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En studie av Soticlestat som tilleggsterapi hos barn og voksne med Dravet-syndrom eller Lennox-Gastaut-syndrom

14. august 2026 oppdatert av: Takeda

En fase 3, prospektiv, åpen etikett, multisite, utvidelse av fase 3-studier for å vurdere den langsiktige sikkerheten og toleransen til Soticlestat som tilleggsterapi hos personer med Dravet-syndrom eller Lennox-Gastaut-syndrom (ENDYMION 2)

Hovedmålet med studien er å finne ut om soticlestat, når det gis som tilleggsbehandling, reduserer antallet anfall hos barn og voksne med Dravet syndrom (DS) eller Lennox-Gastaut syndrom (LGS).

Deltakerne vil motta sin standard anti-anfallsbehandling, pluss tabletter med soticlestat. Det vil være planlagte besøk og oppfølgingstelefoner gjennom hele studiet.

Studieoversikt

Status

Avsluttet

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

Legemidlet som testes i denne studien kalles soticlestat (TAK-935). Soticlestat administrert langtids hos pediatriske og voksne deltakere som deltok i en av de forutgående fase 3 kliniske studiene, TAK-935-3001 [NCT04940624] (deltakere med DS) eller TAK-935-3002 [NCT04938427] (deltakere med LGS) vurderes for ytterligere sikkerhets- og tolerabilitetsdata sammen med effektanalyse.

Studien vil inkludere omtrent 376 deltakere.

Alle deltakere vil motta soticlestat basert på vekten deres i den 2-ukers titreringsperioden. Etter titreringsperioden vil deltakerne fortsette å motta den samme dosen i vedlikeholdsperioden. Ved slutten av vedlikeholdsperioden vil dosen trappes ned (med mindre allerede ved laveste dose) og deretter stoppet. Deltakere som ikke tolererer minimumsdose på 100 mg to ganger daglig (BID), vil bli avbrutt fra studien.

Denne multisenterprøven vil bli gjennomført over hele verden. Den samlede tiden for å delta i studien vil være ca. 4 år, eller til studien stoppes etter sponsorens skjønn, eller produktet er godkjent for markedsføring. Deltakere som avbryter studiemedikamentell behandling før fullføring av studien, vil fortsette å bli fulgt i henhold til protokoll og føre en daglig anfallsdagbok frem til den siste oppfølgingstelefonen

Studietype

Intervensjonell

Registrering (Faktiske)

352

Fase

  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

  • 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
  • Belgia
    • Antwerpen
      • Edegem, Antwerpen, Belgia, 2650
        • UZ Antwerpen
    • Brabant Wallon
      • Ottignies-Louvain-la-Neuve, Brabant Wallon, Belgia, 1340
        • Centre Neurologique William Lennox
    • Brussels Capital
      • Brussels, Brussels Capital, Belgia, 1020
        • Hôpital Universitaire Des Enfants Reine Fabiola
  • 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
  • Canada
    • British Columbia
      • Vancouver, British Columbia, Canada, V5Z 4H4
        • Child and Family Research Institute
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • Hospital for Sick Children
  • Forente stater
    • Arizona
      • Phoenix, Arizona, Forente stater, 85016
        • Phoenix Childrens Hospital
      • Tucson, Arizona, Forente stater, 85718
        • Center For Neurosciences
    • California
      • Los Angeles, California, Forente stater, 90095
        • David Geffen School of Medicine at UCLA
      • San Francisco, California, Forente stater, 94143
        • University of California Benioff Children's Hospital
    • Colorado
      • Denver, Colorado, Forente stater, 80218
        • Colorado Children's Hospital
    • Florida
      • Winter Park, Florida, Forente stater, 32789
        • Pediatric Neurology PA
    • Georgia
      • Atlanta, Georgia, Forente stater, 30328
        • Clinical Integrative Research Center of Atlanta
      • Marietta, Georgia, Forente stater, 30066
        • Sunrise Pediatric Neurology
    • Iowa
      • Iowa City, Iowa, Forente stater, 52242
        • University of Iowa Hospitals & Clinics - (CRS)
    • Maryland
      • Bethesda, Maryland, Forente stater, 20817
        • Midatlantic Epilepsy and Sleep Center
    • Minnesota
      • Saint Paul, Minnesota, Forente stater, 55102
        • Minnesota Epilepsy Group PA
    • New Jersey
      • Livingston, New Jersey, Forente stater, 07039
        • Institute of Neurology and Neurosurgery at Saint Barnabas, LLC
    • New York
      • New York, New York, Forente stater, 10016
        • NYU Comprehensive Epilepsy Center
      • New York, New York, Forente stater, 10003
        • Boston Children's Health Physicians (BCHP)
      • New York, New York, Forente stater, 10016
        • Northwell Health Physician Partners - Neurology at Lenox Hill
    • Ohio
      • Toledo, Ohio, Forente stater, 43614
        • University of Toledo
    • Oregon
      • Portland, Oregon, Forente stater, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, Forente stater, 19104
        • Children's Hospital of Philadelphia
      • Philadelphia, Pennsylvania, Forente stater, 19107
        • Thomas Jefferson University
      • Philadelphia, Pennsylvania, Forente stater, 19134
        • St. Christopher's Hospital for Children
      • York, Pennsylvania, Forente stater, 17403
        • WellSpan Oncology Research
    • South Carolina
      • Charleston, South Carolina, Forente stater, 29425
        • Medical University of South Carolina Children Hospital - PIN
    • Texas
      • Fort Worth, Texas, Forente stater, 76104
        • Cook Children's Medical Center - Jane and John Justin Neurosciences Center
    • Utah
      • Salt Lake City, Utah, Forente stater, 84113
        • University of Utah - Primary Children's Hospital - PPDS
    • Washington
      • Seattle, Washington, Forente stater, 98105
        • Seattle Children's Hospital
      • Tacoma, Washington, Forente stater, 98402
        • MultiCare Institute for Research & Innovation (Tacoma)
  • Frankrike
      • Paris, Frankrike, 75019
        • Hopital Robert Debre
      • Paris, Frankrike, 75015
        • Hopital Necker - Enfants Malades
    • Bouches-du-Rhone
      • Marseille, Bouches-du-Rhone, Frankrike, 13005
        • Hopitaux de La Timone
    • Cote-d'Or
      • Dijon, Cote-d'Or, Frankrike, 21079
        • CHRU Dijon Hopital General
    • Nord
      • Lille, Nord, Frankrike, 59000
        • Hôpital Roger Salengro
  • Hellas
      • Larissa, Hellas, 411 10
        • University General Hospital of Larissa
      • Thessaloniki, Hellas, 546 42
        • Hippokration Hospital
    • Attica
      • Athens, Attica, Hellas, 115 27
        • Childrens' Hospital of Athens 'P. and A. Kyriakou'
      • Chaïdári, Attica, Hellas, 124 62
        • Attikon University General Hospital
  • 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
  • Japan
    • Aiti
      • Nagakute-Shi, Aiti, Japan, 480-1195
        • Aichi Medical University Hospital
    • Hukuoka
      • Fukuoka, Hukuoka, Japan, 813-0017
        • Fukuoka Children's Hospital
    • Kanagawa
      • Yokohama, Kanagawa, Japan, 232-0066
        • Kanagawa Prefectural Hospital Organization Kanagawa Children's Medical Center
    • Kumamoto
      • Kumamoto, Kumamoto, Japan, 862-0947
        • Kumamoto-Ezuko Medical Center for The Severely Disabled
    • Nagasaki
      • Omura-Shi, Nagasaki, Japan, 856-0835
        • National Hospital Organization Nagasaki Medical Center
    • Niigata
      • Niigata, Niigata, Japan, 950-2074
        • National Hospital Organization Nishi-Niigata Chuo National Hospital
    • Okayama-ken
      • Okayama, Okayama-ken, Japan, 700-8558
        • Okayama University Hospital
    • Osaka
      • Neyagawa, Osaka, Japan, 572-0085
        • Yasuhara Childrens Clinic
      • Osaka, Osaka, Japan, 534-0021
        • Osaka City General Hospital
      • Suita-Shi, Osaka, Japan, 565-0871
        • Osaka University Hospital
    • Shizuoka
      • Shizuoka, Shizuoka, Japan, 420-0953
        • National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders
    • Tokyo
      • Chuo-Ku, Tokyo, Japan, 104-0045
        • Hokkaido University Hospital
      • Kodaira-Shi, Tokyo, Japan, 187-0031
        • National Center of Neurology and Psychiatry
  • Kina
    • Beijing Municipality
      • Beijing, Beijing Municipality, Kina, 100034
        • Peking University First Hospital
      • Beijing, Beijing Municipality, Kina, 100045
        • Beijing Children's Hospital,Capital Medical University
    • Chongqing Municipality
      • Chongqing, Chongqing Municipality, Kina, 400014
        • Children's Hospital of Chongqing Medical University
    • Guangdong
      • Guangzhou, Guangdong, Kina, 510623
        • Guangzhou Women and Children's Medical Center
      • Guangzhou, Guangdong, Kina, 510260
        • The Second Affiliated Hospital of Guangzhou Medical Univeristy
      • Shenzhen, Guangdong, Kina, 518026
        • Shenzhen Children's Hospital
    • Hubei
      • Wuhan, Hubei, Kina, 430010
        • Wuhan Childrens hospital
    • Hunan
      • Changsha, Hunan, Kina, 410008
        • Xiangya Hospital of Central South University
    • Jiangxi
      • Nanchang, Jiangxi, Kina, 330006
        • Jiangxi Provincial Children's Hospital
    • Jilin
      • Changchun, Jilin, Kina, 130021
        • The First Hospital of Jilin University
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, Kina, 201102
        • Children's Hospital of Fudan University
      • Shanghai, Shanghai Municipality, Kina, 200040
        • Children's Hospital of Shanghai
  • Latvia
      • Riga, Latvia, LV-1004
        • Childrens University Hospital
  • Mexico
    • Jalisco
      • El Retiro, Jalisco, Mexico, 44280
        • Hospital Civil Fray Antonio Alcalde
  • Nederland
    • North Brabant
      • Heeze, North Brabant, Nederland, 5591 VE
        • Kempenhaeghe - PPDS
    • Overijssel
      • Zwolle, Overijssel, Nederland, 8025 BV
        • Stichting Epilepsie Instellingen Nederland
  • Polen
      • Gdansk, Polen, 80-952
        • Uniwersyteckie Centrum Kliniczne
      • Poznan, Polen, 60-355
        • Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
    • Lesser Poland Voivodeship
      • Krakow, Lesser Poland Voivodeship, Polen, 30-363
        • Centrum Medyczne Plejady
    • Masovian Voivodeship
      • Warsaw, Masovian Voivodeship, Polen, 02-091
        • Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie
  • Russland
      • Krasnoyarsk, Russland, 660022
        • Krasnoyarsk State Medical University n.a. V.F. Voyno-Ysenetskiy
      • Moscow, Russland, 125412
        • Russian National Research Medical University n.a. N.I.Pirogov
      • Tyumen, Russland, 625023
        • Tyumen State Medical Academy
      • Yekaterinburg, Russland, 620144
        • UGMK-Zdorojie, LLC
    • Moscow
      • Moscow, Moscow, Russland, 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
  • Spania
      • Almería, Spania, 04009
        • Hospital Regional Universitario de Malaga Hospital General
      • Barcelona, Spania, 8035
        • Hospital Universitario Vall d'Hebron - PPDS
      • Granada, Spania, 18008
        • Hospital Vithas La Salud
      • Seville, Spania, 41013
        • Centro de Neurologia Avanzada
      • Valencia, Spania, 46026
        • Hospital Universitari i Politecnic La Fe de Valencia
    • Navarre
      • Pamplona, Navarre, Spania, 31008
        • Clinica Universidad Navarra
  • Tyskland
    • Baden-Wurttemberg
      • Freiburg im Breisgau, Baden-Wurttemberg, Tyskland, 79106
        • Alberta Childrens Hospital
    • Hesse
      • Frankfurt am Main, Hesse, Tyskland, 60528
        • Klinikum der Johann-Wolfgang Goethe-Universitat
    • North Rhine-Westphalia
      • Bielefeld, North Rhine-Westphalia, Tyskland, 33617
        • Krankenhaus Mara gGmbH - Epilepsiezentrum Bethel
    • Saxony
      • Radeberg, Saxony, Tyskland, 01454
        • Kleinwachau Sachsisches Epilepsiezentrum Radeberg Gemeinnutzige Gmbh
  • Ukraina
      • Ivano-Frankivsk, Ukraina, 76018
        • Communal Non-commercial Enterprise Iv-Frank Regional Childrens Clinical Hosp of Iv-Frank RC
      • Kyiv, Ukraina, 4080
        • CNPE Clinical Hospital Psychiatry of the Executive Body of the Kyiv City Council KCSA
      • Kyiv, Ukraina, 4209
        • SI Ukr. Med. Rehabilitation Center For Children With Organic Injury of Nervous System of MoH of Ukr
    • Dnipropetrovsk Oblast
      • Dnipro, Dnipropetrovsk Oblast, Ukraina, 49100
        • Municipal Institution Dnipropetrovsk Regional Children Clinical Hospital of DRC
      • Dnipro, Dnipropetrovsk Oblast, Ukraina, 49101
        • Communal Non-profit Enterprise Dnipro City Children Clinical Hospital #5 of DCC
  • Ungarn
      • Budapest, Ungarn, 1145
        • Országos Klinikai Idegtudományi Intézet
      • Budapest, Ungarn, 1023
        • Szent Janos Korhaz es Eszak-budai Egyesitett Korhazak
      • Budapest, Ungarn, 1143
        • Bethesda Gyermekkorhaz
    • Baranya
      • Pécs, Baranya, Ungarn, 7623
        • Pecsi Tudomanyegyetem

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

2 år til 56 år (Barn, Voksen)

Tar imot friske frivillige

Nei

Beskrivelse

Inklusjonskriterier:

1. Deltakeren må ha:

  • Har tidligere vært registrert i soticlestat klinisk studie TAK-935-3001 (NCT04940624) eller TAK-935-3002 (NCT04938427).

Ekskluderingskriterier:

  1. Ustabil, klinisk signifikant nevrologisk (annet enn sykdommen som studeres), psykiatrisk, kardiovaskulær, oftalmologisk, lunge-, lever-, nyre-, metabolsk, gastrointestinal, urologisk, immunologisk, hematopoetisk, endokrin sykdom, malignitet inkludert progressive svulster eller andre abnormiteter som kan påvirke muligheten til å delta i studien eller som potensielt kan forvirre studieresultatene. Det er etterforskerens ansvar å vurdere den kliniske betydningen; konsultasjon med den medisinske monitoren kan imidlertid være berettiget.
  2. Unormal og klinisk signifikant EKG-avvik ved besøk 1 inkludert QT-intervall med Fridericia-korreksjonsmetode (QTcF) >450 millisekunder (ms) bekreftet med et gjentatt EKG ved bruk av manuell måling av QTcF.
  3. Deltakeren anses av etterforskeren å ha overhengende risiko for selvmord eller skade på seg selv, andre eller eiendom. Deltakere som har positive svar på varenummer 4 eller 5 på CSSRS før dosering er ekskludert. Denne skalaen vil kun bli administrert til deltakere i alderen ≥6 år.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: 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.
Legemiddel: Soticlestat
Soticlestat mini-tabletter eller tabletter
Andre navn:
  • TAK-935

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Number of Participants With At Least One Treatment Emergent Adverse Event (TEAE)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Percent Change From Baseline in Total Seizure Frequency Per 28 Days
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Takeda

Etterforskere

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

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Hjelpsomme linker

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

4. mars 2022

Primær fullføring (Faktiske)

24. september 2025

Studiet fullført (Faktiske)

24. september 2025

Datoer for studieregistrering

Først innsendt

15. desember 2021

Først innsendt som oppfylte QC-kriteriene

15. desember 2021

Først lagt ut (Faktiske)

20. desember 2021

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

4. september 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

14. august 2026

Sist bekreftet

1. august 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • TAK-935-3003
  • 2021-002482-17 (EudraCT-nummer)
  • jRCT2051210182 (Registeridentifikator: jRCT)
  • 2022-502802-34-00 (Ctis: EU CTIS)

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

JA

IPD-planbeskrivelse

Takeda gir tilgang til de avidentifiserte individuelle deltakerdataene (IPD) for kvalifiserte studier for å hjelpe kvalifiserte forskere med å adressere legitime vitenskapelige mål (Takedas forpliktelse til å dele data er tilgjengelig på https://clinicaltrials.takeda.com/takedas-commitment?commitment= 5). Disse IPD-ene vil bli levert i et sikkert forskningsmiljø etter godkjenning av en forespørsel om datadeling, og under vilkårene i en datadelingsavtale.

Tilgangskriterier for IPD-deling

IPD fra kvalifiserte studier vil bli delt med kvalifiserte forskere i henhold til kriteriene og prosessen beskrevet på https://vivli.org/ourmember/takeda/. For godkjente forespørsler vil forskerne få tilgang til anonymiserte data (for å respektere pasientens personvern i tråd med gjeldende lover og forskrifter) og med informasjon som er nødvendig for å imøtekomme forskningsmålene i henhold til vilkårene i en datadelingsavtale.

IPD-deling Støtteinformasjonstype

  • STUDY_PROTOCOL
  • SEVJE
  • ICF
  • CSR

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

Abonnere