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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03440814
Recruitment Status : Completed
First Posted : February 22, 2018
Last Update Posted : June 8, 2022
Sponsor:
Information provided by (Responsible Party):
Soleno Therapeutics, Inc.

Study Description
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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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 127 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
Actual Primary Completion Date : May 1, 2020
Actual Study Completion Date : May 1, 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
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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


Outcome Measures
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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


Eligibility Criteria
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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
Contacts and Locations
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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


Locations
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Sponsors and Collaborators
Soleno Therapeutics, Inc.
Investigators
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Principal Investigator: Evelien Gevers, MD, PhD Queen Mary University of London, Barts Health NHS Trust
Principal Investigator: Jennifer L. Miller, MD University of Florida
More Information
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Responsible Party: Soleno Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03440814    
Other Study ID Numbers: C601
First Posted: February 22, 2018    Key Record Dates
Last Update Posted: June 8, 2022
Last Verified: November 2021

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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:
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Prader-Willi Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
 Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overweight
Overnutrition
Nutrition Disorders