A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome

• ClinicalTrials.gov Identifier: NCT03440814
• Recruitment Status: Active, not recruiting
• First Posted: February 22, 2018
• Last Update Posted: January 13, 2020
• Sponsor: Soleno Therapeutics, Inc.
• Information provided by (Responsible Party): Soleno Therapeutics, Inc.

Tracking Information

• First Submitted Date: February 13, 2018
• First Posted Date: February 22, 2018
• Last Update Posted Date: January 13, 2020
• Actual Study Start Date: May 9, 2018
• Estimated Primary Completion Date: May 2020 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: February 19, 2018)

Hyperphagia Score [ Time Frame: Baseline to Week 13 ]

Change in hyperphagia-related behavior as measured by total score of a Hyperphagia Questionnaire

• Original Primary Outcome Measures: Same as current
• Change History: Complete list of historical versions of study NCT03440814 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures (submitted: February 19, 2018)

• Body fat mass (DXA) [ Time Frame: Baseline to Week 13 ]
  Change in Body Fat Mass from Baseline to Week 13
• CGI-I [ Time Frame: at Week 13 ]
  Clinical Global Impression of Improvement
• Caregiver GI-C [ Time Frame: at Week 13 ]
  Caregiver Global Impression of Change

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome
• Official Title: A Randomized, Double-Blind, Placebo-Controlled Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome
• Brief Summary: The purpose of this is study is to evaluate the effects of DCCR (diazoxide choline controlled release tablets) in children and adults with Prader-Willi syndrome.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Treatment
• Condition: Prader-Willi Syndrome

Intervention

• Drug: DCCR
  Once daily oral administration
• Drug: Placebo for DCCR
  Once daily oral administration

Study Arms

• Experimental: DCCR
  75 - 450 mg DCCR
  Intervention: Drug: DCCR
• Placebo Comparator: Placebo
  75 - 450 mg placebo for DCCR
  Intervention: Drug: Placebo for DCCR

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Estimated Enrollment (submitted: February 19, 2018): 105
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: May 2020
• Estimated Primary Completion Date: May 2020 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (patients, as appropriate)
• Genetically-confirmed Prader-Willi syndrome and hyperphagic
• In a stable care setting for at least 6 months prior to Visit 1
• Caregiver must have been caring for the patient for at least 6 months prior to Visit 1

Exclusion Criteria:

• Have participated in an interventional clinical study (i.e., investigational drug or device, approved drugs or device evaluated for unapproved use) within prior 3 months
• 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 30 days after study participation
• Any other known disease and/or condition, which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 4 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United Kingdom, United States

Administrative Information

• NCT Number: NCT03440814
• Other Study ID Numbers: C601
• Has Data Monitoring Committee: Yes

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: Not Provided
• Responsible Party: Soleno Therapeutics, Inc.
• Study Sponsor: Soleno Therapeutics, Inc.
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Soleno Therapeutics, Inc.
• Verification Date: December 2019