Denne side blev automatisk oversat, og nøjagtigheden af ​​oversættelsen er ikke garanteret. Der henvises til engelsk version for en kildetekst.

En undersøgelse af Soticlestat som en add-on terapi hos børn og voksne med Dravet syndrom eller Lennox-Gastaut syndrom

14. august 2026 opdateret af: Takeda

En fase 3, prospektiv, open-label, multisite, udvidelse af fase 3 undersøgelser for at vurdere langsigtet sikkerhed og tolerabilitet af Soticlestat som supplerende terapi hos forsøgspersoner med Dravet syndrom eller Lennox-Gastaut syndrom (ENDYMION 2)

Hovedformålet med undersøgelsen er at finde ud af, om soticlestat, når det gives som en tillægsbehandling, reducerer antallet af anfald hos børn og voksne med Dravet Syndrom (DS) eller Lennox-Gastaut Syndrom (LGS).

Deltagerne vil modtage deres standard anti-anfaldsbehandling plus tabletter af soticlestat. Der vil være planlagte besøg og opfølgende telefonopkald under hele undersøgelsen.

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

Lægemidlet, der testes i denne undersøgelse, kaldes soticlestat (TAK-935). Soticlestat administreret langtids til pædiatriske og voksne deltagere, som deltog i en af ​​de forudgående fase 3 kliniske undersøgelser, TAK-935-3001 [NCT04940624] (deltagere med DS) eller TAK-935-3002 [NCT04938427] (deltagere med LGS) vurderes for yderligere sikkerheds- og tolerabilitetsdata sammen med effektivitetsanalyse.

Undersøgelsen vil omfatte cirka 376 deltagere.

Alle deltagere vil modtage soticlestat baseret på deres vægt i den 2-ugers titreringsperiode. Efter titreringsperioden vil deltagerne fortsat modtage den samme dosis i vedligeholdelsesperioden. Ved slutningen af ​​vedligeholdelsesperioden vil dosis blive nedtrappet (medmindre den allerede er ved den laveste dosis) og derefter stoppet. Deltagere, der ikke tolererer minimumsdosis på 100 mg to gange dagligt (BID), vil blive afbrudt fra undersøgelsen.

Dette multicenterforsøg vil blive gennemført over hele verden. Den samlede tid til at deltage i undersøgelsen vil være cirka 4 år, eller indtil undersøgelsen stoppes efter sponsorens skøn, eller produktet er godkendt til markedsføring. Deltagere, der afbryder studiemedicinsk behandling før afslutningen af ​​undersøgelsen, vil fortsat blive fulgt i henhold til protokol og føre en daglig anfaldsdagbog indtil det sidste opfølgende telefonopkald

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

352

Fase

  • Fase 3

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

  • Australien
    • New South Wales
      • Randwick, New South Wales, Australien, 2031
        • Sydney Children's Hospital
    • Queensland
      • Brisbane, Queensland, Australien, 4101
        • Queensland Childrens Hospital
    • Victoria
      • Heidelberg, Victoria, Australien, 3084
        • Austin Hospital
      • Melbourne, Victoria, Australien, 3004
        • Alfred Hospital
  • Belgien
    • Antwerpen
      • Edegem, Antwerpen, Belgien, 2650
        • UZ Antwerpen
    • Brabant Wallon
      • Ottignies-Louvain-la-Neuve, Brabant Wallon, Belgien, 1340
        • Centre Neurologique William Lennox
    • Brussels Capital
      • Brussels, Brussels Capital, Belgien, 1020
        • Hopital Universitaire des Enfants Reine Fabiola
  • Brasilien
      • São Paulo, Brasilien, 04023-900
        • Universidade Federal de Sao Paulo
      • São Paulo, Brasilien, 05403-010
        • Universidade de Sao Paulo
    • Paraná
      • Curitiba, Paraná, Brasilien, 81210-310
        • Instituto de Neurologia de Curitiba (INC)
    • Rio Grande do Sul
      • Porto Alegre, Rio Grande do Sul, Brasilien, 90610-000
        • Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
    • São Paulo
      • Campinas, São Paulo, Brasilien, 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
  • Forenede Stater
    • Arizona
      • Phoenix, Arizona, Forenede Stater, 85016
        • Phoenix Childrens Hospital
      • Tucson, Arizona, Forenede Stater, 85718
        • Center For Neurosciences
    • California
      • Los Angeles, California, Forenede Stater, 90095
        • David Geffen School of Medicine at UCLA
      • San Francisco, California, Forenede Stater, 94143
        • University of California Benioff Children's Hospital
    • Colorado
      • Denver, Colorado, Forenede Stater, 80218
        • Colorado Children's Hospital
    • Florida
      • Winter Park, Florida, Forenede Stater, 32789
        • Pediatric Neurology PA
    • Georgia
      • Atlanta, Georgia, Forenede Stater, 30328
        • Clinical Integrative Research Center of Atlanta
      • Marietta, Georgia, Forenede Stater, 30066
        • Sunrise Pediatric Neurology
    • Iowa
      • Iowa City, Iowa, Forenede Stater, 52242
        • University of Iowa Hospitals & Clinics - (CRS)
    • Maryland
      • Bethesda, Maryland, Forenede Stater, 20817
        • Midatlantic Epilepsy and Sleep Center
    • Minnesota
      • Saint Paul, Minnesota, Forenede Stater, 55102
        • Minnesota Epilepsy Group PA
    • New Jersey
      • Livingston, New Jersey, Forenede Stater, 07039
        • Institute of Neurology and Neurosurgery at Saint Barnabas, LLC
    • New York
      • New York, New York, Forenede Stater, 10016
        • NYU Comprehensive Epilepsy Center
      • New York, New York, Forenede Stater, 10003
        • Boston Children's Health Physicians (BCHP)
      • New York, New York, Forenede Stater, 10016
        • Northwell Health Physician Partners - Neurology at Lenox Hill
    • Ohio
      • Toledo, Ohio, Forenede Stater, 43614
        • University of Toledo
    • Oregon
      • Portland, Oregon, Forenede Stater, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, Forenede Stater, 19104
        • Children's Hospital of Philadelphia
      • Philadelphia, Pennsylvania, Forenede Stater, 19107
        • Thomas Jefferson University
      • Philadelphia, Pennsylvania, Forenede Stater, 19134
        • St. Christopher's Hospital for Children
      • York, Pennsylvania, Forenede Stater, 17403
        • WellSpan Oncology Research
    • South Carolina
      • Charleston, South Carolina, Forenede Stater, 29425
        • Medical University of South Carolina Children Hospital - PIN
    • Texas
      • Fort Worth, Texas, Forenede Stater, 76104
        • Cook Children's Medical Center - Jane and John Justin Neurosciences Center
    • Utah
      • Salt Lake City, Utah, Forenede Stater, 84113
        • University of Utah - Primary Children's Hospital - PPDS
    • Washington
      • Seattle, Washington, Forenede Stater, 98105
        • Seattle Children's Hospital
      • Tacoma, Washington, Forenede Stater, 98402
        • MultiCare Institute for Research & Innovation (Tacoma)
  • Frankrig
      • Paris, Frankrig, 75019
        • Hôpital Robert Debré
      • Paris, Frankrig, 75015
        • Hopital Necker - Enfants Malades
    • Bouches-du-Rhone
      • Marseille, Bouches-du-Rhone, Frankrig, 13005
        • Hopitaux de La Timone
    • Cote-d'Or
      • Dijon, Cote-d'Or, Frankrig, 21079
        • CHRU Dijon Hopital General
    • Nord
      • Lille, Nord, Frankrig, 59000
        • Hopital Roger Salengro
  • Grækenland
      • Larissa, Grækenland, 411 10
        • University General Hospital of Larissa
      • Thessaloniki, Grækenland, 546 42
        • Hippokration Hospital
    • Attica
      • Athens, Attica, Grækenland, 115 27
        • Childrens' Hospital of Athens 'P. and A. Kyriakou'
      • Chaïdári, Attica, Grækenland, 124 62
        • Attikon University General Hospital
  • Holland
    • North Brabant
      • Heeze, North Brabant, Holland, 5591 VE
        • Kempenhaeghe - PPDS
    • Overijssel
      • Zwolle, Overijssel, Holland, 8025 BV
        • Stichting Epilepsie Instellingen Nederland
  • Italien
    • Lazio
      • Rome, Lazio, Italien, 00185
        • Ospedale Bellaria
      • Rome, Lazio, Italien, 00197
        • IRCCS Ospedale Pediatrico Bambino Gesu - INCIPIT - PIN
      • Rome, Lazio, Italien
        • Fondazione Policlinico Universitario A Gemelli
    • Lombardy
      • Mantua, Lombardy, Italien, 46100
        • ASST di Mantova - Azienda Ospedaliera Carlo Poma
      • Pavia, Lombardy, Italien, 27100
        • ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCS
    • Tuscany
      • Florence, Tuscany, Italien, 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
  • Letland
      • Riga, Letland, LV-1004
        • Childrens University Hospital
  • Mexico
    • Jalisco
      • El Retiro, Jalisco, Mexico, 44280
        • Hospital Civil Fray Antonio Alcalde
  • 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
  • Rusland
      • Krasnoyarsk, Rusland, 660022
        • Krasnoyarsk State Medical University n.a. V.F. Voyno-Ysenetskiy
      • Moscow, Rusland, 125412
        • Russian National Research Medical University n.a. N.I.Pirogov
      • Tyumen, Rusland, 625023
        • Tyumen State Medical Academy
      • Yekaterinburg, Rusland, 620144
        • UGMK-Zdorojie, LLC
    • Moscow
      • Moscow, Moscow, Rusland, 117437
        • Russian National Research Medical University n.a. N.I.Pirogov
  • Serbien
      • Belgrade, Serbien, 11000
        • Clinic for Neurology and Psychiatry for Children and Youth
      • Belgrade, Serbien, 11000
        • Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
      • Niš, Serbien, 18 000
        • University Clinical Center Nis
      • Novi Sad, Serbien, 21 000
        • Children and Youth Health Care Institute of Vojvodina
  • Spanien
      • Almería, Spanien, 04009
        • Hospital Regional Universitario de Malaga Hospital General
      • Barcelona, Spanien, 8035
        • Hospital Universitario Vall d'Hebron - PPDS
      • Granada, Spanien, 18008
        • Hospital Vithas La Salud
      • Seville, Spanien, 41013
        • Centro de Neurologia Avanzada
      • Valencia, Spanien, 46026
        • Hospital Universitari i Politecnic La Fe de Valencia
    • Navarre
      • Pamplona, Navarre, Spanien, 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
  • Ukraine
      • Ivano-Frankivsk, Ukraine, 76018
        • Communal Non-commercial Enterprise Iv-Frank Regional Childrens Clinical Hosp of Iv-Frank RC
      • Kyiv, Ukraine, 4080
        • CNPE Clinical Hospital Psychiatry of the Executive Body of the Kyiv City Council KCSA
      • Kyiv, Ukraine, 4209
        • SI Ukr. Med. Rehabilitation Center For Children With Organic Injury of Nervous System of MoH of Ukr
    • Dnipropetrovsk Oblast
      • Dnipro, Dnipropetrovsk Oblast, Ukraine, 49100
        • Municipal Institution Dnipropetrovsk Regional Children Clinical Hospital of DRC
      • Dnipro, Dnipropetrovsk Oblast, Ukraine, 49101
        • Communal Non-profit Enterprise Dnipro City Children Clinical Hospital #5 of DCC
  • Ungarn
      • Budapest, Ungarn, 1145
        • Orszagos Klinikai Idegtudomanyi Intezet
      • Budapest, Ungarn, 1023
        • Szent Janos Korhaz es Eszak-budai Egyesitett Korhazak
      • Budapest, Ungarn, 1143
        • Bethesda Gyermekkorhaz
    • Baranya
      • Pécs, Baranya, Ungarn, 7623
        • Pecsi Tudomanyegyetem

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

2 år til 56 år (Barn, Voksen)

Tager imod sunde frivillige

Ingen

Beskrivelse

Inklusionskriterier:

1. Deltager skal have:

  • Har tidligere været optaget i soticlestat kliniske undersøgelse TAK-935-3001 (NCT04940624) eller TAK-935-3002 (NCT04938427).

Ekskluderingskriterier:

  1. Ustabil, klinisk signifikant neurologisk (bortset fra den sygdom, der undersøges), psykiatrisk, kardiovaskulær, oftalmologisk, pulmonal, hepatisk, renal, metabolisk, gastrointestinal, urologisk, immunologisk, hæmatopoietisk, endokrin sygdom, malignitet inklusive progressive tumorer eller andre abnormiteter, der kan påvirke evnen til at deltage i undersøgelsen, eller som potentielt kan forvirre undersøgelsesresultaterne. Det er investigatorens ansvar at vurdere den kliniske betydning; dog kan konsultation med den medicinske monitor være berettiget.
  2. Unormal og klinisk signifikant EKG-abnormitet ved besøg 1 inklusive QT-interval med Fridericia-korrektionsmetode (QTcF) >450 millisekunder (ms) bekræftet med et gentaget EKG ved hjælp af manuel måling af QTcF.
  3. Deltageren anses af efterforskeren for at være i overhængende risiko for selvmord eller skade på sig selv, andre eller ejendom. Deltagere, der har positive svar på varenummer 4 eller 5 på CSSRS før dosering, er udelukket. Denne skala vil kun blive administreret til deltagere i alderen ≥6 år.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Interventionel model: Enkelt gruppeopgave
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: 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.
Medicin: Soticlestat
Soticlestat mini-tabletter eller tabletter
Andre navne:
  • TAK-935

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
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
Foranstaltningsbeskrivelse
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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Takeda

Efterforskere

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

Publikationer og nyttige links

Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.

Hjælpsomme links

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

4. marts 2022

Primær færdiggørelse (Faktiske)

24. september 2025

Studieafslutning (Faktiske)

24. september 2025

Datoer for studieregistrering

Først indsendt

15. december 2021

Først indsendt, der opfyldte QC-kriterier

15. december 2021

Først opslået (Faktiske)

20. december 2021

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

4. september 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

14. august 2026

Sidst verificeret

1. august 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

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

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

Takeda giver adgang til de afidentificerede individuelle deltagerdata (IPD) for kvalificerede undersøgelser for at hjælpe kvalificerede forskere med at løse legitime videnskabelige mål (Takedas tilsagn om datadeling er tilgængelig på https://clinicaltrials.takeda.com/takedas-commitment?commitment= 5). Disse IPD'er vil blive leveret i et sikkert forskningsmiljø efter godkendelse af en anmodning om datadeling og under betingelserne i en datadelingsaftale.

IPD-delingsadgangskriterier

IPD fra kvalificerede undersøgelser vil blive delt med kvalificerede forskere i henhold til kriterierne og processen beskrevet på https://vivli.org/ourmember/takeda/. For godkendte anmodninger vil forskerne få adgang til anonymiserede data (for at respektere patientens privatliv i overensstemmelse med gældende love og regler) og med oplysninger, der er nødvendige for at opfylde forskningsmålene i henhold til vilkårene i en datadelingsaftale.

IPD-deling Understøttende informationstype

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Abonner