- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03714373
Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period
June 26, 2023 updated by: Soleno Therapeutics, Inc.
An Open-Label, Long-Term Safety and Efficacy Evaluation of Diazoxide Choline Extended-Release Tablets in Participants With Prader-Willi Syndrome With a Double-Blind, Placebo-Controlled, Randomized Withdrawal Period
This is a multi-center, multi-period study with an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period evaluating the safety and efficacy of DCCR treatment.
Study Overview
Status
Active, not recruiting
Conditions
Intervention / Treatment
Detailed Description
115 PWS participants who completed clinical study C601 will be enrolled into the OLE Period.
All participants in the Open Label Extension (OLE) Period will receive open-label DCCR.
The actual number of participants eligible to enroll in the double-blind, placebo-controlled, randomized withdrawal (RW) period will be limited to those participants taking DCCR in the OLE Period at the time of the RW Period Visit 1 (Baseline/Randomization Visit).The treatment groups in the C602 RW Period are those participants randomized to receive DCCR and those participants randomized to receive Placebo.
Participants will be randomized in a 1:1 ratio (DCCR:Placebo).
Study Type
Interventional
Enrollment (Estimated)
115
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Birmingham, United Kingdom, B4 6NH
- Birmingham Women's and Children's Hospital
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Cambridge, United Kingdom, CB21 5ER
- Fulbourn Hospital
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Liverpool, United Kingdom, L9 7AL
- Aintree University Hospital NHS Foundation Trust
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London, United Kingdom, SW10 9NH
- Chelsea and Westminster Hospital
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London, United Kingdom, E1 1BB
- Royal London Hospital
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London, United Kingdom, W12 OHS
- Hammersmith Hospital
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Scottland
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Glasgow, Scottland, United Kingdom, G51 4TF
- The Queen Elizabeth University
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Yorkshire
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Hull, Yorkshire, United Kingdom, HU3 2JZ
- Hull and East Yorkshire Hospitals NHS Trust
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California
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Orange, California, United States, 92868
- University of California, Irvine
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Palo Alto, California, United States, 94305
- Stanford University
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San Diego, California, United States, 92123
- Rady Children's Hospital San Diego
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Colorado
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Aurora, Colorado, United States, 80045
- Children's Hospital Colorado
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Florida
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Gainesville, Florida, United States, 32608
- University of Florida Gainesville
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Georgia
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Atlanta, Georgia, United States, 30322
- Emory Children's Center
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Indiana
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Indianapolis, Indiana, United States, 46202
- Indiana University School of Medicine
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Kansas
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Kansas City, Kansas, United States, 66160
- Kansas University Medical Center
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Maryland
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Bethesda, Maryland, United States, 20892
- National Institutes of Health Hatfield Clinical Research Center
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Boston Children's Hospital
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Michigan
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Lansing, Michigan, United States, 48912
- Sparrow Clinical Research Institute
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Minnesota
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Saint Paul, Minnesota, United States, 55102
- Children's Minnesota
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New Jersey
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Paterson, New Jersey, United States, 07503
- St. Joseph's University Medical Center
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New York
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Mineola, New York, United States, 11501
- NYU Winthrop Hospital
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Ohio
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Cleveland, Ohio, United States, 44106
- University Hospitals Cleveland Medical Center
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Columbus, Ohio, United States, 43205
- The Research Institute at Nationwide Children's Hospital
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Tennessee
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Nashville, Tennessee, United States, 37212
- Vanderbilt University
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Texas
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Dallas, Texas, United States, 75231
- Research Institute Of Dallas
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Utah
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Salt Lake City, Utah, United States, 84108
- University of Utah
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Washington
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Seattle, Washington, United States, 98105
- Seattle Children's Hospital
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
4 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
OLE Period 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)
OLE Period 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)
RW Period Key Inclusion Criteria:
- Provide voluntary, written informed consent (parent[s] / legal guardian[s] of participant); provide voluntary, written assent (participants, as appropriate); this includes consent for randomization and potential treatment with placebo for up to 16 weeks
- Currently participating in clinical study C602 and complete the OLE End of Treatment Visit procedures
RW Period Key Exclusion Criteria:
- Positive urine pregnancy test (in females of child-bearing potential)
- Females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 30 days after study participation
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: OLE DCCR
75 - 525 mg DCCR
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Once daily oral administration of open-label DCCR tablet(s) during the OLE Period
Once daily oral administration of double-blind (DCCR) tablet(s) during the RW Period
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Experimental: RW DCCR
75 - 525 mg DCCR
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Once daily oral administration of open-label DCCR tablet(s) during the OLE Period
Once daily oral administration of double-blind (DCCR) tablet(s) during the RW Period
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Placebo Comparator: RW Placebo
75 - 525 mg Placebo for DCCR
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Once daily oral administration of double-blind (placebo for DCCR) tablet(s) during the RW Period
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported
Time Frame: Baseline to end of OLE (up to 4 years)
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Safety analyses will be conducted in all participants who receive at least one dose of DCCR.
Adverse events will be described by type and level of severity.
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Baseline to end of OLE (up to 4 years)
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Change from RW Period Baseline in HQ-CT Total Score
Time Frame: RW Period Baseline to Week 16
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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.
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RW Period Baseline to Week 16
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change from Baseline in HQ-CT Total Score
Time Frame: Baseline to end of OLE (up to 4 years)
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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.
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Baseline to end of OLE (up to 4 years)
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Change in Body Fat Mass
Time Frame: Baseline to end of OLE (up to 4 years)
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Change in Body Fat Mass (kg) using DXA
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Baseline to end of OLE (up to 4 years)
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Clinical Global Impression of Improvement (CGI-I)
Time Frame: RW Period Week 16
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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).
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RW Period Week 16
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Clinical Global Impression of Severity (CGI-S)
Time Frame: RW Period Week 16
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The CGI-S is a single statement designed to assess the Investigator's overall perception of the severity of the participant's illness across the course of the clinical trial.
The Investigator provides a response to "Considering your total clinical experience with this particular population, how ill is this participant at this time:" by rating the subject's behavior using a 7-point response scale (best to worst).
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RW Period Week 16
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Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported
Time Frame: through the end of the RW Period, 16 weeks
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Safety analyses will be conducted in all participants who receive at least one dose of randomized withdrawal study drug.
Adverse events will be described by type and level of severity.
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through the end of the RW Period, 16 weeks
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
October 1, 2018
Primary Completion (Estimated)
August 1, 2023
Study Completion (Estimated)
August 1, 2023
Study Registration Dates
First Submitted
September 27, 2018
First Submitted That Met QC Criteria
October 19, 2018
First Posted (Actual)
October 22, 2018
Study Record Updates
Last Update Posted (Actual)
June 28, 2023
Last Update Submitted That Met QC Criteria
June 26, 2023
Last Verified
November 1, 2022
More Information
Terms related to this study
Keywords
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
- Syndrome
- Prader-Willi Syndrome
Other Study ID Numbers
- C602
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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