Open-Label Extension Study of Diazoxide Choline in Patients With Prader-Willi Syndrome

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

Tracking Information

• First Submitted Date: September 27, 2018
• First Posted Date: October 22, 2018
• Last Update Posted Date: June 18, 2020
• Actual Study Start Date: October 1, 2018
• Estimated Primary Completion Date: April 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (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 (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.

Current Secondary Outcome Measures (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 (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: Open-Label Extension Study of Diazoxide Choline in Patients With Prader-Willi Syndrome
• Official Title: 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: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Prader-Willi Syndrome

Intervention

Drug: DCCR

Once daily oral administration

Study Arms

Experimental: DCCR

75 - 450 mg DCCR

Intervention: Drug: DCCR

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Estimated Enrollment (submitted: October 19, 2018): 105
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: June 2023
• Estimated Primary Completion Date: April 2021 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

• 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: NCT03714373
• Other Study ID Numbers: 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: 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: June 2020