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    Summary
    EudraCT Number:2015-003394-15
    Sponsor's Protocol Code Number:VP-VEC-162-4201
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-07-25
    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 ConcernedGermany - BfArM
    A.2EudraCT number2015-003394-15
    A.3Full title of the trial
    OPEN-LABEL STUDY TO INVESTIGATE THE PHARMACOKINETICS AND SAFETY OF TASIMELTEON IN TOTALLY BLIND CHILDREN AND ADOLESCENTS WITH NON-24-HOUR SLEEP- WAKE DISORDER
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    OPEN-LABEL STUDY TO INVESTIGATE THE PHARMACOKINETICS AND SAFETY OF TASIMELTEON IN TOTALLY BLIND CHILDREN AND ADOLESCENTS WITH NON-24-HOUR SLEEP- WAKE DISORDER
    A.4.1Sponsor's protocol code numberVP-VEC-162-4201
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/123/2015
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorVanda Pharmaceuitcals Inc.
    B.1.3.4CountryUnited States
    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 supportVanda Pharmaceuitcals Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVanda Pharmaceuitcals Inc.
    B.5.2Functional name of contact pointJoseph Hull
    B.5.3 Address:
    B.5.3.1Street Address2200 Pennsylvania Ave NW, Suite 300E
    B.5.3.2Town/ cityWashington
    B.5.3.3Post codeDC 20037
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1202734 3469
    B.5.5Fax number+1202296 1450
    B.5.6E-mailJoseph.Hull@vandapharma.com
    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/3/10/841
    D.3 Description of the IMP
    D.3.1Product nameTasimelteon
    D.3.2Product code VEC-162
    D.3.4Pharmaceutical form Oral suspension
    D.3.4.1Specific paediatric formulation Yes
    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 INNTasimelteon
    D.3.9.1CAS number 609799-22-06
    D.3.9.2Current sponsor codeVEC-162
    D.3.9.3Other descriptive nameTASIMELTEON
    D.3.9.4EV Substance CodeSUB166225
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    This study will be an open-label, single dose, non-controlled trial to evaluate the pharmacokinetics and safety of tasimelteon in children who are 3 years to less than 18 years of age, totally blind with no conscious light perception and exhibit/have Non-24-Hour Sleep-Wake Disorder
    E.1.1.1Medical condition in easily understood language
    This study will evaluate the safety of tasimelteon in children who are 3 years to less than 18 years, totally blind with no conscious light perception and have Non-24-Hour Sleep-Wake Disorder
    E.1.1.2Therapeutic area Not possible to specify
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level PT
    E.1.2Classification code 10040984
    E.1.2Term Sleep disorder
    E.1.2System Organ Class 10037175 - Psychiatric disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • To determine a dose for pediatric subjects for which systemic exposure (AUC) is centered around the value expected in adults based on the nominal 20 mg adult dose.

    • To characterize the pharmacokinetics of an age-appropriate oral formulation of tasimelteon after a single dose administration.

    • To characterize metabolites M9, M11, M12, M13, and M14 pharmacokinetics in children and adolescents after a single dose administration of an age-appropriate oral formulation of tasimelteon.

    • To determine the safety and tolerability of a single oral dose of an age-appropriate oral formulation of tasimelteon.

    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent from parents having parental responsibility or from the legal guardian(s). In the case of a child is aged 12 years or older the written assent of the child is needed in addition to that of parents having responsibility/legal guardian;

    2. Males or females between 3 to <18 years;

    3. Children in cohort 1 (age 3 to < 6) must be daytime toilet trained;

    4. Body Mass Index (BMI) within the 5th and 95th percentile based on age (BMI =
    weight (kg)/ [height (m)]2);

    5. Weigh at least 16 kg;

    6. Post-pubescent girls already having menses must have a negative pregnancy test at the screening and baseline visits;

    7. No perception of light and Diagnosis of Non-24-Hour Sleep-Wake Disorder as determined by:

    a. History (within the last 3 months) of trouble sleeping at night (difficulty initiating sleep or staying asleep), difficulty awakening in the morning, or daytime sleepiness as determined by answering yes to at least one question in the Sleep Complaint Questionnaire, and

    b. A non-entrained urinary aMT6s rhythm defined as a tau ≥ 24.10 h and 95% confidence interval (CI) that does not include 24;

    8. Willing and able to comply with study requirements and restrictions including a commitment to a fixed sleep opportunity during the study;

    9. Subjects who fail to respond to behavior modification/sleep hygiene measures during the Pre-Enrollment Phase as determined by the physician through patient interview and/or sleep diaries
    E.4Principal exclusion criteria
    1. Have a probable diagnosis of a current sleep disorder other than Non-24-Hour Sleep-Wake Disorder that is the primary cause of the sleep disturbance based on clinical investigator medical judgment;

    2. History (within the 12 months prior to screening) of psychiatric disorders including ADHD, Autism, Neurodisabilities, Major Depressive Disorder, Generalized Anxiety Disorder, Axis II Disorders, delirium or any other psychiatric disorder, that is not being successfully treated or has not been resolved and that in the opinion of the clinical investigator would affect participation in the study or full compliance with study procedures;


    3. History of intolerance and/or hypersensitivity to melatonin or melatonin agonists;

    4. Subjects having any suicidal ideation of type 4 or 5 on the C-SSRS at Screening or Baseline;

    5. Subject is at risk of suicide, in the opinion of the Investigator. Evidence of suicide risk could include any suicide attempt within the past year or any other suicidal behavior within the past year;

    6. Current clinically significant cardiovascular, respiratory, neurologic, hepatic, hematopoietic, renal, gastrointestinal or metabolic dysfunction unless currently controlled and stable;

    7. Clinically significant deviation from normal in clinical laboratory results, vital signs measurements, or physical examination findings at screening as determined by the clinical investigator;

    8. Indication of impaired liver function (values for AST, ALT or bilirubin > 2 times
    Upper Limit of Normal);

    9. Pregnant or lactating females;

    10. Smokers;

    11. Exposure to any investigational drug, including placebo, within 30 days or 5 half- lives (whichever was longer) of screening;

    12. Unwilling or unable to follow the medication restrictions described in Section 8.2, or unwilling or unable to sufficiently wash-out from use of a restricted medication;

    13. Any other sound medical reason as determined by the clinical investigator
    E.5 End points
    E.5.1Primary end point(s)
    Pharmacokinetics Endpoints:
    • Individual subject plasma concentration for tasimelteon and its metabolites with actual blood sampling times.

    Due to the sparseness of data, the analysis will not attempt to predict a new set of pharmacokinetic parameters for these subjects. This information will be used to determine an
    optimal model for pediatric subjects, based on the pharmacokinetic parameters in adults, with appropriate scaling for body size. If the PK analysis of the pediatric population shows that the PK profile in children is different to the adult population, the sampling times above will be modified to ensure the optical design for sampling.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Individual subject plasma concentration for tasimelteon, actual blood sampling times, and pharmacokinetic parameters will be listed. Plasma concentrations and pharmacokinetic parameters will be summarized using descriptive statistics.
    E.5.2Secondary end point(s)
    Safety Endpoints:

    • Safety and tolerability of a single age-appropriate dose of tasimelteon will be assessed as follows: subjective tolerability from spontaneous reporting of Adverse Events (AEs) and AEs collected through the Pediatric Adverse Event Reporting System (PAERS), changes in clinical laboratory parameters that are relevant to safety and influence of trial medication on vital signs and ECG parameters.

    • The Columbia-Suicide Severity Scale (C-SSRS) will be used in children > 6 years to assess suicidal behavior and ideation.
    E.5.2.1Timepoint(s) of evaluation of this end point
    All AEs recorded during the study will be listed and tabulated by body system and preferred term.


    Descriptive statistics will be provided for background and demographic variables such as age, weight, height, gender, pubertal stage, and race.


    The statistical analyses will be detailed in the Statistical Analysis Plan (SAP).

    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 No
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Pharmacokinetic study in children and adolescents with a new pediatric formulation
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    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 trial1
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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
    France
    Germany
    United Kingdom
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months11
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months1
    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: 24
    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: 12
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 12
    F.1.2Adults (18-64 years) No
    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 No
    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
    CHILDREN incapable of giving consent according Country legislation in force.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 7
    F.4.2.2In the whole clinical trial 24
    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)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-07-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-08-02
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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