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    Summary
    EudraCT Number:2021-004393-62
    Sponsor's Protocol Code Number:NBI-921352-DEE2013
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-01-17
    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 ConcernedSpain - AEMPS
    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)
    Estudio prospectivo de extensión con tratamiento activo, a largo plazo y de intervención para evaluar la seguridad y la tolerabilidad de NBI-921352 como tratamiento complementario en sujetos con síndrome de encefalopatía epiléptica y del desarrollo asociado al gen 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).
    Estudio de extensión para evaluar qué tan seguro y tolerable es el fármaco NBI-921352 cuando se usa como terapia complementaria en sujetos con síndrome de encefalopatía epiléptica y del desarrollo SCN8A (SCN8A-DEE).
    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 number+34653249484
    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.3.4Pharmaceutical form Granules
    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.3.8 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 number2.5
    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.3.8 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.3.8 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 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 placeboGranules
    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)
    síndrome de encefalopatía epiléptica y del desarrollo asociada con SCN8A
    (SCN8A-DEE)
    E.1.1.1Medical condition in easily understood language
    Epilepsy
    Epilépsia
    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.
    Evaluar la seguridad y tolerabilidad a largo plazo de NBI-921352 cuando se administra hasta por 106 semanas.
    E.2.2Secondary objectives of the trial
    To investigate the effect of NBI-921352 on long-term seizure control.
    Investigar el efecto de NBI-921352 sobre el control de las convulsiones a largo plazo.
    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. Consentimiento pediátrico escrito u oral del sujeto si se considera capaz de proporcionar asentimiento, y consentimiento informado por escrito de los padres o tutores legales del sujeto para sujetos <18 años de edad y para sujetos ≥18 años de edad que no son capaces de dar su consentimiento de acuerdo con los Comités de Ética Independientes (IEC) / Juntas de Revisión Institucional (IRB) que gobiernan y de acuerdo con las leyes y regulaciones locales. Los sujetos mayores de 18 años y capaces de dar su consentimiento deben firmar un Formulario de consentimiento informado (ICF, por sus siglas en inglés). El consentimiento / asentimiento informado se puede realizar de forma remota, si el sitio lo permite y si existen procedimientos de consentimiento remoto.
    2. Completó 16 semanas de tratamiento (ajuste de dosis de 6 semanas y 10 semanas de mantenimiento) en el Estudio NBI-921352-DEE2012.
    3. Continúe utilizando un sistema de alerta nocturna o practique de acuerdo con los estándares de atención durante la duración del estudio. Los sistemas o prácticas aceptables de alerta nocturna incluyen, entre otros:
    • El padre / cuidador duerme en la misma habitación que el sujeto
    • Monitoreo basado en video y / o acústico (p. Ej., Uso de un monitor para bebés)
    • Dispositivo diseñado como un sistema de alerta de convulsiones (por ejemplo, dispositivos de pulsera)
    4. Tener un régimen de medicación de rescate adecuado según el criterio del investigador durante la duración del estudio.
    5. Las mujeres en edad fértil deben aceptar utilizar métodos anticonceptivos de forma constante desde el día 1 hasta la última visita del estudio o 30 días después de la última dosis del tratamiento del estudio, lo que sea más prolongado.
    Una mujer en edad fértil se define como una mujer capaz de quedar embarazada, que incluye a las mujeres que han tenido su primer ciclo menstrual (es decir, menarca) y no son quirúrgicamente estériles (es decir, ooforectomía bilateral, histerectomía o ligadura de trompas bilateral durante al menos 3 meses antes del día 1).
    Se requieren métodos anticonceptivos altamente efectivos para mujeres en edad fértil:
    • Sistema de liberación de hormonas intrauterinas utilizado con un método anticonceptivo no hormonal eficaz (p. Ej., Anticoncepción de barrera utilizada con espermicida).
    • Dispositivo intrauterino
    • Anticoncepción hormonal combinada (que contiene estrógeno y progestágeno) asociada con la inhibición de la ovulación (que puede ser oral, intravaginal o transdérmica) iniciada y utilizada de acuerdo con las indicaciones médicas durante al menos 3 meses antes del Día 1, utilizada con un método no hormonal eficaz de anticoncepción (por ejemplo, anticoncepción de barrera usada con espermicida).
    • Anticoncepción hormonal de progestágeno solo asociada con la inhibición de la ovulación, que puede iniciarse por vía oral, inyectada o implantada y usarse de acuerdo con las instrucciones médicas durante al menos 3 meses antes del Día 1, usada con un método anticonceptivo no hormonal eficaz (p. Ej., Barrera anticoncepción utilizada con espermicida).
    • Ligadura de trompas bilateral
    • Abstinencia total de las relaciones sexuales (la abstinencia periódica no es aceptable).
    • Compañero (s) sexual (es) que habían sido vasectomizados al menos 3 meses antes del Día 1 con un procedimiento exitoso confirmado médicamente.
    6. Un sujeto masculino en edad fértil se define como un sujeto que ha alcanzado la esperma y no ha sido vasectomizado durante al menos 3 meses antes del Día 1. Los sujetos masculinos deben aceptar usar anticonceptivos de manera constante desde el Día 1 hasta 30 días después de la última dosis. del tratamiento del estudio. El método anticonceptivo aceptable para los sujetos masculinos, incluidos los que se han sometido a una orquiectomía bilateral, es el condón con espermicida (crema, spray, espuma, gel, supositorio o película polimérica).
    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. Mujeres embarazadas o en período de lactancia.
    2. Ha desarrollado cualquier otro trastorno para el cual el tratamiento tiene prioridad sobre el tratamiento de SCN8A-DEE o es probable que interfiera con el tratamiento del estudio o perjudique el cumplimiento del tratamiento.
    3. En opinión del investigador, es poco probable que el sujeto o el padre / cuidador del sujeto cumpla con el protocolo, incluido el requisito de viajar a los lugares del estudio para las visitas del estudio, o no es apto por cualquier motivo.
    4. Haber recibido cualquier otro fármaco en investigación dentro de los 30 días o 5 semividas (si se conocen), lo que sea más largo, del Día -1 o planificar el uso de un fármaco en investigación (que no sea el tratamiento del estudio) durante el estudio.
    E.5 End points
    E.5.1Primary end point(s)
    •The subject incidence of serious TEAEs, TEAEs leading to discontinuation of studytreatment, and fatal TEAEs.
    • La incidencia del sujeto de TEAE graves, TEAE que conducen a la interrupción del tratamiento del estudio y TEAE fatales.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Treatment period : Day 1 to Week 106.
    Período de tratamiento: del día 1 a la semana 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.
    Cambio porcentual desde el inicio en la frecuencia de las convulsiones a los 28 días para las convulsiones motoras contables durante el Período de tratamiento del estudio en las Semanas 5, 30, 54, 78 y 104.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Treatment period
    Periodo de tratamiento
    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 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 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 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
    Belgium
    Canada
    Czechia
    Denmark
    France
    Germany
    Italy
    Netherlands
    Poland
    Portugal
    Spain
    United Kingdom
    United States
    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
    última visita del último paciente
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    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: 23
    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.
    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
    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 Decision2022-03-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-03-11
    P. End of Trial
    P.End of Trial StatusOngoing
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