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

A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Soticlestat as Adjunctive Therapy in Pediatric and Adult Subjects With Lennox-Gastaut Syndrome (LGS)

The aims of the study are:

• to learn if soticlestat, when given as add-on therapy, reduces the number of major motor drop seizures in children, teenagers, and adults with Lennox-Gastaut Syndrome.
• to assess the safety profile of soticlestat when given in combination with other therapies.

Participants will receive their standard antiseizure therapy, plus either tablets of soticlestat or placebo. A placebo looks just like soticlestat but will not have any medicine in it. Participants will take soticlestat or placebo for 16 weeks, followed by a gradual dose reduction for 1 week. Then, participants will be followed up for 2 weeks.

Study Overview

• Status: Completed
• Conditions: Lennox Gastaut Syndrome (LGS)
• Intervention / Treatment: Drug: Soticlestat; Drug: Placebo

Detailed Description

The drug being tested in this study is called soticlestat (TAK-935). Soticlestat will be assessed for efficacy, safety, and tolerability in pediatric and adult participants with LGS.

The study will enroll approximately 234 patients. Participants will be randomly assigned (by chance, like flipping a coin) in a 1:1 ratio to receive standard of care (SOC) plus one of the following adjunctive therapies which will remain undisclosed to the participant and study doctor during the study (unless there is an urgent medical need):

1. Soticlestat
2. Placebo (dummy inactive pill - this is a tablet/mini-tablet that looks like the study drug but has no active ingredient)

Participants will receive soticlestat or matching placebo based on their weight in the 4-week Titration Period. Following the Titration Period, participants will continue to receive the same dose in the 12-week Maintenance Period. The dose will then be down-tapered if participants decide to discontinue the treatment and/or are not deemed eligible to continue in Open-label extension (OLE).

This multi-center trial will be conducted worldwide. The overall time to participate in the study will be from 22-25 weeks. At the end of the treatment period, participants have the option to either complete the study and taper off the investigational product or to enter the OLE if they meet eligibility requirements. If participants discontinue, they will be followed-up on phone call approximately 14 days after the last dose of study drug for safety.

• Study Type: Interventional
• Enrollment (Actual): 270
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Randwick, New South Wales, Australia, 2031
      • Sydney Children's Hospital
  • Queensland
    • South Brisbane, Queensland, Australia, 4101
      • Queensland Childrens Hospital
  • Victoria
    • Heidelberg, Victoria, Australia, 3084
      • Austin Hospital
    • Melbourne, Victoria, Australia, 3004
      • Alfred Hospital
• Belgium
  • Brussels, Belgium
    • Hôpital Universitaire des Enfants Reine Fabiola
  • Antwerpen
    • Edegem, Antwerpen, Belgium, 2650
      • UZ Antwerpen PIN
  • Brabant Wallon
    • Ottignies-Louvain-la-Neuve, Brabant Wallon, Belgium, 1340
      • Centre Neurologique William Lennox
• Canada
  • Alberta
    • Calgary, Alberta, Canada, T3B 6A8
      • Alberta Childrens Hospital
  • British Columbia
    • Vancouver, British Columbia, Canada, V5Z 4H4
      • Child and Family Research Institute
  • Ontario
    • Toronto, Ontario, Canada, M5G 1X8
      • Hospital for Sick Children
• China
  • Beijing
    • Beijing, Beijing, China, 100034
      • Peking University First Hospital
    • Beijing, Beijing, China, 100045
      • Beijing Children's Hospital，Capital Medical University
  • Chongqing
    • Chongqing, Chongqing, China, 400014
      • Children's Hospital of Chongqing Medical University
  • Guangdong
    • Guangzhou, Guangdong, China, 510260
      • The Second Affiliated Hospital of Guangzhou Medical University
    • Guangzhou, Guangdong, China, 510623
      • Guangzhou Women And Children's Medical Center
    • Shenzhen, Guangdong, China, 518026
      • Shenzhen Children's Hospital
  • Hubei
    • Wuhan, Hubei, China, 430010
      • Wuhan Childrens Hospital
  • Hunan
    • Changsha, Hunan, China, 410008
      • Xiangya Hospital Central South University
  • Jiangxi
    • Nanchang, Jiangxi, China, 330006
      • Jiangxi Provincial Children's Hospital
  • Jilin
    • Changchun, Jilin, China, 130021
      • The First Hospital of Jilin University
  • Shanghai
    • Shanghai, Shanghai, China, 201102
      • Children's Hospital of Fudan University
    • Shanghai, Shanghai, China, 200040
      • Children's Hospital of Shanghai
• France
  • Marseille, France, 13386
    • Hopitaux de La Timone
  • Paris, France, 75019
    • Hôpital Robert Debré
  • Paris, France, 75015
    • Hôpital Necker - Enfants Malades
  • Cote-d'Or
    • Dijon, Cote-d'Or, France, 21079
      • CHRU Dijon Hopital General
  • Nord
    • Lille, Nord, France, 59000
      • Hôpital Roger Salengro
• Germany
  • Bayern
    • Vogtareuth, Bayern, Germany, 83569
      • Schön Klinik Vogtareuth
  • Hessen
    • Frankfurt am Main, Hessen, Germany, 60528
      • Klinikum der Johann-Wolfgang Goethe-Universitat
  • Nordrhein-Westfalen
    • Bielefeld, Nordrhein-Westfalen, Germany, 33617
      • Krankenhaus Mara gGmbH - Epilepsiezentrum Bethel
  • Sachse
    • Radeberg, Sachse, Germany, 1454
      • Kleinwachau Sächsisches Epilepsiezentrum Radeberg Gemeinnützige Gmbh
• Greece
  • Larisa, Greece, 411 10
    • University General Hospital of Larissa
  • Thessaloniki, Greece, 546 42
    • Hippokration Hospital
  • Attiki
    • Chaidari, Attiki, Greece, 124 62
      • Attikon University General Hospital
• Hungary
  • Budapest, Hungary, 1145
    • Orszagos Mentalis, Ideggyogyaszati es Idegsebeszeti Intezet
  • Budapest, Hungary, 1023
    • Szent Janos Korhaz es Eszak-budai Egyesitett Korhazak
  • Budapest, Hungary, 1143
    • Bethesda Gyermekkorhaz
  • Baranya
    • Pecs, Baranya, Hungary, 7623
      • Pecsi Tudomanyegyetem
• Italy
  • Emilia-Romagna
    • Bologna, Emilia-Romagna, Italy, 40139
      • Ospedale Bellaria
  • Lazio
    • Roma, Lazio, Italy, 168
      • Fondazione Policlinico Universitario A Gemelli
  • Lombardia
    • Pavia, Lombardia, Italy, 27100
      • ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCS
  • Toscana
    • Firenze, Toscana, Italy, 50139
      • Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
• Japan
  • Aiti
    • Nagakute-Shi, Aiti, Japan, 480-1195
      • Aichi Medical University Hospital
  • Hukuoka
    • Fukuoka-Shi, Hukuoka, Japan, 813-0017
      • Fukuoka Children's Hospital
  • Kumamoto
    • Kumamoto-Shi, Kumamoto, Japan, 862-0947
      • Kumamoto-Ezuko Medical Center for The Severely Disabled
  • Nagasaki
    • Omura-Shi, Nagasaki, Japan, 856-0835
      • National Hospital Organization Nagasaki Medical Center
  • Niigata
    • Niigata-Shi, Niigata, Japan, 950-2074
      • National Hospital Organization Nishi-Niigata Chuo National Hospital
  • Okayama
    • Okayama-city, Okayama, Japan, 700-8558
      • Okayama University Hospital
  • Osaka
    • Neyagawa-Shi, Osaka, Japan, 572-0085
      • Yasuhara Childrens Clinic
    • Osaka-Shi, Osaka, Japan, 534-0021
      • Osaka City General Hospital
    • Suita-Shi, Osaka, Japan, 565-0871
      • Osaka University Hospital
  • Sizuoka
    • Shizuoka-Shi, Sizuoka, Japan, 420-0953
      • National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders
  • Tokyo
    • Kodaira-Shi, Tokyo, Japan, 187-0031
      • National Center of Neurology and Psychiatry
• Latvia
  • Riga, Latvia, LV-1004
    • Childrens University Hospital
• Netherlands
  • Noord-Brabant
    • Heeze, Noord-Brabant, Netherlands, 5591 VE
      • Kempenhaeghe - PPDS
  • Overijssel
    • Zwolle, Overijssel, Netherlands, 8025 BV
      • Stichting Epilepsie Instellingen Nederland
• Poland
  • Gdansk, Poland, 80-952
    • Uniwersyteckie Centrum Kliniczne
  • Poznan, Poland, 60-355
    • Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
  • Malopolskie
    • Krakow, Malopolskie, Poland, 30-363
      • Centrum Medyczne Plejady
• Russian Federation
  • Ekaterinburg, Russian Federation, 620144
    • UGMK-Zdorojie, LLC
• Serbia
  • Belgrade, Serbia, 11000
    • Clinic for Neurology and Psychiatry for Children and Youth
  • Belgrade, Serbia, 11000
    • Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
  • Nis, Serbia, 18 000
    • University Clinical Center Niš
  • Novi Sad, Serbia, 21 000
    • Children and Youth Health Care Institute of Vojvodina
• Spain
  • Barcelona, Spain, 8035
    • Hospital Universitario Vall d'Hebron - PPDS
  • Malaga, Spain, 29010
    • Hospital Regional Universitario de Malaga Hospital General
  • Sevilla, Spain, 41013
    • Centro de Neurologia Avanzada
  • Valencia, Spain, 46026
    • Hospital Universitari i Politecnic La Fe de Valencia
  • Navarra
    • Pamplona, Navarra, Spain, 31008
      • Clinica Universidad Navarra
• Ukraine
  • Ivano-Frankivsk, Ukraine, 76018
    • Communal Non-commercial Enterprise Iv-Frank Regional Childrens Clinical Hosp of Iv-Frank RC
  • Kyiv, Ukraine, 4080
    • CNPE Clinical Hospital Psychiatry of the Executive Body of the Kyiv City Council KCSA
  • Kyiv, Ukraine, 4209
    • SI Ukr. Med. Rehabilitation Center For Children With Organic Injury of Nervous System of MoH of Ukr
  • Dnipropetrovs'ka Oblast
    • Dnipro, Dnipropetrovs'ka Oblast, Ukraine, 49100
      • Municipal Institution Dnipropetrovsk Regional Children Clinical Hospital of DRC
    • Dnipro, Dnipropetrovs'ka Oblast, Ukraine, 49101
      • Communal Non-profit Enterprise City Childrens Clinical Hospital #6 of DCC
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85016
      • Phoenix Childrens Hospital
    • Tucson, Arizona, United States, 85718
      • Center for Neurosciences
  • California
    • Los Angeles, California, United States, 90095
      • David Geffen School of Medicine at UCLA
    • San Francisco, California, United States, 94143
      • University of California Benioff Children's Hospital
  • Colorado
    • Denver, Colorado, United States, 80218
      • Children's Hospital Colorado.
  • Florida
    • Winter Park, Florida, United States, 32789
      • Pediatric Neurology PA
  • Georgia
    • Atlanta, Georgia, United States, 30328
      • Clinical Integrative Research Center of Atlanta
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Hospitals & Clinics - (CRS)
  • Maryland
    • Bethesda, Maryland, United States, 20817
      • Midatlantic Epilepsy and Sleep Center
  • Minnesota
    • Roseville, Minnesota, United States, 55113
      • Minnesota Epilepsy Group PA
  • New Jersey
    • Livingston, New Jersey, United States, 07039
      • Institute of Neurology and Neurosurgery at Saint Barnabas, LLC
  • New York
    • New York, New York, United States, 10016
      • NYU Comprehensive Epilepsy Center
    • New York, New York, United States, 10001
      • Premier Healthcare Inc.
    • New York, New York, United States, 10075
      • Northwell Health Physician Partners - Neurology at Lenox Hill
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health and Science University
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
    • Philadelphia, Pennsylvania, United States, 19107
      • Thomas Jefferson University
    • Philadelphia, Pennsylvania, United States, 19134
      • St. Christopher's Hospital for Children
    • York, Pennsylvania, United States, 17403
      • WellSpan Oncology Research
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina Children Hospital - PIN
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Medical Center - Jane and John Justin Neurosciences Center
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • University of Utah - Primary Children's Hospital - PPDS
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital
    • Tacoma, Washington, United States, 98402
      • MultiCare Institute for Research & Innovation (Tacoma)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 55 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Has documented clinical diagnosis of LGS.
2. Has had ≥8 MMD seizures each month in the 3 months prior to Screening based on the historical information and has had ≥8 MMD seizures per 28 days during the 4 to 6 week prospective Baseline Period.
3. Weighs ≥10 kg at the Screening Visit (Visit 1).
4. Failure to control seizures despite appropriate trials of at least 1 ASM based on historical information, and is currently on an antiseizure therapy or other treatment options considered as standard of care (SOC).
5. Artisanal cannabidiols are allowed at a stable dose for at least 4 weeks before the screening visit (Visit 1); the dosing regimen and manufacturer should remain constant throughout the study. (Artisanal cannabidiols will not be counted as ASMs.)
6. Currently taking 0 to 3 ASMs at stable doses for at least 4 weeks before the Screening Visit (Visit 1); Fenfluramine and cannabidiol (Epidiolex) are allowed where available and counted as an ASM. ASM dosing regimen must remain constant throughout the study.

Exclusion Criteria:

1. Admitted to a medical facility and intubated for treatment of status epilepticus 2 or more times in the 3 months immediately before Screening (Visit 1). For the purpose of this exclusion criterion, status is defined as continuous seizure activity lasting longer than 5 minutes or repeated seizures without return to Baseline in between seizures.
2. 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.
3. Considered by the investigator to be at imminent risk of suicide or injury to self, others, or property, or the participant has attempted suicide within 12 months before the Screening Visit (Visit 1). Participants who have positive answers on item numbers 4 or 5 on the Columbia suicide severity rating scale (C-SSRS) before dosing (Visit 2) are excluded. This scale will only be administered to participants aged ≥6 years.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo; Soticlestat placebo-matching mini-tablets or tablets, orally or via G-tube or MIC-KEY button or J-tube, BID, up to 4 weeks during titration. Participants continued to receive soticlestat placebo-matching mini-tablets or tablets for 12 weeks during maintenance. The total duration of the treatment was up to 16 weeks (Full Treatment Period). Soticlestat matching tapering was done to maintain the blind if participants decided to discontinue the treatment.	Drug: Placebo; Soticlestat placebo-matching mini-tablets or tablets.
Experimental: Soticlestat; Participants weighing <45 kg: Soticlestat, mini-tablets, at the dose of 40 mg to 200 mg, orally or via G-tube or MIC-KEY button or J-tube, BID based on body weight up to 4 weeks during titration. Participants continued to receive the dose that they were on at the end of the titration, for 12 weeks during maintenance. The total duration of the treatment was up to 16 weeks (Full Treatment Period) with the dose tapered down if participants decided to discontinue the treatment. Participants weighing ≥45 kg: Soticlestat mini-tablets or tablets with a starting dose of 100 mg BID followed by 200 mg BID and, then 300 mg BID, up to 4 weeks during titration. Participants continued to receive 300 mg BID for 12 weeks during maintenance. The total duration of the treatment was up to 16 weeks (Full Treatment Period) with the dose tapered down if participants decided to discontinue the treatment.	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
Percent Change From Baseline in Major Motor Drop (MMD) Seizure Frequency Per 28 Days During the Full Treatment Period	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 seizures per 28 days during the full Treatment Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100.	Baseline; Full Treatment Period: Weeks 1 to 16
Percent Change From Baseline in Major Motor Drop (MMD) Seizure Frequency Per 28 Days During the Maintenance Period	MMD seizure frequency per 28 days was defined as the 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 was defined as (frequency of seizures per 28 days during the Maintenance Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100.	Baseline; Maintenance Period: Weeks 5 to 16

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Responders During the Maintenance Period	Responders are defined as those with ≥50% reduction from Baseline in MMD seizures during the Maintenance Period. Percentages are rounded off to the nearest single decimal place.	Maintenance Period: Weeks 5 to 16
Percentage of Responders During the Full Treatment Period	Responders are defined as those with ≥50% reduction from Baseline in MMD seizures during the Full Treatment Period. Percentages are rounded off to the nearest single decimal place.	Full Treatment Period: Weeks 1 to 16
Percentage of Participants With ≤0%, >0% to ≤25%, >25% to ≤50%, >50% to ≤75%, >75% to ≤100% Reduction in MMD Seizure During the Full Treatment Period	Percent reduction from Baseline (%) is defined as [(Full Treatment Period MMD Seizure Frequency - Baseline MMD Seizure Frequency) divided by Baseline MMD Seizure Frequency] multiplied by 100. Data is reported as reduction of ≤0%, >0% to ≤25%, >25% to ≤50%, >50% to ≤75%, >75% to ≤100% or more in seizures from Baseline. Percentages are rounded off to the nearest single decimal place.	Full Treatment Period: Weeks 1 to 16
Percentage of Participants With Caregiver Global Impression of Improvement (Care GI-I) Scale Responses as Per the Parent/Caregiver Reported Impression at Week 16	The Care GI-I is a 7-point Likert scale that the caregiver used to rate improvement in overall seizure control, behavior, safety and tolerability after the initiation of study drug relative to Baseline (before treatment with the study drug). The participant was 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. Lower scores indicate improvement. Percentages are rounded off to the nearest single decimal place.	Week 16
Percentage of Participants With Clinical Global Impression of Improvement (CGI-I) Scale Responses as Per the Investigator Reported Impression at Week 16	The CGI-I (Clinician) is a 7-point Likert scale that the investigator 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 the study drug). The participant was 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 investigator or designee will complete the CGI-I. Lower scores indicate improvement. Percentages are rounded off to the nearest single decimal place.	Week 16
Percentage of Participants With CGI-I Nonseizure Symptoms Instrument Responses for Each Domain as Per the Investigator Reported Impression at Week 16	The CGI-I nonseizure symptoms instrument is a series of single-item assessments that the investigator used to rate improvement in the symptoms and impacts in select nonseizure domains (alertness, communication, and disruptive behaviors) since initiating the study drug. The participant was rated by the investigator for each domain 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). Lower scores indicated improvement. Data for percentage of participants categorized based on the responses for each domain are presented. Percentages are rounded off to the nearest single decimal place.	Week 16
Change From Baseline in Quality of Life Inventory-Disability (QI-Disability) Total Score at Week 16	The QI-Disability tool is a parent/caregiver-reported questionnaire evaluated the 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. Each QI-Disability item is rated on a Likert scale of: Never, Rarely, Sometimes, Often, and Very Often. Items were linearly transformed to a scale of 0 to 100, with higher scores representing better quality of life. Domain scores are calculated by averaging item scores. The domain scores are summed and divided by 6 to yield a total score. The total score ranges from 0 to 100, with higher scores indicating a better quality of life. A negative change from Baseline implies deteriorating quality of life. Mixed-effects model for repeated measures (MMRM) was used for analysis.	Baseline, Week 16
Percentage of Participants With CGI-I Seizure Intensity and Duration Instrument Responses as Per the Parent/Caregiver Reported Impression at Week 16	The CGI-I seizure intensity and duration instrument was used by the parent/caregiver to rate changes in intensity and/or duration of the most impactful seizures from the first assessment. The participant's symptoms 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). Lower scores indicate improvement. Percentages are rounded off to the nearest single decimal place.	Week 16
Percent Change From Baseline in Frequency of All Seizures Per 28 Days During the Maintenance Period	Seizure frequency per 28 days was defined as the total number of 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 seizures per 28 days during the Maintenance Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100.	Baseline; Maintenance Period: Weeks 5 to 16
Percent Change From Baseline in Frequency of All Seizures Per 28 Days During the Full Treatment Period	Seizure frequency per 28 days was defined as the total number of 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 seizures per 28 days during the full Treatment Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100.	Baseline; Full Treatment Period: Weeks 1 to 16
Change From Baseline in Percentage of MMD Seizure-free Days During the Full Treatment Period	MMD Seizure-free days was defined as the number of days the participant remained MMD seizure free after initiation of the treatment. The change from baseline in percentage of MMD seizure-free days, was defined as the percentage of seizure-free days during the Full Treatment Period minus the percentage of seizure-free days during the Baseline. A linear model with treatment group and age stratum as factors and baseline percentage as a covariate was used for analysis.	Baseline up to Week 16
Longest MMD Seizure-free Interval During the Full Treatment Period	Longest MMD Seizure-free Interval was defined as the longest time period that the participant remained MMD seizure-free after initiation of the treatment. A linear model with treatment group and age stratum as factors was used for analysis.	Full Treatment Period: Weeks 1 to 16
Number of Days When Rescue Antiseizure Medication (ASM) is Used During the Full Treatment Period	Use of rescue ASM was recorded in the case report form (CRF) along with start and end date of medication. Based on the start and end dates for all rescue ASMs taken by a participant, the number of days during the Full Treatment Period when rescue ASM was used was derived.	Full Treatment Period: Weeks 1 to 16

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Medical Director, Takeda

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): November 8, 2021
• Primary Completion (Actual): January 25, 2024
• Study Completion (Actual): January 25, 2024

Study Registration Dates

• First Submitted: June 18, 2021
• First Submitted That Met QC Criteria: June 23, 2021
• First Posted (Actual): June 24, 2021

Study Record Updates

• Last Update Posted (Actual): August 21, 2024
• Last Update Submitted That Met QC Criteria: July 25, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Keywords: Drug therapy
• Additional Relevant MeSH Terms: Pathologic Processes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes; Disease; Genetic Diseases, Inborn; Epilepsy; Syndrome; Lennox Gastaut Syndrome
• Other Study ID Numbers: TAK-935-3002; 2021-002481-40 (EudraCT Number); jRCT2051210073 (Registry Identifier: jRCT)

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