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

NCT05163314 | Terminated Phase 3 DMC FDA Drug

• 4 other identifiers: TAK-935-30032021-002482-17jRCT20512101822022-502802-34-00
• Study Type: interventional
• Target: 352
• Locations: 20 countries
• Sites: 116

Brief Summary

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.

Conditions

Dravet Syndrome (DS); Lennox Gastaut Syndrome (LGS)

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (6)

• Number of Participants With At Least One Treatment Emergent Adverse Event (TEAE)

  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 Columbia-Suicide Severity Rating Scale (C-SSRS) Score

  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

• Change from Baseline in Body Weight for All Age Groups

  Up to 4 years

• Change from Baseline in Height for All Age Groups

  Up to 4 years

• Absolute Value for Tanner Stage for Children 6 to 17 Years of Age During the Study

  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

  Up to 4 years

Secondary Outcomes (8)

• Percent Change from Baseline in Total Seizure Frequency per 28 Days for DS and LGS Participants

  Up to 4 years

• Percent Change from Baseline in Convulsive Seizure Frequency per 28 Days in DS Cohort

  Up to 4 years

• Percent Change from Baseline in Major Motor Drop (MMD) Seizure Frequency per 28 Days in LGS Cohort

  Up to 4 years

• Clinical Global Impression of Improvement (CGI-I) Score

  Up to 4 years

• Caregiver Global Impression of Improvement (Care GI-I) Score

  Up to 4 years

Study Arms (1)

Soticlestat

EXPERIMENTAL

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

Interventions

Soticlestat DRUG

Soticlestat mini-tablets or tablets

Also known as: TAK-935

Soticlestat

Eligibility Criteria

• Age: 2 Years - 56 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• \. Participant must have:
• Been previously enrolled in a phase 3 soticlestat clinical study.

You may not qualify if:

• 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.
• 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.
• 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.

Sponsors & Collaborators

• Takeda: lead

Study Sites (116)

Phoenix Childrens Hospital

Phoenix, Arizona, 85016, United States

Location

Center For Neurosciences

Tucson, Arizona, 85718, United States

Location

David Geffen School of Medicine at UCLA

Los Angeles, California, 90095, United States

Location

University of California Benioff Children's Hospital

San Francisco, California, 94143, United States

Location

Colorado Children's Hospital

Denver, Colorado, 80218, United States

Location

Pediatric Neurology PA

Winter Park, Florida, 32789, United States

Location

Clinical Integrative Research Center of Atlanta

Atlanta, Georgia, 30328, United States

Location

Sunrise Pediatric Neurology

Marietta, Georgia, 30066, United States

Location

University of Iowa Hospitals & Clinics - (CRS)

Iowa City, Iowa, 52242, United States

Location

Midatlantic Epilepsy and Sleep Center

Bethesda, Maryland, 20817, United States

Location

Minnesota Epilepsy Group PA

Saint Paul, Minnesota, 55102, United States

Location

Institute of Neurology and Neurosurgery at Saint Barnabas, LLC

Livingston, New Jersey, 07039, United States

Location

Boston Children's Health Physicians (BCHP)

New York, New York, 10003, United States

Location

Northwell Health Physician Partners - Neurology at Lenox Hill

New York, New York, 10016, United States

Location

NYU Comprehensive Epilepsy Center

New York, New York, 10016, United States

Location

University of Toledo

Toledo, Ohio, 43614, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

St. Christopher's Hospital for Children

Philadelphia, Pennsylvania, 19134, United States

Location

WellSpan Oncology Research

York, Pennsylvania, 17403, United States

Location

Medical University of South Carolina Children Hospital - PIN

Charleston, South Carolina, 29425, United States

Location

Cook Children's Medical Center - Jane and John Justin Neurosciences Center

Fort Worth, Texas, 76104, United States

Location

University of Utah - Primary Children's Hospital - PPDS

Salt Lake City, Utah, 84113, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

MultiCare Institute for Research & Innovation (Tacoma)

Tacoma, Washington, 98402, United States

Location

Sydney Children's Hospital

Randwick, New South Wales, 2031, Australia

Location

Queensland Childrens Hospital

Brisbane, Queensland, 4101, Australia

Location

Austin Hospital

Heidelberg, Victoria, 3084, Australia

Location

Alfred Hospital

Melbourne, Victoria, 3004, Australia

Location

UZ Antwerpen

Edegem, Antwerpen, 2650, Belgium

Location

Centre Neurologique William Lennox

Ottignies-Louvain-la-Neuve, Brabant Wallon, 1340, Belgium

Location

Hopital Universitaire des Enfants Reine Fabiola

Brussels, Brussels Capital, 1020, Belgium

Location

Instituto de Neurologia de Curitiba (INC)

Curitiba, Paraná, 81210-310, Brazil

Location

Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)

Porto Alegre, Rio Grande do Sul, 90610-000, Brazil

Location

Hospital das Clinicas - UNICAMP

Campinas, São Paulo, 13083-887, Brazil

Location

Universidade Federal de Sao Paulo

São Paulo, 04023-900, Brazil

Location

Universidade de Sao Paulo

São Paulo, 05403-010, Brazil

Location

Child and Family Research Institute

Vancouver, British Columbia, V5Z 4H4, Canada

Location

Hospital For Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Peking University First Hospital

Beijing, Beijing Municipality, 100034, China

Location

Beijing Children's Hospital,Capital Medical University

Beijing, Beijing Municipality, 100045, China

Location

Children's Hospital of Chongqing Medical University

Chongqing, Chongqing Municipality, 400014, China

Location

The Second Affiliated Hospital of Guangzhou Medical Univeristy

Guangzhou, Guangdong, 510260, China

Location

Guangzhou Women And Children's Medical Center

Guangzhou, Guangdong, 510623, China

Location

Shenzhen Children's Hospital

Shenzhen, Guangdong, 518026, China

Location

Wuhan Childrens hospital

Wuhan, Hubei, 430010, China

Location

Xiangya Hospital of Central South University

Changsha, Hunan, 410008, China

Location

Jiangxi Provincial Children's Hospital

Nanchang, Jiangxi, 330006, China

Location

The First Hospital of Jilin University

Changchun, Jilin, 130021, China

Location

Children's Hospital of Shanghai

Shanghai, Shanghai Municipality, 200040, China

Location

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, 201102, China

Location

Hopitaux de La Timone

Marseille, Bouches-du-Rhone, 13005, France

Location

CHRU Dijon Hopital General

Dijon, Cote-d'Or, 21079, France

Location

Hopital Roger Salengro

Lille, Nord, 59000, France

Location

Hopital Necker - Enfants Malades

Paris, 75015, France

Location

Hopital Robert Debre

Paris, 75019, France

Location

Alberta Childrens Hospital

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

Location

Klinikum der Johann-Wolfgang Goethe-Universitat

Frankfurt am Main, Hesse, 60528, Germany

Location

Krankenhaus Mara gGmbH - Epilepsiezentrum Bethel

Bielefeld, North Rhine-Westphalia, 33617, Germany

Location

Kleinwachau Sachsisches Epilepsiezentrum Radeberg Gemeinnutzige Gmbh

Radeberg, Saxony, 01454, Germany

Location

Childrens' Hospital of Athens 'P. and A. Kyriakou'

Athens, Attica, 115 27, Greece

Location

Attikon University General Hospital

Chaïdári, Attica, 124 62, Greece

Location

University General Hospital of Larissa

Larissa, 411 10, Greece

Location

Hippokration Hospital

Thessaloniki, 546 42, Greece

Location

Pecsi Tudomanyegyetem

Pécs, Baranya, 7623, Hungary

Location

Szent Janos Korhaz es Eszak-Budai Egyesitett Korhazak

Budapest, 1023, Hungary

Location

Bethesda Gyermekkorhaz

Budapest, 1143, Hungary

Location

Orszagos Klinikai Idegtudomanyi Intezet

Budapest, 1145, Hungary

Location

Ospedale Bellaria

Rome, Lazio, 00185, Italy

Location

IRCCS Ospedale Pediatrico Bambino Gesu - INCIPIT - PIN

Rome, Lazio, 00197, Italy

Location

Fondazione Policlinico Universitario A Gemelli

Rome, Lazio, Italy

Location

ASST di Mantova - Azienda Ospedaliera Carlo Poma

Mantova, Lombardy, 46100, Italy

Location

ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCS

Pavia, Lombardy, 27100, Italy

Location

Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN

Florence, Tuscany, 50139, Italy

Location

Aichi Medical University Hospital

Nagakute-Shi, Aiti, 480-1195, Japan

Location

Fukuoka Children's Hospital

Fukuoka, Hukuoka, 813-0017, Japan

Location

Kanagawa Prefectural Hospital Organization Kanagawa Children's Medical Center

Yokohama, Kanagawa, 232-0066, Japan

Location

Kumamoto-Ezuko Medical Center for The Severely Disabled

Kumamoto, Kumamoto, 862-0947, Japan

Location

National Hospital Organization Nagasaki Medical Center

Omura-Shi, Nagasaki, 856-0835, Japan

Location

National Hospital Organization Nishi-Niigata Chuo National Hospital

Niigata, Niigata, 950-2074, Japan

Location

Okayama University Hospital

Okayama, Okayama-ken, 700-8558, Japan

Location

Yasuhara Childrens Clinic

Neyagawa, Osaka, 572-0085, Japan

Location

Osaka City General Hospital

Osaka, Osaka, 534-0021, Japan

Location

Osaka University Hospital

Suita-Shi, Osaka, 565-0871, Japan

Location

National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders

Shizuoka, Shizuoka, 420-0953, Japan

Location

Hokkaido University Hospital

Chuo-Ku, Tokyo, 104-0045, Japan

Location

National Center of Neurology and Psychiatry

Kodaira-Shi, Tokyo, 187-0031, Japan

Location

Childrens University Hospital

Riga, LV-1004, Latvia

Location

Hospital Civil Fray Antonio Alcalde

El Retiro, Jalisco, 44280, Mexico

Location

Kempenhaeghe - PPDS

Heeze, North Brabant, 5591 VE, Netherlands

Location

Stichting Epilepsie Instellingen Nederland

Zwolle, Overijssel, 8025 BV, Netherlands

Location

Centrum Medyczne Plejady

Krakow, Lesser Poland Voivodeship, 30-363, Poland

Location

Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie

Warsaw, Masovian Voivodeship, 02-091, Poland

Location

Uniwersyteckie Centrum Kliniczne

Gdansk, 80-952, Poland

Location

Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu

Poznan, 60-355, Poland

Location

Russian National Research Medical University n.a. N.I.Pirogov

Moscow, Moscow, 117437, Russia

Location

Krasnoyarsk State Medical University n.a. V.F. Voyno-Ysenetskiy

Krasnoyarsk, 660022, Russia

Location

Russian National Research Medical University n.a. N.I.Pirogov

Moscow, 125412, Russia

Location

Tyumen State Medical Academy

Tyumen, 625023, Russia

Location

UGMK-Zdorojie, LLC

Yekaterinburg, 620144, Russia

Location

Clinic for Neurology and Psychiatry for Children and Youth

Belgrade, 11000, Serbia

Location

Mother and Child Health Care Institute of Serbia Dr Vukan Cupic

Belgrade, 11000, Serbia

Location

University Clinical Center Nis

Niš, 18 000, Serbia

Location

Children and Youth Health Care Institute of Vojvodina

Novi Sad, 21 000, Serbia

Location

Clinica Universidad Navarra

Pamplona, Navarre, 31008, Spain

Location

Hospital Regional Universitario de Malaga Hospital General

Almería, 04009, Spain

Location

Hospital Universitario Vall d'Hebron - PPDS

Barcelona, 8035, Spain

Location

Hospital Vithas La Salud

Granada, 18008, Spain

Location

Centro de Neurologia Avanzada

Seville, 41013, Spain

Location

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, 46026, Spain

Location

Municipal Institution Dnipropetrovsk Regional Children Clinical Hospital of DRC

Dnipro, Dnipropetrovsk Oblast, 49100, Ukraine

Location

Communal Non-profit Enterprise Dnipro City Children Clinical Hospital #5 of DCC

Dnipro, Dnipropetrovsk Oblast, 49101, Ukraine

Location

Communal Non-commercial Enterprise Iv-Frank Regional Childrens Clinical Hosp of Iv-Frank RC

Ivano-Frankivsk, 76018, Ukraine

Location

CNPE Clinical Hospital Psychiatry of the Executive Body of the Kyiv City Council KCSA

Kyiv, 4080, Ukraine

Location

SI Ukr. Med. Rehabilitation Center For Children With Organic Injury of Nervous System of MoH of Ukr

Kyiv, 4209, Ukraine

Location

Related Links

• 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.

MeSH Terms

Conditions

Epilepsies, Myoclonic; Lennox Gastaut Syndrome

Interventions

soticlestat

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2021
• First Posted: December 20, 2021
• Study Start: March 4, 2022
• Primary Completion: September 24, 2025
• Study Completion: September 24, 2025
• Last Updated: October 8, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• 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.

Locations

PL (4); ME (1); RU (5); JP (13); BE (3); AU (4); IT (6); CA (2); NL (2); SE (4); HU (4); ES (6); US (26); LA (1); GR (4); CN (12); FR (5); DE (4); UA (5); BR (5)