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Un estudio de soticlestat como terapia complementaria en niños y adultos con síndrome de Dravet o síndrome de Lennox-Gastaut

14 de agosto de 2026 actualizado por: Takeda

Una extensión de fase 3, prospectiva, abierta, multisitio, de estudios de fase 3 para evaluar la seguridad y tolerabilidad a largo plazo de soticlestat como terapia adyuvante en sujetos con síndrome de Dravet o síndrome de Lennox-Gastaut (ENDYMION 2)

El objetivo principal del estudio es saber si soticlestat, cuando se administra como terapia complementaria, reduce el número de convulsiones en niños y adultos con síndrome de Dravet (SD) o síndrome de Lennox-Gastaut (SLG).

Los participantes recibirán su terapia anticonvulsiva estándar, además de tabletas de soticlestat. Habrá visitas programadas y llamadas telefónicas de seguimiento durante todo el estudio.

Descripción general del estudio

Estado

Terminado

Condiciones

Intervención / Tratamiento

Descripción detallada

El fármaco que se está probando en este estudio se llama soticlestat (TAK-935). Soticlestat administrado a largo plazo en participantes pediátricos y adultos que participaron en cualquiera de los estudios clínicos de fase 3 antecedentes, TAK-935-3001 [NCT04940624] (participantes con SD) o TAK-935-3002 [NCT04938427] (participantes con LGS) evaluarse para obtener datos adicionales de seguridad y tolerabilidad junto con el análisis de eficacia.

El estudio inscribirá a aproximadamente 376 participantes.

Todos los participantes recibirán soticlestat en función de su peso en el período de titulación de 2 semanas. Después del Período de Titulación, los participantes continuarán recibiendo la misma dosis en el Período de Mantenimiento. Al final del período de mantenimiento, la dosis se reducirá gradualmente (a menos que ya esté en la dosis más baja) y luego se detendrá. Los participantes que no toleren la dosis mínima de 100 mg dos veces al día (BID) serán descontinuados del estudio.

Este ensayo multicéntrico se llevará a cabo en todo el mundo. El tiempo total para participar en el estudio será de aproximadamente 4 años, o hasta que se detenga el estudio a discreción del patrocinador, o hasta que se apruebe la comercialización del producto. Los participantes que interrumpan el tratamiento con el fármaco del estudio antes de la finalización del estudio, seguirán siendo seguidos según el protocolo y mantendrán un diario de convulsiones hasta la última llamada telefónica de seguimiento.

Tipo de estudio

Intervencionista

Inscripción (Actual)

352

Fase

  • Fase 3

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

  • Alemania
    • Baden-Wurttemberg
      • Freiburg im Breisgau, Baden-Wurttemberg, Alemania, 79106
        • Alberta Childrens Hospital
    • Hesse
      • Frankfurt am Main, Hesse, Alemania, 60528
        • Klinikum der Johann-Wolfgang Goethe-Universitat
    • North Rhine-Westphalia
      • Bielefeld, North Rhine-Westphalia, Alemania, 33617
        • Krankenhaus Mara gGmbH - Epilepsiezentrum Bethel
    • Saxony
      • Radeberg, Saxony, Alemania, 01454
        • Kleinwachau Sachsisches Epilepsiezentrum Radeberg Gemeinnutzige Gmbh
  • Australia
    • New South Wales
      • Randwick, New South Wales, Australia, 2031
        • Sydney Children's Hospital
    • Queensland
      • Brisbane, Queensland, Australia, 4101
        • Queensland Childrens Hospital
    • Victoria
      • Heidelberg, Victoria, Australia, 3084
        • Austin Hospital
      • Melbourne, Victoria, Australia, 3004
        • Alfred Hospital
  • Brasil
      • São Paulo, Brasil, 04023-900
        • Universidade Federal de Sao Paulo
      • São Paulo, Brasil, 05403-010
        • Universidade de São Paulo
    • Paraná
      • Curitiba, Paraná, Brasil, 81210-310
        • Instituto de Neurologia de Curitiba (INC)
    • Rio Grande do Sul
      • Porto Alegre, Rio Grande do Sul, Brasil, 90610-000
        • Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
    • São Paulo
      • Campinas, São Paulo, Brasil, 13083-887
        • Hospital das Clinicas - UNICAMP
  • Bélgica
    • Antwerpen
      • Edegem, Antwerpen, Bélgica, 2650
        • UZ Antwerpen
    • Brabant Wallon
      • Ottignies-Louvain-la-Neuve, Brabant Wallon, Bélgica, 1340
        • Centre Neurologique William Lennox
    • Brussels Capital
      • Brussels, Brussels Capital, Bélgica, 1020
        • Hôpital Universitaire Des Enfants Reine Fabiola
  • Canadá
    • British Columbia
      • Vancouver, British Columbia, Canadá, V5Z 4H4
        • Child and Family Research Institute
    • Ontario
      • Toronto, Ontario, Canadá, M5G 1X8
        • Hospital for Sick Children
  • España
      • Almería, España, 04009
        • Hospital Regional Universitario de Malaga Hospital General
      • Barcelona, España, 8035
        • Hospital Universitario Vall d'Hebron - PPDS
      • Granada, España, 18008
        • Hospital Vithas La Salud
      • Seville, España, 41013
        • Centro de Neurologia Avanzada
      • Valencia, España, 46026
        • Hospital Universitari i Politecnic La Fe de Valencia
    • Navarre
      • Pamplona, Navarre, España, 31008
        • Clinica Universidad Navarra
  • Estados Unidos
    • Arizona
      • Phoenix, Arizona, Estados Unidos, 85016
        • Phoenix Childrens Hospital
      • Tucson, Arizona, Estados Unidos, 85718
        • Center For Neurosciences
    • California
      • Los Angeles, California, Estados Unidos, 90095
        • David Geffen School of Medicine at UCLA
      • San Francisco, California, Estados Unidos, 94143
        • University of California Benioff Children's Hospital
    • Colorado
      • Denver, Colorado, Estados Unidos, 80218
        • Colorado Children's Hospital
    • Florida
      • Winter Park, Florida, Estados Unidos, 32789
        • Pediatric Neurology PA
    • Georgia
      • Atlanta, Georgia, Estados Unidos, 30328
        • Clinical Integrative Research Center of Atlanta
      • Marietta, Georgia, Estados Unidos, 30066
        • Sunrise Pediatric Neurology
    • Iowa
      • Iowa City, Iowa, Estados Unidos, 52242
        • University of Iowa Hospitals & Clinics - (CRS)
    • Maryland
      • Bethesda, Maryland, Estados Unidos, 20817
        • Midatlantic Epilepsy and Sleep Center
    • Minnesota
      • Saint Paul, Minnesota, Estados Unidos, 55102
        • Minnesota Epilepsy Group PA
    • New Jersey
      • Livingston, New Jersey, Estados Unidos, 07039
        • Institute of Neurology and Neurosurgery at Saint Barnabas, LLC
    • New York
      • New York, New York, Estados Unidos, 10016
        • NYU Comprehensive Epilepsy Center
      • New York, New York, Estados Unidos, 10003
        • Boston Children's Health Physicians (BCHP)
      • New York, New York, Estados Unidos, 10016
        • Northwell Health Physician Partners - Neurology at Lenox Hill
    • Ohio
      • Toledo, Ohio, Estados Unidos, 43614
        • University of Toledo
    • Oregon
      • Portland, Oregon, Estados Unidos, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, Estados Unidos, 19104
        • Children's Hospital of Philadelphia
      • Philadelphia, Pennsylvania, Estados Unidos, 19107
        • Thomas Jefferson University
      • Philadelphia, Pennsylvania, Estados Unidos, 19134
        • St. Christopher's Hospital for Children
      • York, Pennsylvania, Estados Unidos, 17403
        • WellSpan Oncology Research
    • South Carolina
      • Charleston, South Carolina, Estados Unidos, 29425
        • Medical University of South Carolina Children Hospital - PIN
    • Texas
      • Fort Worth, Texas, Estados Unidos, 76104
        • Cook Children's Medical Center - Jane and John Justin Neurosciences Center
    • Utah
      • Salt Lake City, Utah, Estados Unidos, 84113
        • University of Utah - Primary Children's Hospital - PPDS
    • Washington
      • Seattle, Washington, Estados Unidos, 98105
        • Seattle Children's Hospital
      • Tacoma, Washington, Estados Unidos, 98402
        • MultiCare Institute for Research & Innovation (Tacoma)
  • Francia
      • Paris, Francia, 75019
        • Hopital Robert Debre
      • Paris, Francia, 75015
        • Hopital Necker - Enfants Malades
    • Bouches-du-Rhone
      • Marseille, Bouches-du-Rhone, Francia, 13005
        • Hopitaux de La Timone
    • Cote-d'Or
      • Dijon, Cote-d'Or, Francia, 21079
        • CHRU Dijon Hopital General
    • Nord
      • Lille, Nord, Francia, 59000
        • Hôpital Roger Salengro
  • Grecia
      • Larissa, Grecia, 411 10
        • University General Hospital of Larissa
      • Thessaloniki, Grecia, 546 42
        • Hippokration Hospital
    • Attica
      • Athens, Attica, Grecia, 115 27
        • Childrens' Hospital of Athens 'P. and A. Kyriakou'
      • Chaïdári, Attica, Grecia, 124 62
        • Attikon University General Hospital
  • Hungría
      • Budapest, Hungría, 1145
        • Országos Klinikai Idegtudományi Intézet
      • Budapest, Hungría, 1023
        • Szent Janos Korhaz es Eszak-budai Egyesitett Korhazak
      • Budapest, Hungría, 1143
        • Bethesda Gyermekkorhaz
    • Baranya
      • Pécs, Baranya, Hungría, 7623
        • Pecsi Tudomanyegyetem
  • Italia
    • Lazio
      • Rome, Lazio, Italia, 00185
        • Ospedale Bellaria
      • Rome, Lazio, Italia, 00197
        • IRCCS Ospedale Pediatrico Bambino Gesu - INCIPIT - PIN
      • Rome, Lazio, Italia
        • Fondazione Policlinico Universitario A Gemelli
    • Lombardy
      • Mantua, Lombardy, Italia, 46100
        • ASST di Mantova - Azienda Ospedaliera Carlo Poma
      • Pavia, Lombardy, Italia, 27100
        • ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCS
    • Tuscany
      • Florence, Tuscany, Italia, 50139
        • Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
  • Japón
    • Aiti
      • Nagakute-Shi, Aiti, Japón, 480-1195
        • Aichi Medical University Hospital
    • Hukuoka
      • Fukuoka, Hukuoka, Japón, 813-0017
        • Fukuoka Children's Hospital
    • Kanagawa
      • Yokohama, Kanagawa, Japón, 232-0066
        • Kanagawa Prefectural Hospital Organization Kanagawa Children's Medical Center
    • Kumamoto
      • Kumamoto, Kumamoto, Japón, 862-0947
        • Kumamoto-Ezuko Medical Center for The Severely Disabled
    • Nagasaki
      • Omura-Shi, Nagasaki, Japón, 856-0835
        • National Hospital Organization Nagasaki Medical Center
    • Niigata
      • Niigata, Niigata, Japón, 950-2074
        • National Hospital Organization Nishi-Niigata Chuo National Hospital
    • Okayama-ken
      • Okayama, Okayama-ken, Japón, 700-8558
        • Okayama University Hospital
    • Osaka
      • Neyagawa, Osaka, Japón, 572-0085
        • Yasuhara Childrens Clinic
      • Osaka, Osaka, Japón, 534-0021
        • Osaka City General Hospital
      • Suita-Shi, Osaka, Japón, 565-0871
        • Osaka University Hospital
    • Shizuoka
      • Shizuoka, Shizuoka, Japón, 420-0953
        • National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders
    • Tokyo
      • Chuo-Ku, Tokyo, Japón, 104-0045
        • Hokkaido University Hospital
      • Kodaira-Shi, Tokyo, Japón, 187-0031
        • National Center of Neurology and Psychiatry
  • Letonia
      • Riga, Letonia, LV-1004
        • Childrens University Hospital
  • México
    • Jalisco
      • El Retiro, Jalisco, México, 44280
        • Hospital Civil Fray Antonio Alcalde
  • Países Bajos
    • North Brabant
      • Heeze, North Brabant, Países Bajos, 5591 VE
        • Kempenhaeghe - PPDS
    • Overijssel
      • Zwolle, Overijssel, Países Bajos, 8025 BV
        • Stichting Epilepsie Instellingen Nederland
  • Polonia
      • Gdansk, Polonia, 80-952
        • Uniwersyteckie Centrum Kliniczne
      • Poznan, Polonia, 60-355
        • Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
    • Lesser Poland Voivodeship
      • Krakow, Lesser Poland Voivodeship, Polonia, 30-363
        • Centrum Medyczne Plejady
    • Masovian Voivodeship
      • Warsaw, Masovian Voivodeship, Polonia, 02-091
        • Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie
  • Porcelana
    • Beijing Municipality
      • Beijing, Beijing Municipality, Porcelana, 100034
        • Peking University First Hospital
      • Beijing, Beijing Municipality, Porcelana, 100045
        • Beijing Children's Hospital,Capital Medical University
    • Chongqing Municipality
      • Chongqing, Chongqing Municipality, Porcelana, 400014
        • Children's Hospital of Chongqing Medical University
    • Guangdong
      • Guangzhou, Guangdong, Porcelana, 510623
        • Guangzhou Women and Children's Medical Center
      • Guangzhou, Guangdong, Porcelana, 510260
        • The Second Affiliated Hospital of Guangzhou Medical Univeristy
      • Shenzhen, Guangdong, Porcelana, 518026
        • Shenzhen Children's Hospital
    • Hubei
      • Wuhan, Hubei, Porcelana, 430010
        • Wuhan Childrens hospital
    • Hunan
      • Changsha, Hunan, Porcelana, 410008
        • Xiangya Hospital of Central South University
    • Jiangxi
      • Nanchang, Jiangxi, Porcelana, 330006
        • Jiangxi Provincial Children's Hospital
    • Jilin
      • Changchun, Jilin, Porcelana, 130021
        • The First Hospital of Jilin University
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, Porcelana, 201102
        • Children's Hospital of Fudan University
      • Shanghai, Shanghai Municipality, Porcelana, 200040
        • Children's Hospital of Shanghai
  • Rusia
      • Krasnoyarsk, Rusia, 660022
        • Krasnoyarsk State Medical University n.a. V.F. Voyno-Ysenetskiy
      • Moscow, Rusia, 125412
        • Russian National Research Medical University n.a. N.I.Pirogov
      • Tyumen, Rusia, 625023
        • Tyumen State Medical Academy
      • Yekaterinburg, Rusia, 620144
        • UGMK-Zdorojie, LLC
    • Moscow
      • Moscow, Moscow, Rusia, 117437
        • Russian National Research Medical University n.a. N.I.Pirogov
  • Serbia
      • Belgrade, Serbia, 11000
        • Clinic for Neurology and Psychiatry for Children and Youth
      • Belgrade, Serbia, 11000
        • Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
      • Niš, Serbia, 18 000
        • University Clinical Center Nis
      • Novi Sad, Serbia, 21 000
        • Children and Youth Health Care Institute of Vojvodina
  • Ucrania
      • Ivano-Frankivsk, Ucrania, 76018
        • Communal Non-commercial Enterprise Iv-Frank Regional Childrens Clinical Hosp of Iv-Frank RC
      • Kyiv, Ucrania, 4080
        • CNPE Clinical Hospital Psychiatry of the Executive Body of the Kyiv City Council KCSA
      • Kyiv, Ucrania, 4209
        • SI Ukr. Med. Rehabilitation Center For Children With Organic Injury of Nervous System of MoH of Ukr
    • Dnipropetrovsk Oblast
      • Dnipro, Dnipropetrovsk Oblast, Ucrania, 49100
        • Municipal Institution Dnipropetrovsk Regional Children Clinical Hospital of DRC
      • Dnipro, Dnipropetrovsk Oblast, Ucrania, 49101
        • Communal Non-profit Enterprise Dnipro City Children Clinical Hospital #5 of DCC

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

2 años a 56 años (Niño, Adulto)

Acepta Voluntarios Saludables

No

Descripción

Criterios de inclusión:

1. El participante debe tener:

  • Ha estado previamente inscrito en el estudio clínico soticlestat TAK-935-3001 (NCT04940624) o TAK-935-3002 (NCT04938427).

Criterio de exclusión:

  1. Enfermedad neurológica (que no sea la enfermedad en estudio), psiquiátrica, cardiovascular, oftalmológica, pulmonar, hepática, renal, metabólica, gastrointestinal, urológica, inmunológica, hematopoyética, endocrina, neoplasia maligna, incluidos tumores progresivos, u otra anomalía que pueda afectar la capacidad de participar en el estudio o que puedan potencialmente confundir los resultados del estudio. Es responsabilidad del investigador evaluar la importancia clínica; sin embargo, puede estar justificada la consulta con el monitor médico.
  2. Anomalía anormal y clínicamente significativa en el ECG en la Visita 1, incluido el intervalo QT con el método de corrección de Fridericia (QTcF) >450 milisegundos (ms) confirmado con un ECG repetido utilizando la medición manual de QTcF.
  3. El investigador considera que el participante está en riesgo inminente de suicidio o lesiones a sí mismo, a otros oa la propiedad. Se excluyen los participantes que tienen respuestas positivas en los puntos 4 o 5 del CSSRS antes de la dosificación. Esta escala solo se administrará a participantes de ≥6 años.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: N / A
  • Modelo Intervencionista: Asignación de un solo grupo
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Soticlestat
Participants with DS and LGS received:Participants weighing<45 kilograms(kg):Soticlestat,mini-tablets,titrated from lower dose level (60milligrams[mg]-140mg)to higher dose level(100mg-200mg) twice daily(BID),based on body weight,orally or via enteral feeding tubes including but not limited to nasogastric(NG) tube,gastrostomy tube(G-tube),MIC-KEY button,up to 2 weeks in Titration Period(TP).Participants continued to receive the dose they were on at end of TP,for approximately 4 years in Maintenance Period(MP).Dose was tapered down to lower dose(not less than lowest dose level based on weight) every 3 days until study drug was discontinued(up to 1 week) in Taper Period.Participants weighing≥45kg/adults:Soticlestat, mini-tablets/tablets with starting dose of 200mgBID followed by 300mgBID,up to 2 weeks in TP.Participants continued to receive 300mgBID for approximately 4years in MP.Dose was tapered down to 100mg every 3 days until study drug was discontinued(up to 1 week) in Taper Period.
Droga: Soticlestat
Soticlestat mini-tabletas o tabletas
Otros nombres:
  • TAK-935

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Number of Participants With At Least One Treatment Emergent Adverse Event (TEAE)
Periodo de tiempo: Up to end of study (approximately 3.6 years)
An Adverse Event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE was therefore any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it was considered related to the drug. A TEAE was defined as any AE that started or increased in severity after the first dose of the study drug in this open label extension (OLE) study.
Up to end of study (approximately 3.6 years)
Number of Participants in Each Category of the Columbia-Suicide Severity Rating Scale (C-SSRS) Over Time
Periodo de tiempo: At first visit post-baseline (Visit 1 [V1], Day 1 of the current study), Week 13 (V4), Week 26 (V5), Week 52 (V7), Week 78 (V9), Week104 (v11), Week 130 (V12) and Week 156 (V13)
C-SSRS systematically tracks suicidal ideation and behavior. Responses to questions in the C-SSRS at baseline and each post-baseline time point were collected for participants ≥6 years of age. C-SSRS incidences were categorized as follows: No Suicidal Ideation or Suicidal Behavior or Non-suicidal Self-injurious Behavior (No SI or SB or NSSJB), Non-suicidal Self-injurious Behavior (NSSJB), Suicidal Ideation (SI), and Suicidal Behavior (SB). For each visit (V), the "Yes" answer to the question with the highest severity rank was used to determine the C-SSRS category of a participant. The category Missing indicates the number of participants for whom no data was collected at the particular time point. For each time point only categories with at least 1 participant are presented. BL denotes Baseline and W denotes Week for the reported categories.
At first visit post-baseline (Visit 1 [V1], Day 1 of the current study), Week 13 (V4), Week 26 (V5), Week 52 (V7), Week 78 (V9), Week104 (v11), Week 130 (V12) and Week 156 (V13)
Change From Baseline in Body Weight for All Age Groups
Periodo de tiempo: From Baseline to Week 104
BL denotes Baseline, CFB denotes Change from Baseline and W denotes Week for the reported categories.
From Baseline to Week 104
Change From Baseline in Height for All Age Groups
Periodo de tiempo: Baseline to Week 104
BL denotes Baseline, CFB denotes Change from Baseline and W denotes Week for the reported categories.
Baseline to Week 104
Number of Participants in Each Tanner Stage for Children 6 to 17 Years of Age During the Study
Periodo de tiempo: From Baseline to Week 130
Tanner assessment scores were used to document the stage of development of puberty by assessing the secondary sexual characteristics, rated in 5 stages: Stage 1 (no development) to 5 (adult-like development in quantity and size) by age (6 to 17 years of age) and sex (boys and girls) during the study. Tanner scale boy clinical classification test names for each stage are reported as follows: TANN02-Genitalia and TANN02-Pubic Hair. Tanner scale girl clinical classification test names for each stage are reported as follows: TANN01-Breast and TANN01-Pubic Hair.
From Baseline to Week 130
Absolute Value for Insulin-like Growth Factor 1 (IGF-1) for Children 2 to 17 Years of Age During the Study
Periodo de tiempo: From Baseline to Week 130
Absolute values of IGF1 were summarized descriptively as a continuous variable by age and by sex (male and female). If multiple assessments for a participant at different visits at a particular age were available, then the average of all data for that participant were used for the summary.
From Baseline to Week 130

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Percent Change From Baseline in Total Seizure Frequency Per 28 Days
Periodo de tiempo: From Baseline to Week 156
Seizure frequency per 28 days was defined as total number of seizures reported during the period divided by number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline was defined as (frequency of seizures per 28 days during Treatment Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100.
From Baseline to Week 156
Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days in DS Cohort
Periodo de tiempo: From Baseline to Week 156
Convulsive seizure frequency per 28 days was defined as total number of convulsive seizures reported during the period divided by number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline was defined as (frequency of convulsive seizures per 28 days during Treatment Period - frequency of convulsive seizures per 28 days at Baseline) divided by the frequency of convulsive seizures per 28 days at Baseline multiplied by 100.
From Baseline to Week 156
Percent Change From Baseline in Major Motor Drop (MMD) Seizure Frequency Per 28 Days in LGS Cohort
Periodo de tiempo: From Baseline to Week 144
MMD seizure frequency per 28 days was defined as the total number of MMD seizures reported during the period divided by the number of days during the period seizures were assessed multiplied by 28. Percent change from baseline was defined as (frequency of MMD seizures per 28 days during the Treatment Period - frequency of MMD seizures per 28 days at Baseline) divided by the frequency of MMD seizures per 28 days at Baseline multiplied by 100.
From Baseline to Week 144
Number of Participants With Improvement in the Clinical Global Impression of Improvement (CGI-I) Score
Periodo de tiempo: From Baseline to Week 156
The CGI-I Clinician is a 7-point Likert scale that the investigator uses to rate a participant's change in overall seizure control, behavior, safety and tolerability, after the initiation of study drug relative to Baseline (before treatment with study drug). The participants were rated as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). Reported here is the number of participants with improvement, which includes the CGI-I responses: very much improved, much improved, minimally improved.
From Baseline to Week 156
Number of Participants With Improvement in the Caregiver Global Impression of Improvement (Care GI-I) Score
Periodo de tiempo: From Baseline to Week 156
The Care GI-I is a 7-point Likert scale that the caregiver used to rate a participant's change in overall seizure control, behavior, safety and tolerability after the initiation of study drug relative to Baseline (before treatment with study drug). The participants were rated as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). The parent/caregiver completed the Care GI-I via interview. Reported here is the number of participants with improvement, which includes the Care GI-I responses: very much improved, much improved, minimally improved.
From Baseline to Week 156
Number of Participants With Improvement in the CGI-I Seizure Intensity and Duration Score
Periodo de tiempo: From Baseline to Week 156
The CGI-I seizure intensity and duration instrument was used by the parent/caregiver to rate a participant's change in intensity and duration of convulsive seizures (DS Cohort) or MMD seizures (LGS Cohort) from Baseline. The participant's symptoms were rated on 7-point scale as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). Reported here is the number of participants with improvement, which includes the CGI-I Seizure responses: very much improved, much improved, minimally improved.
From Baseline to Week 156
Number of Participants With Improvement in CGI-I Nonseizure Symptoms Score
Periodo de tiempo: From Baseline to Week 156
The CGI-I nonseizure symptoms instrument is a series of single-item assessments that the investigator used to rate a participant's change in the caregiver-identified symptoms and impacts in select nonseizure domains since initiating the study drug. The participants were rated on 7-point scale by the investigator as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). At Baseline, a symptoms form was completed by the clinician in collaboration with the primary caregiver to assess the participants status based on the presence of any nonseizure symptoms. Reported here is the number of participants with improvement, which includes the CGI-I nonseizure symptoms responses: very much improved, much improved, minimally improved.
From Baseline to Week 156
Change From Baseline in Quality of Life Inventory-Disability (QI-Disability) Score
Periodo de tiempo: From Baseline to Week 156
The QI-Disability tool is a parent/caregiver-reported questionnaire that evaluates quality of life in children with intellectual disabilities. It contains 32 items covering 6 domains of quality of life: physical health, positive emotions, negative emotions, social interaction, leisure and the outdoors, and independence. Items were rated on a 5-point Likert scale and then transformed to a scale of 0 to 100. Possible scores range from 0-100, with higher scores indicating better quality of life. Negative change from baseline indicates worsening of quality of life.
From Baseline to Week 156

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Takeda

Investigadores

  • Director de estudio: Medical Director, Takeda (Note: This product was divested to Mistrau Bio, Inc. in 2026)

Publicaciones y enlaces útiles

La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.

Enlaces Útiles

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

4 de marzo de 2022

Finalización primaria (Actual)

24 de septiembre de 2025

Finalización del estudio (Actual)

24 de septiembre de 2025

Fechas de registro del estudio

Enviado por primera vez

15 de diciembre de 2021

Primero enviado que cumplió con los criterios de control de calidad

15 de diciembre de 2021

Publicado por primera vez (Actual)

20 de diciembre de 2021

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

4 de septiembre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

14 de agosto de 2026

Última verificación

1 de agosto de 2026

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • TAK-935-3003
  • 2021-002482-17 (Número EudraCT)
  • jRCT2051210182 (Identificador de registro: jRCT)
  • 2022-502802-34-00 (Ctis: EU CTIS)

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

SÍ

Descripción del plan IPD

Takeda brinda acceso a los datos de participantes individuales anonimizados (IPD) para estudios elegibles para ayudar a los investigadores calificados a abordar objetivos científicos legítimos (el compromiso de intercambio de datos de Takeda está disponible en https://clinicaltrials.takeda.com/takedas-commitment?commitment= 5). Estos IPD se proporcionarán en un entorno de investigación seguro luego de la aprobación de una solicitud de intercambio de datos y bajo los términos de un acuerdo de intercambio de datos.

Criterios de acceso compartido de IPD

La IPD de los estudios elegibles se compartirá con investigadores calificados de acuerdo con los criterios y el proceso descritos en https://vivli.org/ourmember/takeda/. Para las solicitudes aprobadas, los investigadores tendrán acceso a datos anónimos (para respetar la privacidad del paciente de acuerdo con las leyes y regulaciones aplicables) y con la información necesaria para abordar los objetivos de la investigación según los términos de un acuerdo de intercambio de datos.

Tipo de información de apoyo para compartir IPD

  • PROTOCOLO DE ESTUDIO
  • SAVIA
  • CIF
  • RSC

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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