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    Summary
    EudraCT Number:2018-002485-39
    Sponsor's Protocol Code Number:TAK-935-18-001
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2019-05-07
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2018-002485-39
    A.3Full title of the trial
    A Phase 2, Prospective, Interventional, Open-Label, Multisite, Extension Study To Assess the Long-Term Safety and Tolerability of Soticlestat (TAK-935) as Adjunctive Therapy in Subjects With Developmental Epileptic Encephalopathies Including Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome (ENDYMION 1).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2, Prospective, Interventional, Open-Label, Multisite, Extension Study To Assess the Long-Term Safety and Tolerability of Soticlestat (TAK-935) as Adjunctive Therapy in Subjects With Developmental Epileptic Encephalopathies Including Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome (ENDYMION 1).
    A.4.1Sponsor's protocol code numberTAK-935-18-001
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/491/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorTakeda Development Center Americas, 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 supportTakeda Development Center Americas, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTakeda Development Center Americas, Inc.
    B.5.2Functional name of contact pointMedical Director
    B.5.3 Address:
    B.5.3.1Street Address35/F, New Bund Center, No. 688 Jiyang Road,Pudong district,
    B.5.3.2Town/ cityShanghai
    B.5.3.3Post code200124
    B.5.3.4CountryChina
    B.5.4Telephone number+86 18513725073
    B.5.6E-mailyuan.yao@takeda.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 numberEMA/OD/0000066312
    D.3 Description of the IMP
    D.3.1Product nameSoticlestat
    D.3.2Product code TAK-935
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPGastroenteral use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSoticlestat
    D.3.9.1CAS number 1429505-03-2
    D.3.9.2Current sponsor codeTAK-935
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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.IMP: 2
    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 numberEMA/OD/0000066312
    D.3 Description of the IMP
    D.3.1Product nameSoticlestat
    D.3.2Product code TAK-935
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPGastroenteral use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSoticlestat
    D.3.9.1CAS number 1429505-03-2
    D.3.9.2Current sponsor codeTAK-935
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    Epileptic Encephalopathies: Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome.
    E.1.1.1Medical condition in easily understood language
    Epileptic Encephalopathies: Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome.
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10073682
    E.1.2Term Dravet syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10048816
    E.1.2Term Lennox-Gastaut syndrome
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 22.1
    E.1.2Level PT
    E.1.2Classification code 10083005
    E.1.2Term CDKL5 deficiency disorder
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 23.0
    E.1.2Level LLT
    E.1.2Classification code 10083952
    E.1.2Term Dup15q syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Assess the long-term safety and tolerability of soticlestat when administered as adjunctive therapy to at least one anti-seizure therapy (ie, antiepileptic drugs [AEDs], vagal nerve stimulator, ketogenic diet or modified Atkins diet) in subjects with rare epilepsies.
    E.2.2Secondary objectives of the trial
    The secondary objectives, in subjects receiving soticlestat as adjunctive therapy to at least one anti-seizure therapy, are the following:
    - To assess the effect of soticlestat on seizure frequency.
    - To assess the effect of soticlestat on the Clinical Global Impression of
    Severity (CGI-S) provided by the investigator.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Subjects must have participated in a previous TAK-935 study and meet one of the following conditions:
    • Successfully completed a TAK-935 clinical study.
    • Received at least 10 weeks of treatment (combined Dose Optimization and Maintenance Period) with the study drug in a TAK-935 clinical study and the subjects did not have a serious or severe AE that, in the investigator's or Sponsor's opinion, was related to the study drug and would make it unsafe for the patient to continue receiving the study drug.
    2) In the opinion of the investigator, the subject has the potential to benefit from the administration of TAK935.
    3) The subject provides written informed consent, or the subject's legal representative (parent or legal guardian) provides written informed consent and the subject provides assent, before any study procedures are performed.
    4)The subject and subject's legal representative (ie, parent or guardian [as applicable]) are willing to comply with all study requirements.
    5) From signing of informed consent, throughout the duration of the study, and for 30 days after last dose of study drug, female patients of childbearing potential* who are sexually active with a nonsterilized male partner** must agree to use a highly effective method of contraception (from the list below). In addition, they must not donate ova during this period.
    * Females NOT of childbearing potential are defined as those who are prior to first menarche or who have been surgically sterilized (hysterectomy, bilateral oophorectomy, or tubal ligation) or who are postmenopausal (eg, defined as ≥1 year since last regular menses with a follicle stimulating hormone level >40 IU/L or ≥5 years since last regular menses, confirmed before any study drug is administered).
    **Sterilized males should be ≥1 year postvasectomy and have confirmed that they have obtained documentation of the absence of sperm in the ejaculate.
    A highly effective method of contraception is defined as one that has no higher than a 1% failure rate per year when used consistently and correctly. In this study, the only acceptable methods of contraception are as follows:
    a. Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
    • Oral
    • Intravaginal
    • Transdermal
    b. Progestogen-only hormonal contraception associated with inhibition of ovulation:
    Oral
    • Injectable
    • Implantable
    c. Double-barrier methods (each time the patient has intercourse):
    • Sponge (plus spermicidal cream or jelly) PLUS male condom with or without spermicidal cream or jelly.
    • Cap (plus spermicidal cream or jelly) PLUS male condom with or without spermicidal cream or jelly.
    • Diaphragm (plus spermicidal cream or jelly) PLUS male condom with or without spermicidal cream or jelly.
    d. Intrauterine device (Copper T PLUS condom)
    e. Intrauterine hormone-releasing system
    f. Sterilization:
    • Bilateral tubal occlusion
    • Vasectomized partner (provided that the partner is the sole sexual partner of the patient and the absence of sperm in the ejaculate has been confirmed)
    g. Sexual abstinence, if it is the preferred and usual lifestyle of the patient, will be considered an acceptable method of contraception on a case-by-case basis upon prior approval by the medical monitor. Patients practicing abstinence as a method of contraception must refrain from heterosexual intercourse throughout the duration of the study and for 30 days after last dose of study drug.
    From signing of informed consent, throughout the duration of the study, and for 30 days after last dose of study drug, male patients (post pubertal unless permanently sterilized**) who are sexually active with a female partner of childbearing potential* must agree to use barrier contraception (eg, condom with or without spermicidal cream or jelly).In addition, they must not donate sperm during this period.
    E.4Principal exclusion criteria
    1. Clinically significant disease, that, in the investigator’s opinion, precludes study participation
    2. Enrollment in any other clinical trial involving an investigational drug, device, or treatment in the past 90 days (with the exception of an antecedent study involving TAK-935)
    3. Subject is currently pregnant or breastfeeding or is planning to become pregnant during the study or
    within 30 days of the last study drug administration
    4. Suicide attempt within the last year, at significant risk of suicide (either in the opinion of the investigator or defined as ‘yes’ to suicidal ideation question 4 or 5 on the Columbia- Suicide Severity Rating Scale [C-SSRS] at Screening) or appearing suicidal per investigator judgment
    E.5 End points
    E.5.1Primary end point(s)
    •Incidence of AEs
    •Change from Baseline in behavioral and adaptive functional measures using the Vineland Adaptive
    Behavior Scale (VABS)
    •Change from Baseline in behavior measures using total scores and subscale scores of the Aberrant
    Behavior Checklist-Community Edition (ABC-C) for subjects ≥6 years of age
    •Change from Baseline in the Columbia-Suicide Severity Rating Scale (CSSRS) categorization based onColumbia Classification Algorithm of Suicide Assessment categories 1,2,3,4, and 5 for subjects ≥6 years of age
    ≥6 years of age.
    •Absolute values and change from Baseline in clinical laboratory assessments, vital sign measurements,
    body weight, and ECG parameters
    •Incidence of potentially clinically significant clinical safety laboratory test values, vital signs, weight,
    height/length, and ECG evaluations


    E.5.1.1Timepoint(s) of evaluation of this end point
    Primary end points will be assessed throughout the study.
    E.5.2Secondary end point(s)
    •Percent change from Baseline in all seizure 28-day frequency
    • Percent change from Baseline in drop seizure 28-day frequency (LGS subjects)
    • Percent change from Baseline in convulsive seizure 28-day frequency (DS subjects)
    • Percent change from Baseline in motor seizure 28-day frequency
    • Change from Baseline in CGI-S
    E.5.2.1Timepoint(s) of evaluation of this end point
    At Weeks 12, 24, 36, 48, 64, 80, 104, and 108
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    Australia
    Canada
    China
    Israel
    United States
    Poland
    Spain
    Portugal
    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
    It has been defined as the last subject last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years8
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years8
    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: 165
    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: 83
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 82
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 11
    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
    pediatric population (≥2 years ≤17 years)
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 176
    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)
    Will be provided to subjects for the duration of the study and not afterwards.

    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 Decision2019-08-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-05-15
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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