Hyperphagia associated with Prader-Willi Syndrome (PWS) MedDRA version: 20.0 Level: PT Classification code 10020710 Term: Hyperphagia System Organ Class: 10027433 - Metabolism and nutrition disorders
Conditions
Interventions
Product Name: diazoxide choline
Product Code: DCCR
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Diazoxide choline
CAS Number: 1098065-76-9
Current Sponsor code: DCCR
Other descriptive
Sponsors
Soleno Therapeutics UK Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: The following are considered to be the Principal Inclusion Criteria for Study C602: 1) Successful completion of clinical study C601; 2) Provide voluntary, written informed consent (parent(s) / legal guardian(s) of subject); provide voluntary, written assent (subject, as appropriate); 3) Primary caregiver must be able to communicate with Investigator and study site personnel as well as read and complete the study-required questionnaires. Are the trial subjects under 18? yes Number of subjects for this age range: 25 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 25 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: The following are considered to be the Principal Exclusion Criteria for Study C602. Participants cannot enter C602 if not completed C601: 1) Anticipated requirement for use at any time during the study of the following prohibited medications: • Anti-obesity medications or other medications (including herbal preparations, over-the-counter products) or procedures for weight reduction; • Medications, including homeopathy and herbal preparations, that are strong inhibitors or inducers of CYP450 1A2 or 3A4 (Refer to http://medicine.iupui.edu/CLINPHARM/ddis/clinical-table, Flockhart Table); • Medications known to prolong the QTc interval (Refer to QT Drugs List on https://crediblemeds.org/healthcare-providers/), except citalopram and escitalopram; • Systemic steroids (i.e., oral, IM, or IV) for > 7 days; • Any drugs medications, herbal preparation, homeopathy, nutraceuticals, or procedures (i.e., acupuncture, vagal stimulation), that may have an effect on safety endpoints; • Use of any investigational drugs or devices; 2) Positive urine pregnancy test (in females of childbearing potential); 3) Females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 90 days after study participation; 4) 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); 5) New history in a first degree relative since the subject enrolled in clinical study C601 or a change in the subject’s physical examination that, in the opinion of the investigator, significantly increases the subject’s risk for thromboembolic event (e.g., venous thrombosis, pulmonary embolism, etc.).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to evaluate the long-term safety of diazoxide choline controlled-release (DCCR) tablets in PWS patients.;Secondary Objective: The secondary objectives of this study are to evaluate changes in hyperphagia, body fat mass, lean body mass, lean body mass/body fat mass ratio, cardiometabolic markers, insulin resistance (HOMA-IR), Clinical Global Impression of Improvement (CGI-I), and Caregiver Global Impression of Change (GI-C) associated with DCCR treatment of PWS patients. Additional objectives of this study include the evaluation of changes in percent body fat, weight, body mass index (BMI), waist circumference, lipid parameters, patient’s health-related quality of life, caregiver burden, cardiometabolic markers, Clinical Global Impression of Severity (CGI-S), Caregiver Global Impression of Severity, Caregiver Global Impression of Change (GI-C) of Food-Related Behaviours for Hyperphagia Questionnaire for Clinical Trials (HQ-CT), Caregiver GI-C of Overall Behaviour, Environmental controls on food access, Developmental Behaviour Checklist 2-Parent (DBC2-P), PWS behaviours with DCCR treatment of PWS patients.;Primary end point(s): The following safety & efficacy endpoints will be evaluated in this study. Safety: • Adverse events • Vital signs • Electrocardiogram (ECG) • Peripheral edema assessment • Thromboembolic event assessment • Hirsutism / hypertrichosis assessment • Clinical laboratory assessments: • Fasting plasma glucose - Fasting plasma insulin - HbA1c - Chemistry panel - Liver function tests - Hematology panel - Prothrombin time, (PT), activated partial thromboplastin time (APTT), and international normalized ratio (INR) - Insulin resistance (HOMA-IR) - Urinalysis • Insulin-like Growth Factor-1 (IGF-1) • Columbia Suicide Severity Rating Scale (C-SSRS, caregiver response) • PWS Behaviours associated with suicide risk. Efficacy Endpoints: • Hyperphagia change from Baseline (using HQ-CT) • Body fat mass (D | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): For all exploratory endpoints, Baseline 1 = clinical study C601 Visit 2 and Baseline 2 = clinical study C601 Visit 7 / clinical study C602 Visit 1. All exploratory endpoints will evaluate the changes or percent change from both Baseline 1 and Baseline 2. The following exploratory endpoints will also be evaluated in this study: • Change in percent body fat (DXA) from Baseline • Weight change from Baseline • BMI change from Baseline • Waist circumference change from Baseline • Triglyceride percent change from Baseline • Total cholesterol percent change from Baseline • Non-high-density lipoprotein (HDL) cholesterol percent change from Baseline • Low-density lipoprotein (LDL) and high-density lipoprotein (HDL) cholesterol percent change from Baseline • Change in subject’s health related Quality of Life (using EQ-5D-Y:1, Proxy Version) from Baseline • Caregiver burden (by Zarit Burden Interview [ZBI]) change from Baseline • Change in Clinical Global Impression of Severity from Baseline • Change in Caregiver Global Impression of Severity from Baseline • Change in Caregiver Global Impression of Change of Food-Related Behaviors for HQ-CT • Change in Caregiver Global Impression of Change of Overall Behaviour • Change in environmental controls on food access (using Food Safe Zone) from Baseline 1 • Change in the Developmental Behaviour Checklist 2 – Parent (DBC2-P) from Baseline • Change in externalizing behaviors, aggressive problems (PWS Profile questionnaire - Aggression domain) from Baseline • Change in anxious behaviors (PWS Profile questionnaire – Anxiety domain) from Baseline • Change in compulsivity (PWS Profile questionnaire – Compulsivity domain) from Baseline • Change in irritability (PWS Profile questionnaire – Rigidity, Irritability domain) from Baseline • Change in depressive behaviors (PWS Profile questionnaire – Depression domain) from Baseline • Change in disordered thinking (PWS Profile questionnaire – Disordered | — |
Countries
United Kingdom, United States
Contacts
Public ContactClinical Trial Information
Soleno Therapeutics UK Limited
Outcome results
None listed