Working…
Help guide our efforts to modernize ClinicalTrials.gov.
Send us your comments by March 14, 2020.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Open-Label Extension 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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03714373
Recruitment Status : Recruiting
First Posted : October 22, 2018
Last Update Posted : January 29, 2020
Sponsor:
Information provided by (Responsible Party):
Soleno Therapeutics, Inc.

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
The purpose of this is study is to evaluate the long term safety of DCCR (diazoxide choline controlled release tablets) in children and adults with Prader-Willi syndrome. After completion of 1 year in this study, subjects will have the option to continue treatment for up to 2 additional years.

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Drug: DCCR Phase 3

Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 105 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Long-Term Safety Evaluation of Diazoxide Choline Controlled-Release Tablet in Patients With Prader-Willi Syndrome
Actual Study Start Date : October 1, 2018
Estimated Primary Completion Date : April 2021
Estimated Study Completion Date : June 2023

Resource links provided by the National Library of Medicine

Drug Information available for: Diazoxide

Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
Experimental: DCCR
75 - 450 mg DCCR
 Drug: DCCR
Once daily oral administration


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported [ Time Frame: Baseline to Week 54 or until resolution of certain adverse events at the discretion of the Investigator and Sponsor ]
    Safety analyses will be conducted in all subjects who receive at least one dose of DCCR. Adverse events will be described by type and level of severity.


Secondary Outcome Measures :
  1. Hyperphagia Score [ Time Frame: Baseline to Week 52 ]
    Hyperphagia-related behaviors will be assessed by the hyperphagia questionnaire for clinical trials (HQ-CT), an instrument designed to measure symptoms of food related preoccupations and behaviors. The HQ-CT consists of nine items with responses ranging from 0-4 (best to worst). Scores from 9 items will be summed for a possible total score range of 0−36.

  2. Body fat mass (DXA) [ Time Frame: Baseline at Week 52 ]
    Change in Body Fat Mass from Baseline to Week 52

  3. Clinical Global Impression of Improvement (CGI-I) [ Time Frame: at Week 52 ]
    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 provides 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 (best to worst).

  4. Caregiver Global Impression of Change (Caregiver GI-C) [ Time Frame: at Week 52 ]
    The Caregiver 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 provides 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 (best to worst).


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Successful completion of clinical study C601
  • Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (subjects, as appropriate)

Key Exclusion Criteria:

  • 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 90 days after study participation
  • Any new disease, condition, or circumstance which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol (e.g., an anticipated change of care setting)
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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): NCT03714373


Contacts
Layout table for location contacts
Contact: C602 Project Manager 650-353-2051 C602ProjectManager@soleno.life

Locations
Show Show 19 study locations
Sponsors and Collaborators
Soleno Therapeutics, Inc.
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for additonal information
Responsible Party: Soleno Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03714373    
Other Study ID Numbers: C602
First Posted: October 22, 2018    Key Record Dates
Last Update Posted: January 29, 2020
Last Verified: January 2020

Layout table for additional information
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:
Layout table for MeSH terms
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
 Overnutrition
Nutrition Disorders
Diazoxide
Choline
Lipotropic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Gastrointestinal Agents
Lipid Regulating Agents
Nootropic Agents
Antihypertensive Agents
Vasodilator Agents