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    Summary
    EudraCT Number:2021-004393-62
    Sponsor's Protocol Code Number:NBI-921352-DEE2013
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2022-03-15
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2021-004393-62
    A.3Full title of the trial
    A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects with SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)
    Studio di estensione con sostanza attiva, prospettico, a lungo termine, interventistico per valutare la sicurezza e la tollerabilità di NBI-921352 come terapia aggiuntiva in soggetti con sindrome da encefalopatia epilettica e dello sviluppo SCN8A (SCN8A-DEE)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Extension Study to Evaluate how safe and tolerable the drug NBI-921352 is when used as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE).
    Studio di estensione per valutare quando sia sicuro ed efficace il farmaco NBI-921352 quando usato come terapia aggiuntiva in soggetti con sindrome da encefalopatia epilettica e dello sviluppo SCN8A (SCN8A-DEE)
    A.3.2Name or abbreviated title of the trial where available
    NBI-921352-DEE2013
    NBI-921352-DEE2013
    A.4.1Sponsor's protocol code numberNBI-921352-DEE2013
    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 SponsorNeurocrine Biosciences, 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 supportNeurocrine Biosciences, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNeurocrine Biosciences, Inc.
    B.5.2Functional name of contact pointMedical Information Call Center
    B.5.3 Address:
    B.5.3.1Street Address12780 El Camino Real
    B.5.3.2Town/ citySan Diego
    B.5.3.3Post codeCA 92130
    B.5.3.4CountryUnited States
    B.5.4Telephone number0018776413461
    B.5.6E-mailmedinfo@neurocrine.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameNBI-921352
    D.3.2Product code [NBI-921352]
    D.3.4Pharmaceutical form Pillules
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPGastric use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNBI-921352
    D.3.9.2Current sponsor codeNBI-921352
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameNBI-921352
    D.3.2Product code [NBI-921352]
    D.3.4Pharmaceutical form Pillules
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPGastric use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNBI-921352
    D.3.9.2Current sponsor codeNBI-921352
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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: 3
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameNBI-921352
    D.3.2Product code [NBI-921352]
    D.3.4Pharmaceutical form Pillules
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPGastric use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNBI-921352
    D.3.9.2Current sponsor codeNBI-921352
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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: 4
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameNBI-921352
    D.3.2Product code [NBI-921352]
    D.3.4Pharmaceutical form Pillules
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPGastric use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNBI-921352
    D.3.9.2Current sponsor codeNBI-921352
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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: 5
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameNBI-921352
    D.3.2Product code [NBI-921352]
    D.3.4Pharmaceutical form Pillules
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPGastric use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNBI-921352
    D.3.9.2Current sponsor codeNBI-921352
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 placeboPillules
    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
    SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8ADEE)
    Sindrome da encefalopatia epilettica di Sviluppo SCN8A (SCN8ADEE)
    E.1.1.1Medical condition in easily understood language
    Epilepsy
    Epilessia
    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 PT
    E.1.2Classification code 10077380
    E.1.2Term Epileptic encephalopathy
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the long-term safety and tolerability of NBI-921352 when administered for up to 106 weeks.
    Valutare la sicurezza e la tollerabilità a lungo termine di NBI-921352 quando somministrato per un massimo di 106 settimane.
    E.2.2Secondary objectives of the trial
    To investigate the effect of NBI-921352 on long-term seizure control.
    Indagare l’effetto di NBI-921352 sul controllo delle crisi convulsive a lungo termine.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written or oral pediatric assent from the subject and written informed consent from the subject's parent(s) or legal guardian(s) for subjects<18 years of age and for subjects =18 years of age.
    2. Completed 16 weeks of treatment in Study NBI-921352-DEE2012.
    3. Continue to use a nocturnal alerting system or practice consistent with standards of care for the duration of the study. Acceptable nocturnal alerting systems or practices
    4. Have an adequate rescue medication regimen per the investigator's judgment in place for the duration of the study.
    5. Female subjects of childbearing potential must agree to use contraception consistently from Day 1 until the final study visit or 30 days after the last dose of study treatment, whichever is longer.
    6. A male subject of childbearing potential is defined as a subject who has reached spermarche and has not been vasectomized for at least 3 months prior to Day 1. Male subjects must agree to use contraception consistently from Day 1 until 30 days after the last dose of study treatment
    1. Assenso pediatrico orale o scritto del soggetto e consenso informato scritto del/i genitore/i o del/i tutore/i legale/i del soggetto per i soggetti con un’età < 18 anni e per i soggetti con un’età > 18 anni di età.
    2. Completamento di 16 settimane di trattamento nello studio NBI-921352-DEE2012.
    3. Continuare a usare un sistema di allerta notturna o una pratica coerente con gli standard di cura per la durata dello studio. Sistema o pratiche di allerta notturna accettabili.
    4. Avere un adeguato regime farmacologico di soccorso in base al giudizio dello sperimentatore in atto per la durata dello studio.
    5. I soggetti di sesso femminile in età fertile devono accettare di utilizzare un metodo contraccettivo in maniera costante dal Giorno 1 fino alla visita finale dello studio o 30 giorni dopo l’ultima dose del trattamento dello studio, a seconda di quale periodo sia più lungo.
    6. Un soggetto di sesso maschile in età fertile viene definito come un soggetto che ha raggiunto lo spermarca e non si è sottoposto a vasectomia almeno 3 mesi prima del Giorno 1. I soggetti di sesso maschile devono acconsentire a utilizzare metodi contraccettivi in maniera costante a partire dal Giorno 1 fino a 30 giorni dopo l’ultima dose del trattamento dello studio.
    E.4Principal exclusion criteria
    1.Females who are pregnant or currently breastfeeding.
    2.Have developed any other disorder for which the treatment takes priority over treatment ofSCN8A-DEE or is likely to interfere with study treatment or impair treatment compliance.
    3.The subject or subject's parent/caregiver is, in the investigator's opinion, unlikely to comply with the protocol, including the requirement to travel to the study sites for study visits, or is unsuitable for any reason.
    4.Have received any other investigational drug within 30 days or 5 half-lives (if known),whichever is longer, of Day -1 or plan to use an investigational drug (other than the study treatment) during the study.
    1. Soggetti di sesso femminile in stato di gravidanza o in fase di allattamento.
    2. Aver sviluppato qualsiasi altro disturbo per il quale il trattamento è prioritario rispetto al trattamento di SCN8A-DEE o che potrebbe interferire con il trattamento dello studio o compromettere la conformità al trattamento.
    3. Il soggetto o il genitore/tutore del soggetto è, secondo l'opinione dello sperimentatore, improbabile che si attenga al protocollo, ivi compreso l'obbligo di recarsi nei siti di studio per le visite di studio, o è inadatto/a per un qualsiasi motivo.
    4. Aver ricevuto qualsiasi altro farmaco sperimentale entro 30 giorni o 5 emivite (se noti), a seconda di quale periodo sia più lungo, dal giorno -1 o pianificare l'uso di un farmaco sperimentale (diverso dal trattamento dello studio) durante lo studio.
    E.5 End points
    E.5.1Primary end point(s)
    The subject incidence of serious TEAEs, TEAEs leading to discontinuation of study treatment, and fatal TEAEs.
    L’incidenza di eventi avversi durante il trattamento (TEAE) gravi, di TEAE che portano all’interruzione del trattamento dello studio e di TEAE fatali.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Treatment period : Day 1 to Week 106.
    Periodo di trattamento: Dal Giorno 1 alla Settimana 106.
    E.5.2Secondary end point(s)
    Percentage change from baseline in 28-day seizure frequency for countable motorseizures during the Treatment Period of the study at Weeks 5, 30, 54, 78, and 104.
    Variazione percentuale dal basale nella frequenza delle crisi a 28 giorni per le crisi motorie conteggiabili durante il periodo di trattamento dello studio alle Settimane 5, 30, 54, 78 e 104.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Treatment period
    Periodo di trattamento
    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 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) Yes
    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 No
    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 Yes
    E.8.1.7.1Other trial design description
    in aperto
    Open
    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 concerned3
    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 Information not present in EudraCT
    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
    United States
    Belgium
    Czechia
    Denmark
    France
    Germany
    Italy
    Netherlands
    Poland
    Portugal
    Spain
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months0
    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 months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 23
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 7
    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
    The study population may include adult subjects who are not capable of providing consent due to developmental delay and cognitive impairment common in patients with SCN8A-DEE.
    La popolazione in studio può includere soggetti adulti che non sono in grado di fornire il consenso a causa del ritardo dello sviluppo e del deterioramento cognitivo comuni nei pazienti con SCN8A-DEE.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 46
    F.4.2.2In the whole clinical trial 52
    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)
    Normal treatment as discussed with their doctor
    Trattamento normale come discusso con il loro specialista
    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 Decision2022-04-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-04-12
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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