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.
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
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
115
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
Once daily oral administration of double-blind (placebo for DCCR) tablet(s) during the RW Period
Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported
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.
Time frame: Baseline to end of OLE (up to 4 years)
Change from RW Period Baseline in HQ-CT Total Score
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.
Time frame: RW Period Baseline to Week 16
Change from Baseline in HQ-CT Total Score
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.
Time frame: Baseline to end of OLE (up to 4 years)
Change in Body Fat Mass
Change in Body Fat Mass (kg) using DXA
Time frame: Baseline to end of OLE (up to 4 years)
Clinical Global Impression of Improvement (CGI-I)
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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University of California, Irvine
Orange, California, United States
Stanford University
Palo Alto, California, United States
Rady Children's Hospital San Diego
San Diego, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
University of Florida Gainesville
Gainesville, Florida, United States
Emory Children's Center
Atlanta, Georgia, United States
Indiana University School of Medicine
Indianapolis, Indiana, United States
Kansas University Medical Center
Kansas City, Kansas, United States
National Institutes of Health Hatfield Clinical Research Center
Bethesda, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
...and 18 more locations
Time frame: RW Period Week 16
Clinical Global Impression of Severity (CGI-S)
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).
Time frame: RW Period Week 16
Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported
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.
Time frame: through the end of the RW Period, 16 weeks