Clinical trial NCT03714373: Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Prader-Willi Syndrome
Keywords: PWS
Prader-Willi Syndrome
Lead sponsor: Soleno Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2018-10-22
Enrollment: 115
Sex: ALL
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

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).

Eligibility criteria: 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

Primary outcomes:
measure: Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported
timeFrame: Baseline to end of OLE (up to 4 years)
measure: Change from RW Period Baseline in HQ-CT Total Score
timeFrame: RW Period Baseline to Week 16
Secondary outcomes:
measure: Change from Baseline in HQ-CT Total Score
timeFrame: Baseline to end of OLE (up to 4 years)
measure: Change in Body Fat Mass
timeFrame: Baseline to end of OLE (up to 4 years)
measure: Clinical Global Impression of Improvement (CGI-I)
timeFrame: RW Period Week 16
measure: Clinical Global Impression of Severity (CGI-S)
timeFrame: RW Period Week 16
measure: Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported
timeFrame: through the end of the RW Period, 16 weeks
Other outcomes:
Locations:
city: Orange
country: United States
facility: University of California, Irvine
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital San Diego
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Gainesville
country: United States
facility: University of Florida Gainesville
state: Florida
city: Atlanta
country: United States
facility: Emory Children's Center
state: Georgia
city: Indianapolis
country: United States
facility: Indiana University School of Medicine
state: Indiana
city: Kansas City
country: United States
facility: Kansas University Medical Center
state: Kansas
city: Bethesda
country: United States
facility: National Institutes of Health Hatfield Clinical Research Center
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Lansing
country: United States
facility: Sparrow Clinical Research Institute
state: Michigan
city: Saint Paul
country: United States
facility: Children's Minnesota
state: Minnesota
city: Paterson
country: United States
facility: St. Joseph's University Medical Center
state: New Jersey
city: Mineola
country: United States
facility: NYU Winthrop Hospital
state: New York
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center
state: Ohio
city: Columbus
country: United States
facility: The Research Institute at Nationwide Children's Hospital
state: Ohio
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
city: Dallas
country: United States
facility: Research Institute of Dallas
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Glasgow
country: United Kingdom
facility: The Queen Elizabeth University
state: Scottland
city: Hull
country: United Kingdom
facility: Hull and East Yorkshire Hospitals NHS Trust
state: Yorkshire
city: Birmingham
country: United Kingdom
facility: Birmingham Women's and Children's Hospital
city: Cambridge
country: United Kingdom
facility: Fulbourn Hospital
city: Liverpool
country: United Kingdom
facility: Aintree University Hospital NHS Foundation Trust
city: London
country: United Kingdom
facility: Royal London Hospital
city: London
country: United Kingdom
facility: Chelsea and Westminster Hospital
city: London
country: United Kingdom
facility: Hammersmith Hospital
Study officials:
References: