Paterson, New Jersey, United States, 07503
      • St. Joseph's University Medical Center
  • New York
    • Mineola, New York, United States, 11501
      • NYU Winthrop Hospital
  • Ohio
    • Columbus, Ohio, United States, 43205
      • The Research Institute at Nationwide Children's Hospital
  • Tennessee
    • Nashville, Tennessee, United States, 37212
      • Vanderbilt University
  • Texas
    • Dallas, Texas, United States, 75231
      • Research Institute of Dallas
  • Utah
    • Salt Lake City, Utah, United States, 84112
      • University of Utah
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (subjects, as appropriate)

2. Participant must:

   1. Have participated in and completed the Study C602 Randomized Withdrawal (RW) Period;
   2. Have participated in but discontinued from the Study C602 RW Period and at least 16 weeks have elapsed since the date of their randomization into the C602 RW Period; or
   3. Have participated in Study C602 OLE period, did not consent to participate in the RW Period, and at least 16 weeks have elapsed since the date of their C602 Open-Label Extension End of Treatment Visit.

Exclusion Criteria:

1. Positive urine pregnancy test (in females of child-bearing potential)
2. Females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 30 days after study participation.
3. Participation in a clinical study of an investigational drug (including approved drugs for unapproved uses), investigational device, or therapeutic intervention subsequent to the C602 Open-Label Extension End of Treatment Visit.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: DCCR; 75 - 525 mg DCCR	Drug: DCCR; Once daily oral administration

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of DCCR (adverse events. Safety analyses will be conducted in all subjects who receive at least one dose of DCCR in the C614 study. ( Adverse events)	Assess the safety of DCCR by evaluating the incidence and severity of adverse events. Safety analyses will be conducted in all subjects who receive at least one dose of DCCR in the C614 study. Adverse events will be described by type and level of severity.	Baseline to Week 262 or until resolution of certain adverse events

Collaborators and Investigators

• Sponsor: Soleno Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): January 31, 2023
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): June 1, 2028

Study Registration Dates

• First Submitted: January 18, 2023
• First Submitted That Met QC Criteria: January 26, 2023
• First Posted (Actual): January 27, 2023

Study Record Updates

• Last Update Posted (Actual): April 2, 2024
• Last Update Submitted That Met QC Criteria: March 29, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Overnutrition; Nutrition Disorders; Overweight; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Imprinting Disorders; Syndrome; Prader-Willi Syndrome
• Other Study ID Numbers: C614

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No