Une étude sur le soticlestat en tant que traitement complémentaire chez les enfants et les adultes atteints du syndrome de Dravet ou du syndrome de Lennox-Gastaut
14 août 2026 mis à jour par: Takeda
Une phase 3, prospective, ouverte, multisite, extension des études de phase 3 pour évaluer l'innocuité et la tolérabilité à long terme du soticlestat en tant que traitement d'appoint chez les sujets atteints du syndrome de Dravet ou du syndrome de Lennox-Gastaut (ENDYMION 2)
L'objectif principal de l'étude est de savoir si le soticlestat, lorsqu'il est administré en tant que traitement d'appoint, réduit le nombre de crises chez les enfants et les adultes atteints du syndrome de Dravet (DS) ou du syndrome de Lennox-Gastaut (LGS).
Les participants recevront leur traitement anti-épileptique standard, ainsi que des comprimés de soticlestat. Il y aura des visites programmées et des appels téléphoniques de suivi tout au long de l'étude.
Aperçu de l'étude
Statut
Résilié
Les conditions
Intervention / Traitement
Description détaillée
Le médicament testé dans cette étude est appelé soticlestat (TAK-935). Soticlestat administré à long terme chez les participants pédiatriques et adultes qui ont participé à l'une des études cliniques de phase 3 précédentes, TAK-935-3001 [NCT04940624] (participants atteints de SD) ou TAK-935-3002 [NCT04938427] (participants atteints de LGS) être évalué pour des données supplémentaires sur l'innocuité et la tolérabilité ainsi qu'une analyse de l'efficacité.
L'étude recrutera environ 376 participants.
Tous les participants recevront du soticlestat en fonction de leur poids au cours de la période de titration de 2 semaines. Après la période de titration, les participants continueront de recevoir la même dose pendant la période de maintenance. À la fin de la période d'entretien, la dose sera réduite (sauf si elle est déjà à la dose la plus faible) puis arrêtée. Les participants ne tolérant pas la dose minimale de 100 mg deux fois par jour (BID) seront retirés de l'étude.
Cet essai multicentrique sera mené dans le monde entier. La durée totale de participation à l'étude sera d'environ 4 ans, ou jusqu'à ce que l'étude soit arrêtée à la discrétion du promoteur, ou que le produit soit approuvé pour la commercialisation. Les participants qui interrompent le traitement médicamenteux à l'étude avant la fin de l'étude continueront à être suivis conformément au protocole et à tenir un journal quotidien des crises jusqu'au dernier appel téléphonique de suivi.
Type d'étude
Interventionnel
Inscription (Réel)
352
Phase
- Phase 3
Contacts et emplacements
Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.
Lieux d'étude
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Baden-Wurttemberg
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Freiburg im Breisgau, Baden-Wurttemberg, Allemagne, 79106
- Alberta Childrens Hospital
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Hesse
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Frankfurt am Main, Hesse, Allemagne, 60528
- Klinikum der Johann-Wolfgang Goethe-Universitat
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North Rhine-Westphalia
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Bielefeld, North Rhine-Westphalia, Allemagne, 33617
- Krankenhaus Mara gGmbH - Epilepsiezentrum Bethel
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Saxony
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Radeberg, Saxony, Allemagne, 01454
- Kleinwachau Sachsisches Epilepsiezentrum Radeberg Gemeinnutzige Gmbh
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New South Wales
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Randwick, New South Wales, Australie, 2031
- Sydney Children's Hospital
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Queensland
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Brisbane, Queensland, Australie, 4101
- Queensland Childrens Hospital
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Victoria
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Heidelberg, Victoria, Australie, 3084
- Austin Hospital
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Melbourne, Victoria, Australie, 3004
- Alfred Hospital
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Antwerpen
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Edegem, Antwerpen, Belgique, 2650
- UZ Antwerpen
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Brabant Wallon
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Ottignies-Louvain-la-Neuve, Brabant Wallon, Belgique, 1340
- Centre Neurologique William Lennox
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Brussels Capital
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Brussels, Brussels Capital, Belgique, 1020
- Hôpital Universitaire Des Enfants Reine Fabiola
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São Paulo, Brésil, 04023-900
- Universidade Federal de Sao Paulo
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São Paulo, Brésil, 05403-010
- Universidade de São Paulo
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Paraná
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Curitiba, Paraná, Brésil, 81210-310
- Instituto de Neurologia de Curitiba (INC)
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Rio Grande do Sul
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Porto Alegre, Rio Grande do Sul, Brésil, 90610-000
- Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
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São Paulo
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Campinas, São Paulo, Brésil, 13083-887
- Hospital das Clinicas - UNICAMP
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British Columbia
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Vancouver, British Columbia, Canada, V5Z 4H4
- Child and Family Research Institute
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Ontario
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Toronto, Ontario, Canada, M5G 1X8
- Hospital for Sick Children
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Beijing Municipality
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Beijing, Beijing Municipality, Chine, 100034
- Peking University First Hospital
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Beijing, Beijing Municipality, Chine, 100045
- Beijing Children's Hospital,Capital Medical University
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Chongqing Municipality
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Chongqing, Chongqing Municipality, Chine, 400014
- Children's Hospital of Chongqing Medical University
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Guangdong
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Guangzhou, Guangdong, Chine, 510623
- Guangzhou Women and Children's Medical Center
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Guangzhou, Guangdong, Chine, 510260
- The Second Affiliated Hospital of Guangzhou Medical Univeristy
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Shenzhen, Guangdong, Chine, 518026
- Shenzhen Children's Hospital
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Hubei
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Wuhan, Hubei, Chine, 430010
- Wuhan Childrens hospital
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Hunan
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Changsha, Hunan, Chine, 410008
- Xiangya Hospital of Central South University
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Jiangxi
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Nanchang, Jiangxi, Chine, 330006
- Jiangxi Provincial Children's Hospital
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Jilin
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Changchun, Jilin, Chine, 130021
- The First Hospital of Jilin University
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Shanghai Municipality
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Shanghai, Shanghai Municipality, Chine, 201102
- Children's Hospital of Fudan University
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Shanghai, Shanghai Municipality, Chine, 200040
- Children's Hospital of Shanghai
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Almería, Espagne, 04009
- Hospital Regional Universitario de Malaga Hospital General
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Barcelona, Espagne, 8035
- Hospital Universitario Vall d'Hebron - PPDS
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Granada, Espagne, 18008
- Hospital Vithas La Salud
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Seville, Espagne, 41013
- Centro de Neurologia Avanzada
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Valencia, Espagne, 46026
- Hospital Universitari i Politecnic La Fe de Valencia
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Navarre
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Pamplona, Navarre, Espagne, 31008
- Clinica Universidad Navarra
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Paris, France, 75019
- Hopital Robert Debre
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Paris, France, 75015
- Hopital Necker - Enfants Malades
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Bouches-du-Rhone
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Marseille, Bouches-du-Rhone, France, 13005
- Hopitaux de La Timone
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Cote-d'Or
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Dijon, Cote-d'Or, France, 21079
- CHRU Dijon Hopital General
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Nord
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Lille, Nord, France, 59000
- Hôpital Roger Salengro
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Larissa, Grèce, 411 10
- University General Hospital of Larissa
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Thessaloniki, Grèce, 546 42
- Hippokration Hospital
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Attica
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Athens, Attica, Grèce, 115 27
- Childrens' Hospital of Athens 'P. and A. Kyriakou'
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Chaïdári, Attica, Grèce, 124 62
- Attikon University General Hospital
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Budapest, Hongrie, 1145
- Országos Klinikai Idegtudományi Intézet
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Budapest, Hongrie, 1023
- Szent Janos Korhaz es Eszak-budai Egyesitett Korhazak
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Budapest, Hongrie, 1143
- Bethesda Gyermekkorhaz
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Baranya
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Pécs, Baranya, Hongrie, 7623
- Pecsi Tudomanyegyetem
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Lazio
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Rome, Lazio, Italie, 00185
- Ospedale Bellaria
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Rome, Lazio, Italie, 00197
- IRCCS Ospedale Pediatrico Bambino Gesu - INCIPIT - PIN
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Rome, Lazio, Italie
- Fondazione Policlinico Universitario A Gemelli
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Lombardy
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Mantua, Lombardy, Italie, 46100
- ASST di Mantova - Azienda Ospedaliera Carlo Poma
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Pavia, Lombardy, Italie, 27100
- ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCS
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Tuscany
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Florence, Tuscany, Italie, 50139
- Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
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Aiti
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Nagakute-Shi, Aiti, Japon, 480-1195
- Aichi Medical University Hospital
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Hukuoka
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Fukuoka, Hukuoka, Japon, 813-0017
- Fukuoka Children's Hospital
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Kanagawa
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Yokohama, Kanagawa, Japon, 232-0066
- Kanagawa Prefectural Hospital Organization Kanagawa Children's Medical Center
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Kumamoto
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Kumamoto, Kumamoto, Japon, 862-0947
- Kumamoto-Ezuko Medical Center for The Severely Disabled
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Nagasaki
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Omura-Shi, Nagasaki, Japon, 856-0835
- National Hospital Organization Nagasaki Medical Center
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Niigata
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Niigata, Niigata, Japon, 950-2074
- National Hospital Organization Nishi-Niigata Chuo National Hospital
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Okayama-ken
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Okayama, Okayama-ken, Japon, 700-8558
- Okayama University Hospital
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Osaka
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Neyagawa, Osaka, Japon, 572-0085
- Yasuhara Childrens Clinic
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Osaka, Osaka, Japon, 534-0021
- Osaka City General Hospital
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Suita-Shi, Osaka, Japon, 565-0871
- Osaka University Hospital
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Shizuoka
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Shizuoka, Shizuoka, Japon, 420-0953
- National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders
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Tokyo
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Chuo-Ku, Tokyo, Japon, 104-0045
- Hokkaido University Hospital
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Kodaira-Shi, Tokyo, Japon, 187-0031
- National Center of Neurology and Psychiatry
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Riga, Lettonie, LV-1004
- Childrens University Hospital
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Jalisco
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El Retiro, Jalisco, Mexique, 44280
- Hospital Civil Fray Antonio Alcalde
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North Brabant
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Heeze, North Brabant, Pays-Bas, 5591 VE
- Kempenhaeghe - PPDS
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Overijssel
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Zwolle, Overijssel, Pays-Bas, 8025 BV
- Stichting Epilepsie Instellingen Nederland
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Gdansk, Pologne, 80-952
- Uniwersyteckie Centrum Kliniczne
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Poznan, Pologne, 60-355
- Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
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Lesser Poland Voivodeship
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Krakow, Lesser Poland Voivodeship, Pologne, 30-363
- Centrum Medyczne Plejady
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Masovian Voivodeship
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Warsaw, Masovian Voivodeship, Pologne, 02-091
- Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie
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Krasnoyarsk, Russie, 660022
- Krasnoyarsk State Medical University n.a. V.F. Voyno-Ysenetskiy
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Moscow, Russie, 125412
- Russian National Research Medical University n.a. N.I.Pirogov
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Tyumen, Russie, 625023
- Tyumen State Medical Academy
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Yekaterinburg, Russie, 620144
- UGMK-Zdorojie, LLC
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Moscow
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Moscow, Moscow, Russie, 117437
- Russian National Research Medical University n.a. N.I.Pirogov
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Belgrade, Serbie, 11000
- Clinic for Neurology and Psychiatry for Children and Youth
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Belgrade, Serbie, 11000
- Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
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Niš, Serbie, 18 000
- University Clinical Center Nis
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Novi Sad, Serbie, 21 000
- Children and Youth Health Care Institute of Vojvodina
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Ivano-Frankivsk, Ukraine, 76018
- Communal Non-commercial Enterprise Iv-Frank Regional Childrens Clinical Hosp of Iv-Frank RC
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Kyiv, Ukraine, 4080
- CNPE Clinical Hospital Psychiatry of the Executive Body of the Kyiv City Council KCSA
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Kyiv, Ukraine, 4209
- SI Ukr. Med. Rehabilitation Center For Children With Organic Injury of Nervous System of MoH of Ukr
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Dnipropetrovsk Oblast
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Dnipro, Dnipropetrovsk Oblast, Ukraine, 49100
- Municipal Institution Dnipropetrovsk Regional Children Clinical Hospital of DRC
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Dnipro, Dnipropetrovsk Oblast, Ukraine, 49101
- Communal Non-profit Enterprise Dnipro City Children Clinical Hospital #5 of DCC
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Arizona
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Phoenix, Arizona, États-Unis, 85016
- Phoenix Childrens Hospital
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Tucson, Arizona, États-Unis, 85718
- Center For Neurosciences
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California
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Los Angeles, California, États-Unis, 90095
- David Geffen School of Medicine at UCLA
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San Francisco, California, États-Unis, 94143
- University of California Benioff Children's Hospital
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Colorado
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Denver, Colorado, États-Unis, 80218
- Colorado Children's Hospital
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Florida
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Winter Park, Florida, États-Unis, 32789
- Pediatric Neurology PA
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Georgia
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Atlanta, Georgia, États-Unis, 30328
- Clinical Integrative Research Center of Atlanta
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Marietta, Georgia, États-Unis, 30066
- Sunrise Pediatric Neurology
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Iowa
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Iowa City, Iowa, États-Unis, 52242
- University of Iowa Hospitals & Clinics - (CRS)
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Maryland
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Bethesda, Maryland, États-Unis, 20817
- Midatlantic Epilepsy and Sleep Center
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Minnesota
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Saint Paul, Minnesota, États-Unis, 55102
- Minnesota Epilepsy Group PA
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New Jersey
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Livingston, New Jersey, États-Unis, 07039
- Institute of Neurology and Neurosurgery at Saint Barnabas, LLC
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New York
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New York, New York, États-Unis, 10016
- NYU Comprehensive Epilepsy Center
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New York, New York, États-Unis, 10003
- Boston Children's Health Physicians (BCHP)
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New York, New York, États-Unis, 10016
- Northwell Health Physician Partners - Neurology at Lenox Hill
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Ohio
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Toledo, Ohio, États-Unis, 43614
- University of Toledo
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Oregon
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Portland, Oregon, États-Unis, 97239
- Oregon Health and Science University
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Pennsylvania
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Philadelphia, Pennsylvania, États-Unis, 19104
- Children's Hospital of Philadelphia
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Philadelphia, Pennsylvania, États-Unis, 19107
- Thomas Jefferson University
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Philadelphia, Pennsylvania, États-Unis, 19134
- St. Christopher's Hospital for Children
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York, Pennsylvania, États-Unis, 17403
- WellSpan Oncology Research
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South Carolina
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Charleston, South Carolina, États-Unis, 29425
- Medical University of South Carolina Children Hospital - PIN
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Texas
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Fort Worth, Texas, États-Unis, 76104
- Cook Children's Medical Center - Jane and John Justin Neurosciences Center
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Utah
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Salt Lake City, Utah, États-Unis, 84113
- University of Utah - Primary Children's Hospital - PPDS
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Washington
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Seattle, Washington, États-Unis, 98105
- Seattle Children's Hospital
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Tacoma, Washington, États-Unis, 98402
- MultiCare Institute for Research & Innovation (Tacoma)
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Critères de participation
Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.
Critère d'éligibilité
Âges éligibles pour étudier
2 ans à 56 ans (Enfant, Adulte)
Accepte les volontaires sains
Non
La description
Critère d'intégration:
1. Le participant doit avoir :
- A déjà été inscrit dans l'étude clinique soticlestat TAK-935-3001 (NCT04940624) ou TAK-935-3002 (NCT04938427).
Critère d'exclusion:
- Troubles neurologiques instables et cliniquement significatifs (autres que la maladie étudiée), psychiatriques, cardiovasculaires, ophtalmologiques, pulmonaires, hépatiques, rénaux, métaboliques, gastro-intestinaux, urologiques, immunologiques, hématopoïétiques, endocriniens, malins, y compris les tumeurs évolutives, ou autres anomalies pouvant avoir un impact la capacité de participer à l'étude ou qui peuvent potentiellement confondre les résultats de l'étude. Il est de la responsabilité de l'investigateur d'évaluer la signification clinique ; cependant, une consultation avec le moniteur médical peut être justifiée.
- Anomalie ECG anormale et cliniquement significative lors de la visite 1, y compris l'intervalle QT avec la méthode de correction de Fridericia (QTcF) > 450 millisecondes (ms) confirmée par un ECG répété utilisant la mesure manuelle de QTcF.
- Le participant est considéré par l'investigateur comme étant à risque imminent de suicide ou de blessure à soi-même, à autrui ou à des biens. Les participants qui ont des réponses positives aux items 4 ou 5 du CSSRS avant le dosage sont exclus. Cette échelle ne sera administrée qu'aux participants âgés de ≥ 6 ans.
Plan d'étude
Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
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Expérimental: 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.
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Mini-comprimés ou comprimés de Soticlestat
Autres noms:
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Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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Number of Participants With At Least One Treatment Emergent Adverse Event (TEAE)
Délai: Up to end of study (approximately 3.6 years)
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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.
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Up to end of study (approximately 3.6 years)
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Number of Participants in Each Category of the Columbia-Suicide Severity Rating Scale (C-SSRS) Over Time
Délai: 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)
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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.
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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)
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Change From Baseline in Body Weight for All Age Groups
Délai: From Baseline to Week 104
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BL denotes Baseline, CFB denotes Change from Baseline and W denotes Week for the reported categories.
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From Baseline to Week 104
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Change From Baseline in Height for All Age Groups
Délai: Baseline to Week 104
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BL denotes Baseline, CFB denotes Change from Baseline and W denotes Week for the reported categories.
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Baseline to Week 104
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Number of Participants in Each Tanner Stage for Children 6 to 17 Years of Age During the Study
Délai: From Baseline to Week 130
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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.
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From Baseline to Week 130
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Absolute Value for Insulin-like Growth Factor 1 (IGF-1) for Children 2 to 17 Years of Age During the Study
Délai: From Baseline to Week 130
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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.
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From Baseline to Week 130
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Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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Percent Change From Baseline in Total Seizure Frequency Per 28 Days
Délai: From Baseline to Week 156
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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.
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From Baseline to Week 156
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Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days in DS Cohort
Délai: From Baseline to Week 156
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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.
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From Baseline to Week 156
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Percent Change From Baseline in Major Motor Drop (MMD) Seizure Frequency Per 28 Days in LGS Cohort
Délai: From Baseline to Week 144
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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.
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From Baseline to Week 144
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Number of Participants With Improvement in the Clinical Global Impression of Improvement (CGI-I) Score
Délai: From Baseline to Week 156
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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.
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From Baseline to Week 156
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Number of Participants With Improvement in the Caregiver Global Impression of Improvement (Care GI-I) Score
Délai: From Baseline to Week 156
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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.
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From Baseline to Week 156
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Number of Participants With Improvement in the CGI-I Seizure Intensity and Duration Score
Délai: From Baseline to Week 156
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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.
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From Baseline to Week 156
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Number of Participants With Improvement in CGI-I Nonseizure Symptoms Score
Délai: From Baseline to Week 156
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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.
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From Baseline to Week 156
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Change From Baseline in Quality of Life Inventory-Disability (QI-Disability) Score
Délai: From Baseline to Week 156
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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.
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From Baseline to Week 156
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Collaborateurs et enquêteurs
C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.
Parrainer
Les enquêteurs
- Directeur d'études: Medical Director, Takeda (Note: This product was divested to Mistrau Bio, Inc. in 2026)
Publications et liens utiles
La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.
Liens utiles
- Click here for more information about this trial in easy-to-understand language, including a Plain Language Summary of the results if the trial has been completed.
- Click here to ask Takeda's chatbot for comprehensive and easy-to-understand information about clinical trials - even across products and indications - in your local language.
Dates d'enregistrement des études
Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.
Dates principales de l'étude
Début de l'étude (Réel)
4 mars 2022
Achèvement primaire (Réel)
24 septembre 2025
Achèvement de l'étude (Réel)
24 septembre 2025
Dates d'inscription aux études
Première soumission
15 décembre 2021
Première soumission répondant aux critères de contrôle qualité
15 décembre 2021
Première publication (Réel)
20 décembre 2021
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
4 septembre 2026
Dernière mise à jour soumise répondant aux critères de contrôle qualité
14 août 2026
Dernière vérification
1 août 2026
Plus d'information
Termes liés à cette étude
Mots clés
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- TAK-935-3003
- 2021-002482-17 (Numéro EudraCT)
- jRCT2051210182 (Identificateur de registre: jRCT)
- 2022-502802-34-00 (Ctis: EU CTIS)
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
OUI
Description du régime IPD
Takeda donne accès aux données anonymisées des participants individuels (DPI) pour les études éligibles afin d'aider les chercheurs qualifiés à atteindre des objectifs scientifiques légitimes (l'engagement de partage de données de Takeda est disponible sur https://clinicaltrials.takeda.com/takedas-commitment?commitment= 5).
Ces IPD seront fournis dans un environnement de recherche sécurisé après l'approbation d'une demande de partage de données et selon les termes d'un accord de partage de données.
Critères d'accès au partage IPD
Les IPD des études éligibles seront partagées avec des chercheurs qualifiés selon les critères et le processus décrits sur https://vivli.org/ourmember/takeda/.
Pour les demandes approuvées, les chercheurs auront accès à des données anonymisées (afin de respecter la vie privée des patients conformément aux lois et réglementations applicables) et aux informations nécessaires pour répondre aux objectifs de la recherche dans le cadre d'un accord de partage de données.
Type d'informations de prise en charge du partage d'IPD
- PROTOCOLE D'ÉTUDE
- SÈVE
- CIF
- RSE
Informations sur les médicaments et les dispositifs, documents d'étude
Étudie un produit pharmaceutique réglementé par la FDA américaine
Oui
Étudie un produit d'appareil réglementé par la FDA américaine
Non
Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .