A Study of Soticlestat as an Add-on Therapy in Children and Adults With Dravet Syndrome or Lennox-Gastaut Syndrome

February 20, 2024 updated by: Takeda

A Phase 3, Prospective, Open-Label, Multisite, Extension of Phase 3 Studies To Assess the Long-Term Safety and Tolerability of Soticlestat as Adjunctive Therapy in Subjects With Dravet Syndrome or Lennox-Gastaut Syndrome (ENDYMION 2)

The main aim of the study is to learn if soticlestat, when given as an add-on therapy, reduces the number of seizures in children and adults with Dravet Syndrome (DS) or Lennox-Gastaut Syndrome (LGS).

Participants will receive their standard anti-seizure therapy, plus tablets of soticlestat. There will be scheduled visits and follow-up phone calls throughout the study.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

The drug being tested in this study is called soticlestat (TAK-935). Soticlestat administered long-term in pediatric and adult participants who participated in an antecedent soticlestat Phase 3 clinical study will be assessed for additional safety and tolerability data along with efficacy analysis, as well as palatability and acceptability of soticlestat in the pediatric population.

The study will enroll approximately 400 participants.

All participants will receive soticlestat based on their weight in the 2-week Titration Period (for participants who roll over from an antecedent double-blind study). Following the Titration Period, participants will continue to receive the same dose in the Maintenance Period. At the end of maintenance period, the dose will be down-tapered (unless already at the lowest dose) and then stopped. Participants not tolerating minimum dose of 100 mg twice a day (BID) will be discontinued from the study.

This multi-center trial will be conducted worldwide. The overall time to participate in the study will be approximately 4 years, or until the study is stopped at the discretion of the sponsor, or the product is approved and launched. Participants who discontinue study drug treatment before the completion of the study, will continue to be followed per protocol and maintain a daily seizure diary until the final follow-up phone call.

Study Type

Interventional

Enrollment (Estimated)

400

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
    • New South Wales
      • Randwick, New South Wales, Australia, 2031
        • Not yet recruiting
        • Sydney Children's Hospital
        • Contact:
        • Principal Investigator:
          • Michael Cardamore
    • Queensland
      • Brisbane, Queensland, Australia, 4101
        • Not yet recruiting
        • Queensland Childrens Hospital
        • Principal Investigator:
          • Catherine Riney
        • Contact:
    • Victoria
      • Heidelberg, Victoria, Australia, 3084
        • Not yet recruiting
        • Austin Hospital
        • Contact:
        • Principal Investigator:
          • Ingrid Scheffer
      • Melbourne, Victoria, Australia, 3004
        • Not yet recruiting
        • Alfred Hospital
        • Contact:
        • Principal Investigator:
          • Terence OBrien
  • Belgium
    • Antwerpen
      • Edegem, Antwerpen, Belgium, 2650
        • Not yet recruiting
        • UZ Antwerpen
        • Contact:
        • Principal Investigator:
          • An-Sofie Schoonjans
    • Brabant Wallon
      • Ottignies-Louvain-la-Neuve, Brabant Wallon, Belgium, 1340
        • Not yet recruiting
        • Centre Neurologique William Lennox
        • Contact:
        • Principal Investigator:
          • Pascal Vrielynck
    • Brussels
      • Brussel, Brussels, Belgium, 1020
        • Not yet recruiting
        • Hôpital Universitaire des Enfants Reine Fabiola
        • Contact:
        • Principal Investigator:
          • Alec Aeby
  • Brazil
      • Sao Paulo, Brazil, 04023-900
        • Not yet recruiting
        • Universidade Federal de Sao Paulo
        • Principal Investigator:
          • Laura Maria Guilhoto
        • Contact:
      • Sao Paulo, Brazil, 05403-010
        • Not yet recruiting
        • Universidade de São Paulo
        • Contact:
        • Principal Investigator:
          • Kette Dualibi Ramos Valente
    • Parana
      • Curitiba, Parana, Brazil, 81210-310
        • Not yet recruiting
        • Instituto de Neurologia de Curitiba (INC)
        • Principal Investigator:
          • Pedro Kowacs
        • Contact:
    • Rio Grande Do Sul
      • Porto Alegre, Rio Grande Do Sul, Brazil, 90610-000
        • Not yet recruiting
        • Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
        • Principal Investigator:
          • Andre Palmini
        • Contact:
    • Sao Paulo
      • Campinas, Sao Paulo, Brazil, 13083-887
        • Not yet recruiting
        • Hospital das Clinicas - UNICAMP
        • Principal Investigator:
          • Fernando Cendes
        • Contact:
  • Canada
    • British Columbia
      • Vancouver, British Columbia, Canada, V5Z 4H4
        • Not yet recruiting
        • Child and Family Research Institute
        • Contact:
        • Principal Investigator:
          • Cyrus Boelman
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • Not yet recruiting
        • Hospital for Sick Children
        • Contact:
        • Principal Investigator:
          • Cecil Hahn
  • China
    • Beijing
      • Beijing, Beijing, China, 100034
        • Recruiting
        • Peking University First Hospital
        • Contact:
        • Principal Investigator:
          • Yuwu Jiang
      • Beijing, Beijing, China, 100045
        • Recruiting
        • Beijing Children's Hospital,Capital Medical University
        • Contact:
        • Principal Investigator:
          • Fang Fang
    • Chongqing
      • Chongqing, Chongqing, China, 400014
        • Recruiting
        • Children's Hospital of Chongqing Medical University
        • Contact:
        • Principal Investigator:
          • Li Jiang
    • Guangdong
      • Guangzhou, Guangdong, China, 510623
        • Recruiting
        • Guangzhou Women and Children's Medical Center
        • Contact:
        • Principal Investigator:
          • Wen-Xiong Chen
      • Guangzhou, Guangdong, China, 510260
        • Recruiting
        • The Second Affiliated Hospital of Guangzhou Medical Univeristy
        • Contact:
        • Principal Investigator:
          • Wei-Ping Liao
      • Shenzhen, Guangdong, China, 518026
        • Recruiting
        • Shenzhen Children's Hospital
        • Contact:
        • Principal Investigator:
          • Jianxiang Liao
    • Hubei
      • Wuhan, Hubei, China, 430010
        • Recruiting
        • Wuhan Childrens hospital
        • Contact:
        • Principal Investigator:
          • Dan Sun
    • Hunan
      • Changsha, Hunan, China, 410008
        • Recruiting
        • Xiangya Hospital of Central South University
        • Contact:
        • Principal Investigator:
          • Jing Peng
    • Jiangxi
      • Nanchang, Jiangxi, China, 330006
        • Recruiting
        • Jiangxi Provincial Children's Hospital
        • Contact:
        • Principal Investigator:
          • Jianmin Zhong
    • Jilin
      • Changchun, Jilin, China, 130021
        • Not yet recruiting
        • the First Hospital of Jilin University
        • Contact:
        • Principal Investigator:
          • Weihong Lin
    • Shanghai
      • Shanghai, Shanghai, China, 201102
        • Not yet recruiting
        • Children's Hospital of Fudan University
        • Contact:
        • Principal Investigator:
          • Shui-Zhen Zhou
      • Shanghai, Shanghai, China, 200040
        • Not yet recruiting
        • Children's Hospital of Shanghai
        • Contact:
        • Principal Investigator:
          • Yucai Chen
  • France
      • Paris, France, 75019
        • Recruiting
        • Hôpital Robert Debré
        • Contact:
        • Principal Investigator:
          • Stephane Auvin
      • Paris, France, 75015
        • Recruiting
        • Hôpital Necker - Enfants malades
        • Principal Investigator:
          • Rima El Nabbout-Tarantino
        • Contact:
    • Bouches-du-Rhone
      • Marseille, Bouches-du-Rhone, France, 13005
        • Not yet recruiting
        • Hopitaux de La Timone
        • Contact:
        • Principal Investigator:
          • Mathieu Milh
    • Cote-d'Or
      • Dijon, Cote-d'Or, France, 21079
        • Not yet recruiting
        • CHRU Dijon Hopital General
        • Principal Investigator:
          • Martine Lemesle-Martin
        • Contact:
    • Nord
      • Lille, Nord, France, 59000
  • Germany
    • Baden-Wurttemberg
      • Freiburg, Baden-Wurttemberg, Germany, 79106
        • Not yet recruiting
        • Alberta Childrens Hospital
        • Principal Investigator:
          • Julia Jacobs-LeVan
        • Contact:
    • Hessen
      • Frankfurt am Main, Hessen, Germany, 60528
        • Not yet recruiting
        • Klinikum der Johann-Wolfgang Goethe-Universitat
        • Principal Investigator:
          • Felix Rosenow
        • Contact:
    • Nordrhein-Westfalen
      • Bielefeld, Nordrhein-Westfalen, Germany, 33617
        • Not yet recruiting
        • Krankenhaus Mara gGmbH - Epilepsiezentrum Bethel
        • Principal Investigator:
          • Christian Brandt
        • Contact:
    • Sachsen
      • Radeberg, Sachsen, Germany, 01454
        • Not yet recruiting
        • Kleinwachau Sachsisches Epilepsiezentrum Radeberg Gemeinnutzige Gmbh
        • Principal Investigator:
          • Thomas Mayer
        • Contact:
  • Greece
      • Larisa, Greece, 411 10
        • Not yet recruiting
        • University General Hospital of Larissa
        • Principal Investigator:
          • Efthimios Dardiotis
        • Contact:
      • Thessaloniki, Greece, 546 42
        • Not yet recruiting
        • Hippokration Hospital
        • Principal Investigator:
          • Dimitrios Zafeiriou
        • Contact:
    • Attiki
      • Athens, Attiki, Greece, 115 27
        • Not yet recruiting
        • Childrens' Hospital of Athens 'P. and A. Kyriakou'
        • Contact:
        • Principal Investigator:
          • Georgios Vartzelis
      • Chaidari, Attiki, Greece, 124 62
        • Recruiting
        • Attikon University General Hospital
        • Contact:
        • Principal Investigator:
          • Argirios Dinopoulos
  • Hungary
      • Budapest, Hungary, 1023
        • Not yet recruiting
        • Szent Janos Korhaz es Eszak-Budai Egyesitett Korhazak
        • Principal Investigator:
          • Anna Altmann
        • Contact:
      • Budapest, Hungary, 1143
        • Recruiting
        • Bethesda Gyermekkórház
        • Contact:
        • Principal Investigator:
          • Andras Fogarasi
      • Budapest, Hungary, 1145
        • Recruiting
        • Országos Klinikai Idegtudományi Intézet
        • Contact:
        • Principal Investigator:
          • Daniel Fabo
    • Baranya
      • Pecs, Baranya, Hungary, 7623
        • Recruiting
        • Pecsi Tudomanyegyetem
        • Contact:
        • Principal Investigator:
          • Katalin Hollody
  • Italy
    • Lazio
      • Roma, Lazio, Italy, 00185
        • Not yet recruiting
        • Ospedale Bellaria
        • Principal Investigator:
          • Francesca Bisulli
        • Contact:
      • Roma, Lazio, Italy, 00197
        • Not yet recruiting
        • IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN
        • Principal Investigator:
          • Nicola Specchio
        • Contact:
      • Roma, Lazio, Italy
    • Lombardia
      • Mantova, Lombardia, Italy, 46100
        • Not yet recruiting
        • ASST di Mantova - Azienda Ospedaliera Carlo Poma
        • Contact:
        • Principal Investigator:
          • Francesca Beccaria
      • Pavia, Lombardia, Italy, 27100
        • Not yet recruiting
        • ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCS
        • Contact:
        • Principal Investigator:
          • Valentina De Giorgis
    • Toscana
      • Firenze, Toscana, Italy, 50139
        • Recruiting
        • Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
        • Principal Investigator:
          • Renzo Guerrini
        • Contact:
  • Japan
    • Aiti
      • Nagakute-Shi, Aiti, Japan, 480-1195
    • Hukuoka
      • Fukuoka-Shi, Hukuoka, Japan, 813-0017
        • Recruiting
        • Fukuoka Children's Hospital
        • Contact:
        • Principal Investigator:
          • Ryutaro Kira
    • Kanagawa
      • Yokohama-Shi, Kanagawa, Japan, 232-0066
        • Not yet recruiting
        • Kanagawa Prefectural Hospital Organization Kanagawa Children's Medical Center
        • Principal Investigator:
          • Tomohide Goto
        • Contact:
    • Kumamoto
      • Kumamoto-Shi, Kumamoto, Japan, 862-0947
        • Recruiting
        • Kumamoto-Ezuko Medical Center for The Severely Disabled
        • Contact:
        • Principal Investigator:
          • Takateru Ishitsu
    • Nagasaki
      • Omura-Shi, Nagasaki, Japan, 856-0835
        • Recruiting
        • National Hospital Organization Nagasaki Medical Center
        • Contact:
        • Principal Investigator:
          • Ryoko Honda
    • Niigata
      • Niigata-Shi, Niigata, Japan, 950-2074
        • Recruiting
        • National Hospital Organization Nishi-Niigata Chuo National Hospital
        • Contact:
        • Principal Investigator:
          • Jun Tohyama
    • Okayama
      • Okayama-city, Okayama, Japan, 700-8558
        • Not yet recruiting
        • Okayama University Hospital
        • Contact:
        • Principal Investigator:
          • Katsuhiro Kobayashi
    • Osaka
      • Neyagawa-Shi, Osaka, Japan, 572-0085
        • Recruiting
        • Yasuhara Childrens Clinic
        • Contact:
        • Principal Investigator:
          • Akihiro Yasuhara
      • Osaka-Shi, Osaka, Japan, 534-0021
        • Recruiting
        • Osaka City General Hospital
        • Contact:
        • Principal Investigator:
          • Shin Okazaki
      • Suita-Shi, Osaka, Japan, 565-0871
        • Recruiting
        • Osaka University Hospital
        • Contact:
        • Principal Investigator:
          • Shin Nabatame
    • Sizuoka
      • Shizuoka-Shi, Sizuoka, Japan, 420-0953
        • Recruiting
        • National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders
        • Contact:
        • Principal Investigator:
          • Tokito Yamaguchi
    • Tokyo
      • Chuo-Ku, Tokyo, Japan, 104-0045
        • Not yet recruiting
        • Hokkaido University Hospital
        • Principal Investigator:
          • Hideaki Shiraishi
        • Contact:
      • Kodaira-Shi, Tokyo, Japan, 187-0031
        • Recruiting
        • National Center of Neurology and Psychiatry
        • Contact:
        • Principal Investigator:
          • Eiji Nakagawa
  • Latvia
      • Riga, Latvia, LV-1004
        • Recruiting
        • Childrens University Hospital
        • Contact:
        • Principal Investigator:
          • Guntis Rozentals
  • Mexico
    • Jalisco
      • El Retiro, Jalisco, Mexico, 44280
        • Not yet recruiting
        • Hospital Civil Fray Antonio Alcalde
        • Principal Investigator:
          • Hugo Ceja Moreno
        • Contact:
  • Netherlands
    • Noord-Brabant
      • Heeze, Noord-Brabant, Netherlands, 5591 VE
        • Recruiting
        • Kempenhaeghe - PPDS
        • Contact:
        • Principal Investigator:
          • Marian Majoie
    • Overijssel
      • Zwolle, Overijssel, Netherlands, 8025 BV
        • Recruiting
        • Stichting Epilepsie Instellingen Nederland
        • Contact:
        • Principal Investigator:
          • Boudewijn Gunning
  • Poland
      • Gdansk, Poland, 80-952
        • Not yet recruiting
        • Uniwersyteckie Centrum Kliniczne
        • Principal Investigator:
          • Maria Mazurkiewicz-Beldzinska
        • Contact:
      • Poznan, Poland, 60-355
        • Not yet recruiting
        • Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
        • Contact:
        • Principal Investigator:
          • Barbara Steinborn
    • Malopolskie
      • Krakow, Malopolskie, Poland, 30-363
        • Recruiting
        • Centrum Medyczne Plejady
        • Contact:
        • Principal Investigator:
          • Marta Zolnowska
    • Mazowieckie
      • Warszawa, Mazowieckie, Poland, 02-091
        • Not yet recruiting
        • Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie
        • Contact:
        • Principal Investigator:
          • Sergiusz Jozwiak
  • Russian Federation
      • Ekaterinburg, Russian Federation, 620144
        • Not yet recruiting
        • UGMK-Zdorojie, LLC
        • Contact:
        • Principal Investigator:
          • Tatiana Tomenko
      • Krasnoyarsk, Russian Federation, 660022
        • Not yet recruiting
        • Krasnoyarsk State Medical University n.a. V.F. Voyno-Ysenetskiy
        • Contact:
        • Principal Investigator:
          • Diana Dmitrenko
      • Moscow, Russian Federation, 125412
        • Not yet recruiting
        • Russian National Research Medical University n.a. N.I.Pirogov
        • Contact:
        • Principal Investigator:
          • Elena Belousova
      • Tyumen', Russian Federation, 625023
        • Not yet recruiting
        • Tyumen State Medical Academy
        • Contact:
        • Principal Investigator:
          • Olga Rakhmanina
    • Moskva
      • Moscow, Moskva, Russian Federation, 117437
        • Not yet recruiting
        • Russian National Research Medical University n.a. N.I.Pirogov
        • Contact:
        • Principal Investigator:
          • Nikolay Zavadenko
  • Serbia
      • Belgrade, Serbia, 11000
        • Recruiting
        • Clinic for Neurology and Psychiatry for Children and Youth
        • Contact:
        • Principal Investigator:
          • Milan Borkovic
      • Belgrade, Serbia, 11000
        • Recruiting
        • Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
        • Principal Investigator:
          • Ruzica Kravljanac
        • Contact:
      • Nis, Serbia, 18 000
        • Recruiting
        • University Clinical Center Nis
        • Contact:
        • Principal Investigator:
          • Tatjana Tosic
      • Novi Sad, Serbia, 21 000
        • Recruiting
        • Children and Youth Health Care Institute of Vojvodina
        • Contact:
        • Principal Investigator:
          • Marija Knezevic-Pogancev
  • Spain
      • Almeria, Spain, 04009
        • Not yet recruiting
        • Hospital Regional Universitario de Malaga Hospital General
        • Principal Investigator:
          • Pedro Serrano
        • Contact:
      • Barcelona, Spain, 8035
        • Recruiting
        • Hospital Universitario Vall d'Hebron - PPDS
        • Contact:
        • Principal Investigator:
          • Manuel Toledo
      • Granada, Spain, 18008
        • Not yet recruiting
        • Hospital Vithas La Salud
        • Contact:
        • Principal Investigator:
          • Juan Sanchez Alvarez
      • Sevilla, Spain, 41013
        • Not yet recruiting
        • Centro de Neurologia Avanzada
        • Principal Investigator:
          • Juan Jesus Rodriguez Uranga
        • Contact:
      • Valencia, Spain, 46026
        • Recruiting
        • Hospital Universitari i Politecnic La Fe de Valencia
        • Contact:
        • Principal Investigator:
          • Vicente Villanueva
    • Navarra
      • Pamplona, Navarra, Spain, 31008
        • Recruiting
        • Clinica Universidad Navarra
        • Principal Investigator:
          • Rocio Sanchez-Carpintero
        • Contact:
  • Ukraine
      • Ivano-Frankivsk, Ukraine, 76018
        • Not yet recruiting
        • Communal Non-commercial Enterprise Iv-Frank Regional Childrens Clinical Hosp of Iv-Frank RC
        • Contact:
        • Principal Investigator:
          • Dmytro Delva
      • Kyiv, Ukraine, 4080
        • Not yet recruiting
        • CNPE Clinical Hospital Psychiatry of the Executive Body of the Kyiv City Council KCSA
        • Contact:
        • Principal Investigator:
          • Volodymyr Kharytonov
      • Kyiv, Ukraine, 4209
        • Not yet recruiting
        • SI Ukr. Med. Rehabilitation Center For Children With Organic Injury of Nervous System of MoH of Ukr
        • Contact:
        • Principal Investigator:
          • Volodymyr Martyniuk
    • Dnipropetrovs'ka Oblast
      • Dnipro, Dnipropetrovs'ka Oblast, Ukraine, 49100
        • Not yet recruiting
        • Municipal Institution Dnipropetrovsk Regional Children Clinical Hospital of DRC
        • Contact:
        • Principal Investigator:
          • Alla Kyrychenko
      • Dnipro, Dnipropetrovs'ka Oblast, Ukraine, 49101
        • Not yet recruiting
        • Communal Non-profit Enterprise Dnipro City Children Clinical Hospital #5 of DCC
        • Contact:
        • Principal Investigator:
          • Iryna Makedonska
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Not yet recruiting
        • Phoenix Childrens Hospital
        • Contact:
        • Principal Investigator:
          • Angus Wilfong
      • Tucson, Arizona, United States, 85718
        • Not yet recruiting
        • Center for Neurosciences
        • Principal Investigator:
          • Dinesh Talwar
        • Contact:
    • California
      • Los Angeles, California, United States, 90095
        • Not yet recruiting
        • David Geffen School of Medicine at UCLA
        • Contact:
        • Principal Investigator:
          • Shaun Hussain
      • San Francisco, California, United States, 94143
        • Recruiting
        • University of California Benioff Children's Hospital
        • Contact:
        • Principal Investigator:
          • Joseph Sullivan
    • Colorado
      • Denver, Colorado, United States, 80218
        • Not yet recruiting
        • Colorado Children's Hospital
        • Contact:
        • Principal Investigator:
          • Scott Demarest
    • Florida
      • Winter Park, Florida, United States, 32789
        • Not yet recruiting
        • Pediatric Neurology PA
        • Contact:
        • Principal Investigator:
          • Ronald Davis
    • Georgia
      • Atlanta, Georgia, United States, 30328
        • Recruiting
        • Clinical Integrative Research Center of Atlanta
        • Contact:
        • Principal Investigator:
          • Julio Flamini
      • Marietta, Georgia, United States, 30066
        • Not yet recruiting
        • Sunrise Pediatric Neurology
        • Contact:
        • Principal Investigator:
          • Cherise Frazier
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • University of Iowa Hospitals & Clinics - (CRS)
        • Contact:
        • Principal Investigator:
          • Michael Ciliberto
    • Maryland
      • Bethesda, Maryland, United States, 20817
        • Recruiting
        • Midatlantic Epilepsy and Sleep Center
        • Contact:
        • Principal Investigator:
          • Pavel Klein
    • Minnesota
      • Saint Paul, Minnesota, United States, 55102
        • Not yet recruiting
        • Minnesota Epilepsy Group PA
        • Contact:
        • Principal Investigator:
          • Nitin Agarwal
    • New Jersey
      • Livingston, New Jersey, United States, 07039
        • Recruiting
        • Institute of Neurology and Neurosurgery at Saint Barnabas, LLC
        • Contact:
        • Principal Investigator:
          • Rina Goldberg
    • New York
      • New York, New York, United States, 10016
      • New York, New York, United States, 10003
        • Not yet recruiting
        • Boston Children's Health Physicians (BCHP)
        • Contact:
        • Principal Investigator:
          • Steven Wolf
      • New York, New York, United States, 10016
        • Not yet recruiting
        • Northwell Health Physician Partners - Neurology at Lenox Hill
        • Contact:
        • Principal Investigator:
          • Ruben Kuzniecky
    • Ohio
      • Toledo, Ohio, United States, 43614
        • Not yet recruiting
        • University of Toledo
        • Principal Investigator:
          • Imran Ali
        • Contact:
    • Oregon
      • Portland, Oregon, United States, 97239
        • Not yet recruiting
        • Oregon Health and Science University
        • Contact:
        • Principal Investigator:
          • Colin Roberts
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
        • Not yet recruiting
        • Thomas Jefferson University
        • Contact:
        • Principal Investigator:
          • Michael Sperling
      • Philadelphia, Pennsylvania, United States, 19104
        • Not yet recruiting
        • Children's Hospital of Philadelphia
        • Contact:
        • Principal Investigator:
          • Eric Marsh
      • Philadelphia, Pennsylvania, United States, 19134
        • Not yet recruiting
        • St. Christopher's Hospital for Children
        • Contact:
        • Principal Investigator:
          • Ignacio Valencia
      • York, Pennsylvania, United States, 17403
        • Recruiting
        • WellSpan Oncology Research
        • Contact:
        • Principal Investigator:
          • Todd Barron
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Recruiting
        • Medical University of South Carolina Children Hospital - PIN
        • Contact:
        • Principal Investigator:
          • Jonathan Halford
    • Texas
      • Fort Worth, Texas, United States, 76104
        • Recruiting
        • Cook Children's Medical Center - Jane and John Justin Neurosciences Center
        • Contact:
        • Principal Investigator:
          • Michael Perry
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Not yet recruiting
        • University of Utah - Primary Children's Hospital - PPDS
        • Contact:
        • Principal Investigator:
          • Matthew Sweney
    • Washington
      • Seattle, Washington, United States, 98105
        • Not yet recruiting
        • Seattle Children's Hospital
        • Contact:
        • Principal Investigator:
          • Russell Saneto
      • Tacoma, Washington, United States, 98402
        • Not yet recruiting
        • MultiCare Institute for Research & Innovation (Tacoma)
        • Principal Investigator:
          • Steven Phillips
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 56 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

1. Participant must have:

  • Been previously enrolled in a phase 3 soticlestat clinical study.

Exclusion Criteria:

  1. Unstable, clinically significant neurologic (other than the disease being studied), psychiatric, cardiovascular, ophthalmologic, pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, endocrine disease, malignancy including progressive tumors, or other abnormality that may impact the ability to participate in the study or that may potentially confound the study results. It is the responsibility of the investigator to assess the clinical significance; however, consultation with the medical monitor may be warranted.
  2. Abnormal and clinically significant ECG abnormality at Visit 1 including QT interval with Fridericia correction method (QTcF) >450 milliseconds (ms) confirmed with a repeat ECG using manual measurement of QTcF.
  3. Participant is considered by the investigator to be at imminent risk of suicide or injury to self, others, or property. Participants who have positive answers on item numbers 4 or 5 on the CSSRS before dosing are excluded. This scale will only be administered to participants aged ≥6 years.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Soticlestat
Participants with DS and LGS will receive:Participants weighing <45kg:Soticlestat,mini-tablets,titrated from lower dose level(60mg to 140mg) to higher dose(100mg to 200mg) twice daily(BID),based on body weight,orally/via enteral feeding tubes including but not limited to nasogastric(NG)-tube,gastrostomy tube(G-tube),MIC-KEY button,upto 2 weeks in Titration Period. Will continue to receive dose they are on at end of Titration Period,for approximately 4 years in Maintenance Period.Dose will be tapered down to lower dose(not less than lowest dose level based on weight)every 3 days until study drug is discontinued(upto 1week) in Taper Period.Participants weighing ≥45kg/adults:Soticlestat mini-tablets/tablets with starting dose of 200mg BID followed by 300mg BID,up to 2 weeks in Titration Period.Will continue to receive 300mg BID for approximately 4 years in Maintenance Period.Dose will be tapered down upto 100mg every 3 days until study drug is discontinued(up to 1 week) in Taper Period.
Drug: Soticlestat
Soticlestat mini-tablets or tablets
Other Names:
  • TAK-935

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With At Least One Treatment Emergent Adverse Event (TEAE)
Time Frame: Up to 4 years
An Adverse Event (AE) is 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 can therefore be 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 is considered related to the drug. A TEAE is any AE either reported for the first time or worsening of a pre-existing event after first dose of study drug and within 30 days of the last administration of study drug.
Up to 4 years
Change from Baseline in Body Weight for All Age Groups
Time Frame: Up to 4 years
Up to 4 years
Change from Baseline in Height for All Age Groups
Time Frame: Up to 4 years
Up to 4 years
Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS) Score
Time Frame: Up to 4 years
C-SSRS systematically tracks suicidal ideation and behavior. The total score range is 0 (no ideation is present) to 5 (active suicidal ideation with specific plan and intent). The higher the score, the greater one's suicidal ideation.
Up to 4 years
Absolute Value for Tanner Stage for Children 6 to 17 Years of Age During the Study
Time Frame: Up to 4 years
Tanner assessment score is 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).
Up to 4 years
Absolute Value for Insulin-like Growth Factor 1 (IGF-1) for Children 2 to 17 Years of Age During the Study
Time Frame: Up to 4 years
Up to 4 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percent Change from Baseline in Total Seizure Frequency per 28 Days for DS and LGS Participants
Time Frame: Up to 4 years
Seizure frequency per 28 days is 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 is 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.
Up to 4 years
Percent Change from Baseline in Convulsive Seizure Frequency per 28 Days in DS Cohort
Time Frame: Up to 4 years
Convulsive seizure frequency per 28 days is 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 will be 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.
Up to 4 years
Percent Change from Baseline in Major Motor Drop (MMD) Seizure Frequency per 28 Days in LGS Cohort
Time Frame: Up to 4 years
MMD seizure frequency per 28 days is defined as total number of MMD seizures reported during the period divided by number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline will be 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.
Up to 4 years
Clinical Global Impression of Improvement (CGI-I) Score
Time Frame: Up to 4 years
The CGI-I Clinician is a 7-point Likert scale that the investigator uses to rate a participant's change (improvement) in overall seizure control, behavior, safety and tolerability, after the initiation of study drug relative to Baseline (before treatment with study drug). The participant will be 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). Higher score will indicate worse symptoms.
Up to 4 years
Caregiver Global Impression of Improvement (Care GI-I) Score
Time Frame: Up to 4 years
The Care GI-I is a 7-point Likert scale that the caregiver uses to rate improvement in overall seizure control, behavior, safety and tolerability after the initiation of study drug relative to Baseline (before treatment with study drug). The participant will be 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 will complete the Care GI-I via interview. Higher score will indicate worse symptoms.
Up to 4 years
CGI-I Seizure Intensity and Duration Score
Time Frame: Up to 4 years
The CGI-I seizure intensity and duration instrument is used by the parent/caregiver to rate improvement in intensity and duration of convulsive seizures (DS Cohort) or MMD seizures (LGS Cohort) from Baseline. The participant's symptoms will be 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). Higher score will indicate worse symptoms.
Up to 4 years
CGI-I Nonseizure Symptoms Score
Time Frame: Up to 4 years
The CGI-I nonseizure symptoms instrument is a series of single-item assessments that the investigator uses to rate improvement in the symptoms and impacts in select nonseizure domains since initiating the study drug. The participant will be 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 is completed by the clinician in collaboration with the primary caregiver to assess the participants status based on the presence of any nonseizure symptoms. Higher score will indicate worse symptoms.
Up to 4 years
Change in Quality of Life Inventory-Disability (QI-Disability) Score
Time Frame: Up to 4 years
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. Scores are from a 5-point Likert scale and then are transformed to a scale of 0 to 100. Possible scores range from 0-100, with higher scores indicating better quality of life.
Up to 4 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Takeda

Investigators

  • Study Director: Study Director, Takeda

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 4, 2022

Primary Completion (Estimated)

May 22, 2026

Study Completion (Estimated)

May 22, 2026

Study Registration Dates

First Submitted

December 15, 2021

First Submitted That Met QC Criteria

December 15, 2021

First Posted (Actual)

December 20, 2021

Study Record Updates

Last Update Posted (Actual)

February 21, 2024

Last Update Submitted That Met QC Criteria

February 20, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

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

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

IPD Sharing Access Criteria

IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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