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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03714373
Recruitment Status : Active, not recruiting
First Posted : October 22, 2018
Last Update Posted : June 18, 2020
Sponsor:
Information provided by (Responsible Party):
Soleno Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE September 27, 2018
First Posted Date  ICMJE October 22, 2018
Last Update Posted Date June 18, 2020
Actual Study Start Date  ICMJE October 1, 2018
Estimated Primary Completion Date April 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 10, 2019)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: October 19, 2018)
Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported [ Time Frame: Baseline to Week 40 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.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 10, 2019)
  • 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.
  • Body fat mass (DXA) [ Time Frame: Baseline at Week 52 ]
    Change in Body Fat Mass from Baseline to Week 52
  • 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).
  • 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).
Original Secondary Outcome Measures  ICMJE
 (submitted: October 19, 2018)
  • Hyperphagia Score [ Time Frame: Baseline to Week 38 ]
    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.
  • Body fat mass (DXA) [ Time Frame: Baseline at Week 38 ]
    Change in Body Fat Mass from Baseline to Week 38
  • Clinical Global Impression of Improvement (CGI-I) [ Time Frame: at Week 38 ]
    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).
  • Caregiver Global Impression of Change (Caregiver GI-C) [ Time Frame: at Week 38 ]
    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).
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Open-Label Extension Study of Diazoxide Choline in Patients With Prader-Willi Syndrome
Official Title  ICMJE An Open-Label, Long-Term Safety Evaluation of Diazoxide Choline Controlled-Release Tablet in Patients With Prader-Willi Syndrome
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Prader-Willi Syndrome
Intervention  ICMJE Drug: DCCR
Once daily oral administration
Study Arms  ICMJE Experimental: DCCR
75 - 450 mg DCCR
Intervention: Drug: DCCR
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: October 19, 2018)
105
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2023
Estimated Primary Completion Date April 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 4 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03714373
Other Study ID Numbers  ICMJE C602
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Soleno Therapeutics, Inc.
Study Sponsor  ICMJE Soleno Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Soleno Therapeutics, Inc.
Verification Date June 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP