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

Overview

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.

Full Title of Study: “A Randomized, Double-Blind, Placebo-Controlled Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Triple (Participant, Care Provider, Investigator)
  • Study Primary Completion Date: May 1, 2020

Interventions

  • Drug: DCCR
    • Once daily oral administration
  • Drug: Placebo for DCCR
    • Once daily oral administration

Arms, Groups and Cohorts

  • Experimental: DCCR
    • 75 – 450 mg DCCR
  • Placebo Comparator: Placebo
    • 75 – 450 mg placebo for DCCR

Clinical Trial Outcome Measures

Primary Measures

  • Hyperphagia Score
    • Time Frame: Baseline to Week 13
    • Change in hyperphagia-related behavior as measured by total score of a Hyperphagia Questionnaire

Secondary Measures

  • Body fat mass (DXA)
    • Time Frame: Baseline to Week 13
    • Change in Body Fat Mass from Baseline to Week 13
  • CGI-I
    • Time Frame: at Week 13
    • Clinical Global Impression of Improvement
  • Caregiver GI-C
    • Time Frame: at Week 13
    • Caregiver Global Impression of Change

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 4 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Soleno Therapeutics, Inc.
  • Provider of Information About this Clinical Study
    • Sponsor

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