Prader-Willi syndrome (PWS) MedDRA version: 28.0 Level: PT Classification code 10036476 Term: Prader-Willi syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: DCCR (Diazoxide Choline) Extended- Release Tablet
Product Code: DCCR
Pharmaceutical Form: Prolonged-release tablet
INN or Proposed INN: Diazoxide choline
Other descriptive name: Diazoxid
Sponsors
Soleno Therapeutics UK LTD
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (subjects, as appropriate). Participant must: Have participated in and completed the Study C602 Randomized Withdrawal (RW) Period; Have participated in but discontinued from the Study C602 RW Period and at least 16 weeks have elapsed since the date of their randomization into the C602 RW Period; or Have participated in Study C602 OLE period, did not consent to participate in the RW Period, and at least 16 weeks have elapsed since the date of their C602 Open-Label Extension End of Treatment Visit. Are the trial subjects under 18? yes Number of subjects for this age range: 57 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 26 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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. Participation in a clinical study of an investigational drug (including approved drugs for unapproved uses), investigational device, or therapeutic intervention subsequent to the C602 Open-Label Extension End of Treatment Visit.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to monitor long-term safety of DCCR (Diazoxide Choline) Extended-Release Tablets in participants with Prader-Willi syndrome (PWS).;Secondary Objective: There are no secondary objectives in Study C614. Additional objectives of this study are to monitor the long-term efficacy of DCCR in participants with PWS as assessed by changes in hyperphagia (assessed by the Hyperphagia Questionnaire for Clinical Trials [HQCT]) and changes in other PWS behaviors (Anxiety, Compulsivity, Rigidity/Irritability, Aggression, Disordered Thinking, Depression domains) as assessed by the PWS Profile (PWSP) questionnaire.;Primary end point(s): The primary outcome measures include: adverse event reports, clinical laboratory assessments (including fasting plasma glucose and blood glucose by glucometer, HbA1c, chemistry panel, liver function tests, hematology panel, urinalysis, and the Columbia Suicide Severity Rating Scale (C-SSRS)).;Timepoint(s) of evaluation of this end point: Adverse events, C-SSRS, and blood glucose are evaluated at Weeks 0, 2-6, 13, 26, 52, 78, 104, 130, 156, 182, 208, 234, 260. C-SSRS is also evaluated at Week 262. Fasting plasma glucose, HbA1c, chemistry panel, liver function tests, hematology panel, and urinalysis are evaluated at Weeks 0, 52, 104, 156, 208, and 260. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): There are no secondary outcome measures in Study C614. Additional outcome measures include: changes in hyperphagia (HQ-CT Total Score) and changes in other PWS behaviours (Anxiety, Compulsivity, Rigidity/Irritability, Aggression, Disordered Thinking, Depression domains) as assessed by the PWSP questionnaire.;Timepoint(s) of evaluation of this end point: The HQ-CT and the PWSP are evaluated at weeks 0, 13, 26, and 52 in year 1. During years 2-5 evaluation will occur at weeks 104, 156, 208, and 260. | — |
Countries
United Kingdom, United States
Contacts
Public ContactClinical Trial Information
Soleno Therapeutics UK LTD
Outcome results
None listed