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

Primary Purpose

Lennox Gastaut Syndrome (LGS)

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Soticlestat
Placebo
Sponsored by
Takeda
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Lennox Gastaut Syndrome (LGS) focused on measuring Drug therapy

Eligibility Criteria

2 Years - 55 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Phoenix Childrens Hospital
  • Center For Neurosciences
  • David Geffen School of Medicine at UCLA
  • University of California Benioff Children's Hospital
  • Children's Hospital Colorado.
  • Pediatric Neurology PA
  • Clinical Integrative Research Center of Atlanta
  • University of Iowa Hospitals & Clinics - (CRS)
  • Midatlantic Epilepsy and Sleep Center
  • Minnesota Epilepsy Group PA
  • Institute of Neurology and Neurosurgery at Saint Barnabas, LLC
  • Premier Healthcare Inc.
  • Oregon Health and Science University
  • Children's Hospital of Philadelphia
  • Thomas Jefferson University
  • St. Christopher's Hospital for Children
  • WellSpan Oncology Research
  • Medical University of South Carolina Children Hospital - PIN
  • Cook Children's Medical Center - Jane and John Justin Neurosciences Center
  • University of Utah - Primary Children's Hospital - PPDS
  • MultiCare Institute for Research & Innovation (Tacoma)
  • Sydney Children's Hospital
  • Queensland Childrens Hospital
  • Austin Hospital
  • Alfred Hospital
  • UZ Antwerpen PIN
  • Centre Neurologique William Lennox
  • Hopital Universitaire des Enfants Reine Fabiola
  • Alberta Childrens HospitalRecruiting
  • Child and Family Research Institute
  • Hospital For Sick Children
  • Peking University First Hospital
  • Beijing Children's Hospital,Capital Medical University
  • Children's Hospital of Chongqing Medical University
  • The Second Affiliated Hospital of Guangzhou Medical University
  • Guangzhou Women And Children's Medical Center
  • Shenzhen Children's Hospital
  • Wuhan Childrens hospital
  • Xiangya Hospital Central South University
  • Jiangxi Provincial Children's Hospital
  • Children's Hospital of Shanghai
  • Hopital Roger Salengro
  • Hopitaux de La Timone
  • Hopital Necker - Enfants Malades
  • Hopital Robert Debre
  • Schon Klinik VogtareuthRecruiting
  • Klinikum der Johann-Wolfgang Goethe-UniversitatRecruiting
  • Krankenhaus Mara gGmbH - Epilepsiezentrum BethelRecruiting
  • Kleinwachau Sachsisches Epilepsiezentrum Radeberg Gemeinnutzige GmbhRecruiting
  • Attikon University General Hospital
  • University General Hospital of Larissa
  • Hippokration Hospital
  • Pecsi Tudomanyegyetem
  • Szent Janos Korhaz es Eszak-Budai Egyesitett Korhazak
  • Bethesda Gyermekkorhaz
  • Orszagos Mentalis, Ideggyogyaszati es Idegsebeszeti Intezet
  • Ospedale Bellaria
  • Fondazione Policlinico Universitario A Gemelli
  • ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCS
  • Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
  • Aichi Medical University Hospital
  • Fukuoka Children's Hospital
  • National Hospital Organization Nagasaki Medical Center
  • National Hospital Organization Nishi-Niigata Chuo National Hospital
  • Okayama University Hospital
  • Yasuhara Childrens Clinic
  • Osaka City General Hospital
  • Osaka University Hospital
  • National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders
  • National Center of Neurology and Psychiatry
  • Childrens University Hospital
  • Kempenhaeghe - PPDS
  • Stichting Epilepsie Instellingen Nederland
  • Centrum Medyczne Plejady
  • Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
  • UGMK-Zdorojie, LLC
  • Clinic for Neurology and Psychiatry for Children and Youth
  • Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
  • University Clinical Center Nis
  • Children and Youth Health Care Institute of Vojvodina
  • Clinica Universidad Navarra
  • Hospital Universitario Vall d'Hebron - PPDS
  • Hospital Regional Universitario de Malaga Hospital General
  • Centro de Neurologia Avanzada
  • Hospital Universitari i Politecnic La Fe de Valencia
  • Municipal Institution Dnipropetrovsk Regional Children Clinical Hospital of DRC
  • Communal Non-profit Enterprise City Childrens Clinical Hospital #6 of DCC
  • Communal Non-commercial Enterprise Iv-Frank Regional Childrens Clinical Hosp of Iv-Frank RC
  • CNPE Clinical Hospital Psychiatry of the Executive Body of the Kyiv City Council KCSA
  • SI Ukr. Med. Rehabilitation Center For Children With Organic Injury of Nervous System of MoH of Ukr

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Soticlestat

Placebo

Arm Description

Participants weighing <45 kg: Soticlestat, mini-tablets, at the dose of 40 mg to 200 mg, orally or via gastrostomy tube (G-tube) or low-profile gastric tube (MIC-KEY button) or jejunostomy tube (J-tube), twice daily (BID) based on body weight up to 4 weeks in Titration Period. Participants will continue to receive the dose that they are on at the end of the titration period, for 12 weeks in the Maintenance Period. Total duration of the treatment will be up to 16 weeks (Treatment Period). Dose will be tapered down if participants decide 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 in Titration Period. Participants will continue to receive 300 mg BID for 12 weeks in the Maintenance Period. Total duration of the treatment will be up to 16 weeks (Treatment Period). Dose will be tapered down if participants decide to discontinue the treatment.

Soticlestat placebo-matching mini-tablets or tablets, orally or via G-tube or MIC-KEY button or J-tube, BID, up to 4 weeks in the Titration Period. Participants will continue to receive the soticlestat placebo-matching mini-tablets or tablets for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). Soticlestat matching tapering will be done to maintain the blind if participants decide to discontinue the treatment.

Outcomes

Primary Outcome Measures

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 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 seizures per 28 days during 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.
Percent Change from Baseline in Major Motor Drop (MMD) Seizure Frequency Per 28 Days During the Maintenance Period (EMA Region Specific)
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 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. This outcome measure is European Medicines Agency (EMA) region specific.

Secondary Outcome Measures

Percentage of Responders During the Maintenance Period
Responders are defined as those with ≥50% reduction from Baseline in MMD seizures during the Maintenance Period.
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.
Percentage of Participants with ≤0%, >0% to ≤25%, >25% to ≤50%, >50% to ≤75%, >75% to ≤100% Reduction in MMD Seizure in a Cumulative Response Curve
Caregiver Global Impression of Improvement (Care GI-I) Score
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 the 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.
Clinical Global Impression of Improvement (CGI-I) Score
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 the 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.
CGI-I Nonseizure Symptoms Score
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 by the investigator on the 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.
Change in Quality of Life Inventory-Disability (QI-Disability) Score
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.
CGI-I Seizure Intensity and Duration Score
The CGI-I Seizure Intensity and Duration instrument is 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 will be rated on the 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.
Percent Change From Baseline in Frequency of All Seizures per 28 Days During the Maintenance Period
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 Maintenance Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100.
Percent Change from Baseline in Frequency of All Seizures per 28 Days During the Full Treatment Period
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 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.
Percent Change from Baseline in MMD Seizure Frequency per 28 Days During the Maintenance Period
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 is defined as (frequency of seizures per 28 days during Maintenance Period - frequency of seizures per 28 days at Baseline) divided by frequency of seizures per 28 days at Baseline multiplied by 100.
Change From Baseline in Percentage of MMD Seizure-free Days
MMD Seizure-free days will be defined as the number of days the participant remained MMD seizure free after initiation of the treatment.
Longest MMD Seizure-free Interval
Longest MMD Seizure-free Interval will be defined as the longest time period that the participant remained MMD seizure free after initiation of the treatment.
Number of Days When Rescue Antiseizure Medication (ASM) is Used

Full Information

First Posted
June 18, 2021
Last Updated
September 12, 2023
Sponsor
Takeda
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1. Study Identification

Unique Protocol Identification Number
NCT04938427
Brief Title
A Study of Soticlestat as an Add-on Therapy in Children, Teenagers, and Adults With Lennox-Gastaut Syndrome
Official Title
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)
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
November 8, 2021 (Actual)
Primary Completion Date
February 16, 2024 (Anticipated)
Study Completion Date
February 16, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Takeda

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.
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): Soticlestat 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Lennox Gastaut Syndrome (LGS)
Keywords
Drug therapy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
234 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Soticlestat
Arm Type
Experimental
Arm Description
Participants weighing <45 kg: Soticlestat, mini-tablets, at the dose of 40 mg to 200 mg, orally or via gastrostomy tube (G-tube) or low-profile gastric tube (MIC-KEY button) or jejunostomy tube (J-tube), twice daily (BID) based on body weight up to 4 weeks in Titration Period. Participants will continue to receive the dose that they are on at the end of the titration period, for 12 weeks in the Maintenance Period. Total duration of the treatment will be up to 16 weeks (Treatment Period). Dose will be tapered down if participants decide 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 in Titration Period. Participants will continue to receive 300 mg BID for 12 weeks in the Maintenance Period. Total duration of the treatment will be up to 16 weeks (Treatment Period). Dose will be tapered down if participants decide to discontinue the treatment.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Soticlestat placebo-matching mini-tablets or tablets, orally or via G-tube or MIC-KEY button or J-tube, BID, up to 4 weeks in the Titration Period. Participants will continue to receive the soticlestat placebo-matching mini-tablets or tablets for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). Soticlestat matching tapering will be done to maintain the blind if participants decide to discontinue the treatment.
Intervention Type
Drug
Intervention Name(s)
Soticlestat
Other Intervention Name(s)
TAK-935
Intervention Description
Soticlestat mini-tablets or tablets.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Soticlestat placebo-matching mini-tablets or tablets.
Primary Outcome Measure Information:
Title
Percent Change from Baseline in Major Motor Drop (MMD) Seizure Frequency Per 28 Days During the Full Treatment Period
Description
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 seizures per 28 days during 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.
Time Frame
Baseline up to Week 16
Title
Percent Change from Baseline in Major Motor Drop (MMD) Seizure Frequency Per 28 Days During the Maintenance Period (EMA Region Specific)
Description
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 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. This outcome measure is European Medicines Agency (EMA) region specific.
Time Frame
Baseline up to Week 16
Secondary Outcome Measure Information:
Title
Percentage of Responders During the Maintenance Period
Description
Responders are defined as those with ≥50% reduction from Baseline in MMD seizures during the Maintenance Period.
Time Frame
Baseline up to Week 16
Title
Percentage of Responders During the Full Treatment Period
Description
Responders are defined as those with ≥50% reduction from Baseline in MMD seizures during the full Treatment Period.
Time Frame
Baseline up to Week 16
Title
Percentage of Participants with ≤0%, >0% to ≤25%, >25% to ≤50%, >50% to ≤75%, >75% to ≤100% Reduction in MMD Seizure in a Cumulative Response Curve
Time Frame
Baseline up to Week 16
Title
Caregiver Global Impression of Improvement (Care GI-I) Score
Description
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 the 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.
Time Frame
Baseline up to Week 16
Title
Clinical Global Impression of Improvement (CGI-I) Score
Description
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 the 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.
Time Frame
Baseline up to Week 16
Title
CGI-I Nonseizure Symptoms Score
Description
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 by the investigator on the 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.
Time Frame
Baseline up to Week 16
Title
Change in Quality of Life Inventory-Disability (QI-Disability) Score
Description
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.
Time Frame
Baseline up to Week 16
Title
CGI-I Seizure Intensity and Duration Score
Description
The CGI-I Seizure Intensity and Duration instrument is 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 will be rated on the 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.
Time Frame
Baseline up to Week 16
Title
Percent Change From Baseline in Frequency of All Seizures per 28 Days During the Maintenance Period
Description
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 Maintenance Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100.
Time Frame
Baseline up to Week 16
Title
Percent Change from Baseline in Frequency of All Seizures per 28 Days During the Full Treatment Period
Description
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 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.
Time Frame
Baseline up to Week 16
Title
Percent Change from Baseline in MMD Seizure Frequency per 28 Days During the Maintenance Period
Description
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 is defined as (frequency of seizures per 28 days during Maintenance Period - frequency of seizures per 28 days at Baseline) divided by frequency of seizures per 28 days at Baseline multiplied by 100.
Time Frame
Baseline up to Week 16
Title
Change From Baseline in Percentage of MMD Seizure-free Days
Description
MMD Seizure-free days will be defined as the number of days the participant remained MMD seizure free after initiation of the treatment.
Time Frame
Baseline up to Week 16
Title
Longest MMD Seizure-free Interval
Description
Longest MMD Seizure-free Interval will be defined as the longest time period that the participant remained MMD seizure free after initiation of the treatment.
Time Frame
Baseline up to Week 16
Title
Number of Days When Rescue Antiseizure Medication (ASM) is Used
Time Frame
Baseline up to Week 16

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Has documented clinical diagnosis of LGS. 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. Weighs ≥10 kg at the Screening Visit (Visit 1). 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). 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.) 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: 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. 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. 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Takeda Contact
Phone
+1-877-825-3327
Email
medinfoUS@takeda.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Phoenix Childrens Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Center For Neurosciences
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85718
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
David Geffen School of Medicine at UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
University of California Benioff Children's Hospital
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Children's Hospital Colorado.
City
Denver
State/Province
Colorado
ZIP/Postal Code
80218
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Pediatric Neurology PA
City
Winter Park
State/Province
Florida
ZIP/Postal Code
32789
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Clinical Integrative Research Center of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30328
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
University of Iowa Hospitals & Clinics - (CRS)
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Midatlantic Epilepsy and Sleep Center
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20817
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Minnesota Epilepsy Group PA
City
Roseville
State/Province
Minnesota
ZIP/Postal Code
55113
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Institute of Neurology and Neurosurgery at Saint Barnabas, LLC
City
Livingston
State/Province
New Jersey
ZIP/Postal Code
07039
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Premier Healthcare Inc.
City
New York
State/Province
New York
ZIP/Postal Code
10001
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Thomas Jefferson University
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
St. Christopher's Hospital for Children
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19134
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
WellSpan Oncology Research
City
York
State/Province
Pennsylvania
ZIP/Postal Code
17403
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Medical University of South Carolina Children Hospital - PIN
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Cook Children's Medical Center - Jane and John Justin Neurosciences Center
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
University of Utah - Primary Children's Hospital - PPDS
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84113
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
MultiCare Institute for Research & Innovation (Tacoma)
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98402
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Sydney Children's Hospital
City
Randwick
State/Province
New South Wales
ZIP/Postal Code
2031
Country
Australia
Individual Site Status
Active, not recruiting
Facility Name
Queensland Childrens Hospital
City
South Brisbane
State/Province
Queensland
ZIP/Postal Code
4101
Country
Australia
Individual Site Status
Active, not recruiting
Facility Name
Austin Hospital
City
Heidelberg
State/Province
Victoria
ZIP/Postal Code
3084
Country
Australia
Individual Site Status
Active, not recruiting
Facility Name
Alfred Hospital
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3004
Country
Australia
Individual Site Status
Active, not recruiting
Facility Name
UZ Antwerpen PIN
City
Edegem
State/Province
Antwerpen
ZIP/Postal Code
2650
Country
Belgium
Individual Site Status
Active, not recruiting
Facility Name
Centre Neurologique William Lennox
City
Ottignies-Louvain-la-Neuve
State/Province
Brabant Wallon
ZIP/Postal Code
1340
Country
Belgium
Individual Site Status
Active, not recruiting
Facility Name
Hopital Universitaire des Enfants Reine Fabiola
City
Brussels
Country
Belgium
Individual Site Status
Active, not recruiting
Facility Name
Alberta Childrens Hospital
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T3B 6A8
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
(403) 955-7319
Email
julia.jacobs@uniklinik-freiburg.de
First Name & Middle Initial & Last Name & Degree
Julia Jacobs-LeVan
Facility Name
Child and Family Research Institute
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V5Z 4H4
Country
Canada
Individual Site Status
Active, not recruiting
Facility Name
Hospital For Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Individual Site Status
Active, not recruiting
Facility Name
Peking University First Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100034
Country
China
Individual Site Status
Active, not recruiting
Facility Name
Beijing Children's Hospital,Capital Medical University
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100045
Country
China
Individual Site Status
Active, not recruiting
Facility Name
Children's Hospital of Chongqing Medical University
City
Chongqing
State/Province
Chongqing
ZIP/Postal Code
400014
Country
China
Individual Site Status
Active, not recruiting
Facility Name
The Second Affiliated Hospital of Guangzhou Medical University
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510260
Country
China
Individual Site Status
Active, not recruiting
Facility Name
Guangzhou Women And Children's Medical Center
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510623
Country
China
Individual Site Status
Active, not recruiting
Facility Name
Shenzhen Children's Hospital
City
Shenzhen
State/Province
Guangdong
ZIP/Postal Code
518026
Country
China
Individual Site Status
Active, not recruiting
Facility Name
Wuhan Childrens hospital
City
Wuhan
State/Province
Hubei
ZIP/Postal Code
430010
Country
China
Individual Site Status
Active, not recruiting
Facility Name
Xiangya Hospital Central South University
City
Changsha
State/Province
Hunan
ZIP/Postal Code
410008
Country
China
Individual Site Status
Active, not recruiting
Facility Name
Jiangxi Provincial Children's Hospital
City
Nanchang
State/Province
Jiangxi
ZIP/Postal Code
330006
Country
China
Individual Site Status
Active, not recruiting
Facility Name
Children's Hospital of Shanghai
City
Shanghai
State/Province
Shanghai
ZIP/Postal Code
200040
Country
China
Individual Site Status
Active, not recruiting
Facility Name
Hopital Roger Salengro
City
Lille
State/Province
Nord
ZIP/Postal Code
59000
Country
France
Individual Site Status
Active, not recruiting
Facility Name
Hopitaux de La Timone
City
Marseille
ZIP/Postal Code
13386
Country
France
Individual Site Status
Active, not recruiting
Facility Name
Hopital Necker - Enfants Malades
City
Paris
ZIP/Postal Code
75015
Country
France
Individual Site Status
Active, not recruiting
Facility Name
Hopital Robert Debre
City
Paris
ZIP/Postal Code
75019
Country
France
Individual Site Status
Active, not recruiting
Facility Name
Schon Klinik Vogtareuth
City
Vogtareuth
State/Province
Bayern
ZIP/Postal Code
83569
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
498888888888
Email
pringsheim@dhm.mhn.de
First Name & Middle Initial & Last Name & Degree
Milka Pringsheim
Facility Name
Klinikum der Johann-Wolfgang Goethe-Universitat
City
Frankfurt am Main
State/Province
Hessen
ZIP/Postal Code
60528
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
4964215865348
Email
rosenow@med.uni-fra
First Name & Middle Initial & Last Name & Degree
Felix Rosenow
Facility Name
Krankenhaus Mara gGmbH - Epilepsiezentrum Bethel
City
Bielefeld
State/Province
Nordrhein-Westfalen
ZIP/Postal Code
33617
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
4952177278804
Email
christian.brandt@mara.de
First Name & Middle Initial & Last Name & Degree
Christian Brandt
Facility Name
Kleinwachau Sachsisches Epilepsiezentrum Radeberg Gemeinnutzige Gmbh
City
Radeberg
State/Province
Sachse
ZIP/Postal Code
1454
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
4935284311680
Email
t.mayer@kleinwachau.de
First Name & Middle Initial & Last Name & Degree
Thomas Mayer
Facility Name
Attikon University General Hospital
City
Chaidari
State/Province
Attiki
ZIP/Postal Code
124 62
Country
Greece
Individual Site Status
Active, not recruiting
Facility Name
University General Hospital of Larissa
City
Larisa
ZIP/Postal Code
411 10
Country
Greece
Individual Site Status
Active, not recruiting
Facility Name
Hippokration Hospital
City
Thessaloniki
ZIP/Postal Code
546 42
Country
Greece
Individual Site Status
Active, not recruiting
Facility Name
Pecsi Tudomanyegyetem
City
Pecs
State/Province
Baranya
ZIP/Postal Code
7623
Country
Hungary
Individual Site Status
Active, not recruiting
Facility Name
Szent Janos Korhaz es Eszak-Budai Egyesitett Korhazak
City
Budapest
ZIP/Postal Code
1023
Country
Hungary
Individual Site Status
Active, not recruiting
Facility Name
Bethesda Gyermekkorhaz
City
Budapest
ZIP/Postal Code
1143
Country
Hungary
Individual Site Status
Active, not recruiting
Facility Name
Orszagos Mentalis, Ideggyogyaszati es Idegsebeszeti Intezet
City
Budapest
ZIP/Postal Code
1145
Country
Hungary
Individual Site Status
Active, not recruiting
Facility Name
Ospedale Bellaria
City
Bologna
State/Province
Emilia-Romagna
ZIP/Postal Code
40139
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
Fondazione Policlinico Universitario A Gemelli
City
Roma
State/Province
Lazio
ZIP/Postal Code
168
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCS
City
Pavia
State/Province
Lombardia
ZIP/Postal Code
27100
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
City
Firenze
State/Province
Toscana
ZIP/Postal Code
50139
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
Aichi Medical University Hospital
City
Nagakute-Shi
State/Province
Aiti
ZIP/Postal Code
480-1195
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Fukuoka Children's Hospital
City
Fukuoka-Shi
State/Province
Hukuoka
ZIP/Postal Code
813-0017
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
National Hospital Organization Nagasaki Medical Center
City
Omura-Shi
State/Province
Nagasaki
ZIP/Postal Code
856-0835
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
National Hospital Organization Nishi-Niigata Chuo National Hospital
City
Niigata-Shi
State/Province
Niigata
ZIP/Postal Code
950-2074
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Okayama University Hospital
City
Okayama-city
State/Province
Okayama
ZIP/Postal Code
700-8558
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Yasuhara Childrens Clinic
City
Neyagawa-Shi
State/Province
Osaka
ZIP/Postal Code
572-0085
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Osaka City General Hospital
City
Osaka-Shi
State/Province
Osaka
ZIP/Postal Code
534-0021
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Osaka University Hospital
City
Suita-Shi
State/Province
Osaka
ZIP/Postal Code
565-0871
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders
City
Shizuoka-Shi
State/Province
Sizuoka
ZIP/Postal Code
420-0953
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
National Center of Neurology and Psychiatry
City
Kodaira-Shi
State/Province
Tokyo
ZIP/Postal Code
187-0031
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Childrens University Hospital
City
Riga
ZIP/Postal Code
LV-1004
Country
Latvia
Individual Site Status
Active, not recruiting
Facility Name
Kempenhaeghe - PPDS
City
Heeze
State/Province
Noord-Brabant
ZIP/Postal Code
5591 VE
Country
Netherlands
Individual Site Status
Active, not recruiting
Facility Name
Stichting Epilepsie Instellingen Nederland
City
Zwolle
State/Province
Overijssel
ZIP/Postal Code
8025 BV
Country
Netherlands
Individual Site Status
Active, not recruiting
Facility Name
Centrum Medyczne Plejady
City
Krakow
State/Province
Malopolskie
ZIP/Postal Code
30-363
Country
Poland
Individual Site Status
Active, not recruiting
Facility Name
Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
City
Poznan
ZIP/Postal Code
60-355
Country
Poland
Individual Site Status
Active, not recruiting
Facility Name
UGMK-Zdorojie, LLC
City
Ekaterinburg
ZIP/Postal Code
620144
Country
Russian Federation
Individual Site Status
Active, not recruiting
Facility Name
Clinic for Neurology and Psychiatry for Children and Youth
City
Belgrade
ZIP/Postal Code
11000
Country
Serbia
Individual Site Status
Active, not recruiting
Facility Name
Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
City
Belgrade
ZIP/Postal Code
11000
Country
Serbia
Individual Site Status
Active, not recruiting
Facility Name
University Clinical Center Nis
City
Nis
ZIP/Postal Code
18 000
Country
Serbia
Individual Site Status
Active, not recruiting
Facility Name
Children and Youth Health Care Institute of Vojvodina
City
Novi Sad
ZIP/Postal Code
21 000
Country
Serbia
Individual Site Status
Active, not recruiting
Facility Name
Clinica Universidad Navarra
City
Pamplona
State/Province
Navarra
ZIP/Postal Code
31008
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Hospital Universitario Vall d'Hebron - PPDS
City
Barcelona
ZIP/Postal Code
8035
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Hospital Regional Universitario de Malaga Hospital General
City
Malaga
ZIP/Postal Code
29010
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Centro de Neurologia Avanzada
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Hospital Universitari i Politecnic La Fe de Valencia
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Municipal Institution Dnipropetrovsk Regional Children Clinical Hospital of DRC
City
Dnipro
State/Province
Dnipropetrovs'ka Oblast
ZIP/Postal Code
49100
Country
Ukraine
Individual Site Status
Active, not recruiting
Facility Name
Communal Non-profit Enterprise City Childrens Clinical Hospital #6 of DCC
City
Dnipro
State/Province
Dnipropetrovs'ka Oblast
ZIP/Postal Code
49101
Country
Ukraine
Individual Site Status
Active, not recruiting
Facility Name
Communal Non-commercial Enterprise Iv-Frank Regional Childrens Clinical Hosp of Iv-Frank RC
City
Ivano-Frankivsk
ZIP/Postal Code
76018
Country
Ukraine
Individual Site Status
Active, not recruiting
Facility Name
CNPE Clinical Hospital Psychiatry of the Executive Body of the Kyiv City Council KCSA
City
Kyiv
ZIP/Postal Code
4080
Country
Ukraine
Individual Site Status
Active, not recruiting
Facility Name
SI Ukr. Med. Rehabilitation Center For Children With Organic Injury of Nervous System of MoH of Ukr
City
Kyiv
ZIP/Postal Code
4209
Country
Ukraine
Individual Site Status
Active, not recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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 URL
https://vivli.org/ourmember/takeda/
Links:
URL
https://clinicaltrials.takeda.com/study-detail/60e30ebd1f1122001e30a940
Description
Related Info

Learn more about this trial

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

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