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    Summary
    EudraCT Number:2018-004216-22
    Sponsor's Protocol Code Number:C602
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-06-27
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2018-004216-22
    A.3Full title of the trial
    An Open-Label, Long-Term Safety and Efficacy Evaluation of Diazoxide Choline Controlled-Release Tablet in Patients with Prader-Willi Syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to determine the safety of the study drug Diazoxide Choline Controlled-Release Tablet after being given for a long time to patients with the genetic disorder Prader-Willi Syndrome.
    A.4.1Sponsor's protocol code numberC602
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSoleno Therapeutics UK Ltd.
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSoleno Therapeutics, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSoleno Therapeutics UK Limited
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street AddressGarden Cottage, Badgemore Park
    B.5.3.2Town/ cityHenley on Thames, Oxon
    B.5.3.3Post codeRG9 4NR
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+441628876432
    B.5.6E-mailC602ProjectManager@soleno.life
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1941
    D.3 Description of the IMP
    D.3.1Product namediazoxide choline
    D.3.2Product code DCCR
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDiazoxide choline
    D.3.9.1CAS number 1098065-76-9
    D.3.9.2Current sponsor codeDCCR
    D.3.9.3Other descriptive nameDIAZOXIDE CHOLINE
    D.3.9.4EV Substance CodeSUB192374
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number25 to 150
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCoated tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Hyperphagia associated with Prader-Willi Syndrome (PWS)
    E.1.1.1Medical condition in easily understood language
    Genetic disorder (Prader-Willi) causing a weakness in shape and form of muscle, poor growth, delayed development in infants & increased appetite and food-related behaviour later on.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10020710
    E.1.2Term Hyperphagia
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to evaluate the long-term safety of diazoxide choline controlled-release (DCCR) tablets in PWS patients.
    E.2.2Secondary objectives of the trial
    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.
    E.2.3Trial contains a sub-study No
    E.3Principal 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.
    E.4Principal 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.).
    E.5 End points
    E.5.1Primary 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 (DXA) change from Baseline
    • Lean body mass (DXA) change from Baseline
    • Lean body mass / fat mass ratio (DXA) change from Baseline
    • Change in cardiometabolic markers (high-sensitivity C-reactive protein, adiponectin, ghrelin and leptin) from Baseline
    • Change in insulin resistance (HOMA-IR) from Baseline
    • Clinical Global Impression of Improvement
    • Caregiver Global Impression of Change.
    All efficacy and exploratory endpoints may be evaluated from Baseline to Visits 3, 5, 6, 15, 19 and/or 23.

    E.5.1.1Timepoint(s) of evaluation of this end point
    End of study
    E.5.2Secondary 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 Thinking domain) from Baseline
    • Change in individual infrequent behaviours of high clinical significance (PWS Profile questionnaire) from Baseline
    All exploratory endpoints may be evaluated from Baseline to Visits 3, 5, 6, 15, 19 and/or 23.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Visit 15
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned10
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The end of trial is the completion of the analyses of the final data. Data are considered final when all queries related to the data are resolved, the data are clean, and the database has been locked.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months5
    E.8.9.2In all countries concerned by the trial days17
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 25
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 25
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 55
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 25
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Intellectual disability is common in PWS. Patient assent and parent/guardian consent will be obtained. Sexual maturity is usually not achieved without aid and puberty is rarely completed. Where pregnancy is possible, real contraception is required.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 105
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Depending on the safety and efficacy profile of DCCR in this study and in study C601, a further long-term safety study may follow this study. Alternatively, the IMP may be made available to subjects on a named patient basis or they may continue on their normal pre-study treatment for the condition.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Clinical Research Network North Thames
    G.4.3.4Network Country United Kingdom
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-07-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-09-25
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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