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

Primary Purpose

Dravet Syndrome (DS)

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 Dravet Syndrome (DS) focused on measuring Drug Therapy

Eligibility Criteria

2 Years - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Has documented clinical diagnosis of DS.
  2. Had ≥12 convulsive seizures over 12 weeks before screening based on the historical information and has had ≥4 convulsive 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 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 4 ASMs at stable doses for at least 4 weeks before the screening visit (Visit 1); benzodiazepines used chronically (daily) to treat seizures are considered ASMs. Fenfluramine and cannabidiol (Epidiolex) are allowed where available and should be counted as an ASM. ASM dosing regimen must remain constant throughout the study.

Exclusion Criteria:

1. Unstable, clinically significant neurologic (other than the disease being studied), psychiatric, cardiovascular, ophthalmologic, pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, endocrine disease, malignancy including progressive tumors, or other abnormality that may impact the ability to participate in the study or that may potentially confound the study results. It is the responsibility of the investigator to assess the clinical significance; however, consultation with the medical monitor may be warranted.

Sites / Locations

  • Phoenix Childrens HospitalRecruiting
  • David Geffen School of Medicine at UCLARecruiting
  • University of California Benioff Children's HospitalRecruiting
  • Pediatric Neurology PARecruiting
  • Clinical Integrative Research Center of AtlantaRecruiting
  • University of Iowa Hospitals & Clinics - (CRS)Recruiting
  • NYU Comprehensive Epilepsy CenterRecruiting
  • University of ToledoRecruiting
  • Oregon Health and Science UniversityRecruiting
  • Medical University of South CarolinaRecruiting
  • Cook Children's Medical Center
  • Seattle Children's HospitalRecruiting
  • Multicare Health System - Mary Bridge PediatricsRecruiting
  • Sydney Children's HospitalRecruiting
  • Queensland Childrens HospitalRecruiting
  • Austin HospitalRecruiting
  • Alfred HospitalRecruiting
  • UZ Antwerpen PINRecruiting
  • Hopital Universitaire des Enfants Reine FabiolaRecruiting
  • Instituto de Neurologia de Curitiba (INC)
  • Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
  • Hospital das Clinicas - UNICAMP
  • Universidade Federal de Sao Paulo
  • Universidade de Sao PauloRecruiting
  • Alberta Childrens HospitalRecruiting
  • Child and Family Research InstituteRecruiting
  • Hospital For Sick ChildrenRecruiting
  • Peking University First HospitalRecruiting
  • Beijing Children's Hospital,Capital Medical UniversityRecruiting
  • Children's Hospital of Chongqing Medical University
  • The Second Affiliated Hospital of Guangzhou Medical UniversityRecruiting
  • Guangzhou Women And Children's Medical CenterRecruiting
  • Shenzhen Children's HospitalRecruiting
  • Wuhan Childrens hospitalRecruiting
  • Xiangya Hospital Central South UniversityRecruiting
  • The First Hospital of Jilin UniversityRecruiting
  • Children's Hospital of ShanghaiRecruiting
  • Children's Hospital of Fudan UniversityRecruiting
  • CHRU Dijon Hopital General
  • Hopital Roger SalengroRecruiting
  • Hopitaux de La TimoneRecruiting
  • Hopital Necker - Enfants MaladesRecruiting
  • Hopital Robert DebreRecruiting
  • Schon Klinik VogtareuthRecruiting
  • Klinikum der Johann-Wolfgang Goethe-UniversitatRecruiting
  • Krankenhaus Mara gGmbH - Epilepsiezentrum BethelRecruiting
  • Kleinwachau Sachsisches Epilepsiezentrum Radeberg Gemeinnutzige GmbhRecruiting
  • Childrens' Hospital of Athens 'P. and A. Kyriakou'
  • Attikon University General HospitalRecruiting
  • University General Hospital of Larissa
  • Hippokration Hospital
  • Szent Janos Korhaz es Eszak-Budai Egyesitett KorhazakRecruiting
  • Orszagos Mentalis, Ideggyogyaszati es Idegsebeszeti IntezetRecruiting
  • IRCCS Ospedale Pediatrico Bambino Gesu - INCIPIT - PINRecruiting
  • Fondazione Policlinico Universitario A GemelliRecruiting
  • ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCSRecruiting
  • Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PINRecruiting
  • Aichi Medical University HospitalRecruiting
  • Fukuoka Children's HospitalRecruiting
  • Kanagawa Prefectural Hospital Organization Kanagawa Children's Medical CenterRecruiting
  • Kumamoto-Ezuko Medical Center for The Severely DisabledRecruiting
  • National Hospital Organization Nagasaki Medical CenterRecruiting
  • National Hospital Organization Nishi-Niigata Chuo National HospitalRecruiting
  • Okayama University HospitalRecruiting
  • Yasuhara Childrens ClinicRecruiting
  • Osaka City General HospitalRecruiting
  • Osaka University HospitalRecruiting
  • National Hospital Organization Shizuoka Institute of Epilepsy and Neurological DisordersRecruiting
  • Hokkaido University HospitalRecruiting
  • National Center of Neurology and PsychiatryRecruiting
  • Childrens University HospitalRecruiting
  • Kempenhaeghe - PPDSRecruiting
  • Stichting Epilepsie Instellingen NederlandRecruiting
  • Centrum Medyczne PlejadyRecruiting
  • Neurosphera SP. Z O.ORecruiting
  • Uniwersyteckie Centrum Kliniczne
  • Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w PoznaniuRecruiting
  • Russian National Research Medical University n.a. N.I.Pirogov
  • UGMK-Zdorojie, LLC
  • Krasnoyarsk State Medical University n.a. V.F. Voyno-Ysenetskiy
  • Russian National Research Medical University n.a. N.I.Pirogov
  • Tyumen State Medical Academy
  • Clinic for Neurology and Psychiatry for Children and YouthRecruiting
  • Mother and Child Health Care Institute of Serbia Dr Vukan CupicRecruiting
  • University Clinical Center NisRecruiting
  • Children and Youth Health Care Institute of VojvodinaRecruiting
  • Clinica Universidad Navarra
  • Hospital Universitario Vall d'Hebron - PPDSRecruiting
  • Hospital Regional Universitario de Malaga Hospital GeneralRecruiting
  • Centro de Neurologia AvanzadaRecruiting
  • Hospital Universitari i Politecnic La Fe de ValenciaRecruiting
  • 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 <45kg: Soticlestat, mini-tablets, at the dose of 40mg to 200mg, 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 the body weight up to 4 weeks in Titration Period. Participants will continue to receive dose that they are on at the end of Titration Period, for 12 weeks in Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). The dose will be tapered down if participants decide to discontinue the treatment. Participants weighing ≥45kg: Soticlestat mini-tablets or tablets with a starting dose of 100mg BID followed by 200 mg BID and, then 300mg BID, up to 4 weeks in Titration Period. Participants will continue to receive 300mg BID for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). The 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. 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 Convulsive Seizure Frequency per 28 days During the Full Treatment Period
Convulsive seizure frequency per 28 days is defined as total number of convulsive seizures reported during the period divided by number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline will be defined as (frequency of seizures per 28 days during Treatment Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100.
Percent Change From Baseline in Convulsive Seizure Frequency per 28 days During the Maintenance Period (EMA Region Specific)
Convulsive seizure frequency per 28 days is defined as total number of convulsive seizures reported during the period divided by number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline will be defined as (frequency of 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. This outcome measure is European Medicines Agency (EMA) specific.

Secondary Outcome Measures

Percentage of Responders During Maintenance Period
Responders are defined as those with ≥50% reduction from baseline in convulsive seizures during the Maintenance Period.
Percentage of Responders During the Full Treatment Period
Responders are defined as those with ≥50% reduction from baseline in convulsive seizures during the full Treatment Period.
Percentage of Participants with ≤0%, >0% to ≤25%, >25% to ≤50%, >50% to ≤75%, and >75% to ≤100% Reduction in Convulsive Seizures 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 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 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 on 7-point scale by the investigator as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). At baseline, a symptoms form is completed by the clinician in collaboration with the primary caregiver to assess the participants status based on the presence of any nonseizure symptoms. Higher score will indicate worse symptoms.
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 improvement in intensity and/or duration of the most impactful seizures from the first assessment. The participant's symptoms will be rated on 7-point scale as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). Higher score will indicate worse symptoms.
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 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 Convulsive Seizure Frequency per 28 days During the Maintenance Period
Convulsive seizure frequency per 28 days is defined as total number of convulsive seizures reported during the period divided by number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline 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 Convulsive Seizure-free Days
Convulsive seizure-free days will be defined as number of days a participant remained convulsive seizure free after initiation of the treatment.
Longest Convulsive Seizure-free Interval.
Longest convulsive seizure-free interval will be defined as the longest time period that the participant remained convulsive seizure free after initiation of the treatment.
Number of Days When Rescue Antiseizure Medication (ASM) is Used

Full Information

First Posted
June 16, 2021
Last Updated
September 12, 2023
Sponsor
Takeda
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1. Study Identification

Unique Protocol Identification Number
NCT04940624
Brief Title
A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet 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 Young Adult Subjects With Dravet Syndrome (DS)
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 28, 2021 (Actual)
Primary Completion Date
April 18, 2024 (Anticipated)
Study Completion Date
April 18, 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
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The main aim of the study is to learn if soticlestat, when given as an add-on therapy, reduces the number of convulsive seizures in children and young adults with DS. Participants will receive their standard antiseizure therapy, plus either a tablet of soticlestat or placebo for 16 weeks. A placebo looks just like soticlestat but will not have any medicine in it. Participants may continue treatment in an extension study, based on the extension study's entry criteria. Those that want to stop treatment will have a gradual dose reduction during 1 week and then be followed up for 2 weeks.
Detailed Description
The drug being tested in this study is called soticlestat (TAK-935). Soticlestat as an adjunctive therapy will be assessed for efficacy, safety, and tolerability in pediatric and adult participants with DS. The study will enroll approximately 142 pediatric and young adult patients. Participants will be randomized at a 1:1 ratio to receive standard of care (SOC) plus one of the following adjunctive therapies: Soticlestat or Placebo The total daily dose of study drug will be calculated based on body weight in the 4 weeks Titration Period. Following the Titration Period, participants will continue to receive the same dose in the 12-weeks Maintenance Period. 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
Dravet Syndrome (DS)
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
142 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Soticlestat
Arm Type
Experimental
Arm Description
Participants weighing <45kg: Soticlestat, mini-tablets, at the dose of 40mg to 200mg, 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 the body weight up to 4 weeks in Titration Period. Participants will continue to receive dose that they are on at the end of Titration Period, for 12 weeks in Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). The dose will be tapered down if participants decide to discontinue the treatment. Participants weighing ≥45kg: Soticlestat mini-tablets or tablets with a starting dose of 100mg BID followed by 200 mg BID and, then 300mg BID, up to 4 weeks in Titration Period. Participants will continue to receive 300mg BID for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). The 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. 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 Convulsive Seizure Frequency per 28 days During the Full Treatment Period
Description
Convulsive seizure frequency per 28 days is defined as total number of convulsive seizures reported during the period divided by number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline will be defined as (frequency of seizures per 28 days during Treatment Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100.
Time Frame
Baseline up to Week 16
Title
Percent Change From Baseline in Convulsive Seizure Frequency per 28 days During the Maintenance Period (EMA Region Specific)
Description
Convulsive seizure frequency per 28 days is defined as total number of convulsive seizures reported during the period divided by number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline will be defined as (frequency of 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. This outcome measure is European Medicines Agency (EMA) specific.
Time Frame
Baseline up to Week 16
Secondary Outcome Measure Information:
Title
Percentage of Responders During Maintenance Period
Description
Responders are defined as those with ≥50% reduction from baseline in convulsive 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 convulsive 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%, and >75% to ≤100% Reduction in Convulsive Seizures 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 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 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 on 7-point scale by the investigator as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). At baseline, a symptoms form is completed by the clinician in collaboration with the primary caregiver to assess the participants status based on the presence of any nonseizure symptoms. Higher score will indicate worse symptoms.
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 improvement in intensity and/or duration of the most impactful seizures from the first assessment. The participant's symptoms will be rated on 7-point scale as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). Higher score will indicate worse symptoms.
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 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 Convulsive Seizure Frequency per 28 days During the Maintenance Period
Description
Convulsive seizure frequency per 28 days is defined as total number of convulsive seizures reported during the period divided by number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline 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 Convulsive Seizure-free Days
Description
Convulsive seizure-free days will be defined as number of days a participant remained convulsive seizure free after initiation of the treatment.
Time Frame
Baseline up to Week 16
Title
Longest Convulsive Seizure-free Interval.
Description
Longest convulsive seizure-free interval will be defined as the longest time period that the participant remained convulsive 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
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Has documented clinical diagnosis of DS. Had ≥12 convulsive seizures over 12 weeks before screening based on the historical information and has had ≥4 convulsive 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 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 4 ASMs at stable doses for at least 4 weeks before the screening visit (Visit 1); benzodiazepines used chronically (daily) to treat seizures are considered ASMs. Fenfluramine and cannabidiol (Epidiolex) are allowed where available and should be counted as an ASM. ASM dosing regimen must remain constant throughout the study. Exclusion Criteria: 1. Unstable, clinically significant neurologic (other than the disease being studied), psychiatric, cardiovascular, ophthalmologic, pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, endocrine disease, malignancy including progressive tumors, or other abnormality that may impact the ability to participate in the study or that may potentially confound the study results. It is the responsibility of the investigator to assess the clinical significance; however, consultation with the medical monitor may be warranted.
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
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
602-933-0970
Email
awilfong@phoenixchildrens.com
First Name & Middle Initial & Last Name & Degree
Angus Wilfong
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
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
310-825-6196
Email
shussain@mednet.ucla.edu
First Name & Middle Initial & Last Name & Degree
Shaun Hussain
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
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
415-353-8440
Email
joseph.sullivan@ucsf.edu
First Name & Middle Initial & Last Name & Degree
Joseph Sullivan
Facility Name
Pediatric Neurology PA
City
Winter Park
State/Province
Florida
ZIP/Postal Code
32789
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
407-293-1122
Email
rgdavis@pediatricneurologypa.com
First Name & Middle Initial & Last Name & Degree
Ronald Davis
Facility Name
Clinical Integrative Research Center of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30328
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
678-705-7341
Email
rflamini@pandaneuro.com
First Name & Middle Initial & Last Name & Degree
Julio Flamini
Facility Name
University of Iowa Hospitals & Clinics - (CRS)
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
319-384-5437
Email
michael-ciliberto@uiowa.edu
First Name & Middle Initial & Last Name & Degree
Michael Ciliberto
Facility Name
NYU Comprehensive Epilepsy Center
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
646-558-0842
Email
Orrin.Devinsky@nyulangone.org; Od4@nyu.edu
First Name & Middle Initial & Last Name & Degree
Orrin Devinsky
Facility Name
University of Toledo
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43606
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
419-383-3801
Email
andrea.korsnack@utoledo.edu
First Name & Middle Initial & Last Name & Degree
Imran Ali
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
503-494-5856
Email
robertco@ohsu.edu
First Name & Middle Initial & Last Name & Degree
Colin Roberts
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29403
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
843-792-3221
Email
halfordj@musc.edu
First Name & Middle Initial & Last Name & Degree
Jonathan Halford
Facility Name
Cook Children's Medical Center
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
206-987-2078
Email
russ.saneto@seattlechildrens.org
First Name & Middle Initial & Last Name & Degree
Russell Saneto
Facility Name
Multicare Health System - Mary Bridge Pediatrics
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98405
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
253-403-7251
Email
saphillips@multicare.org
First Name & Middle Initial & Last Name & Degree
Steven Phillips
Facility Name
Sydney Children's Hospital
City
Randwick
State/Province
New South Wales
ZIP/Postal Code
2031
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+61293821658
Email
michael.cardamone@health.nsw.gov.au
First Name & Middle Initial & Last Name & Degree
Michael Cardamone
Facility Name
Queensland Childrens Hospital
City
South Brisbane
State/Province
Queensland
ZIP/Postal Code
4101
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
61730621957
Email
kate.riney@health.qld.gov.au
First Name & Middle Initial & Last Name & Degree
Catherine Riney
Facility Name
Austin Hospital
City
Heidelberg
State/Province
Victoria
ZIP/Postal Code
3084
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+61390357112
Email
i.scheffer@unimelb.edu.au
First Name & Middle Initial & Last Name & Degree
Ingrid Scheffer
Facility Name
Alfred Hospital
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3004
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+61390762000
Email
te.obrien@alfred.org.au
First Name & Middle Initial & Last Name & Degree
Terence OBrien
Facility Name
UZ Antwerpen PIN
City
Edegem
State/Province
Antwerpen
ZIP/Postal Code
2650
Country
Belgium
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+3234660610
Email
berten.ceulemans@uza.be
First Name & Middle Initial & Last Name & Degree
Berten Ceulemans
Facility Name
Hopital Universitaire des Enfants Reine Fabiola
City
Brussels
Country
Belgium
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+3224773174
Email
alec.aeby@huderf.be
First Name & Middle Initial & Last Name & Degree
Alec Aeby
Facility Name
Instituto de Neurologia de Curitiba (INC)
City
Curitiba
State/Province
Parana
ZIP/Postal Code
81210-310
Country
Brazil
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
554130288545
Email
pkowacs@gmail.com
First Name & Middle Initial & Last Name & Degree
Pedro Kowacs
Facility Name
Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
City
Porto Alegre
State/Province
Rio Grande Do Sul
ZIP/Postal Code
90610-000
Country
Brazil
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
555133205133
Email
apalmini@uol.com.br
First Name & Middle Initial & Last Name & Degree
Andre Palmini
Facility Name
Hospital das Clinicas - UNICAMP
City
Campinas
State/Province
Sao Paulo
ZIP/Postal Code
13083-888
Country
Brazil
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+551937887481
Email
fcendes@unicamp.br
First Name & Middle Initial & Last Name & Degree
Fernando Cendes
Facility Name
Universidade Federal de Sao Paulo
City
Vila Clementino
State/Province
Sao Paulo
ZIP/Postal Code
04023-062
Country
Brazil
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+551155792581
Email
lauragui@gmail.com
First Name & Middle Initial & Last Name & Degree
Laura Maria Guilhoto
Facility Name
Universidade de Sao Paulo
City
Sao Paulo
ZIP/Postal Code
04039-032
Country
Brazil
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+551130696000
Email
kettevalente@msn.com
First Name & Middle Initial & Last Name & Degree
Kette Dualibi Ramos Valente
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
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
(604) 875-2612
Email
cyrus.boelman@cw.bc.ca
First Name & Middle Initial & Last Name & Degree
Cyrus Boelman
Facility Name
Hospital For Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
(416) 813-7037
Email
cecil.hahn@sickkids.ca
First Name & Middle Initial & Last Name & Degree
Cecil Hahn
Facility Name
Peking University First Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100034
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+8613701398992
Email
jiangyuwu@bjmu.edu.cn
First Name & Middle Initial & Last Name & Degree
Yuwu Jiang
Facility Name
Beijing Children's Hospital,Capital Medical University
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100045
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+861059616161
Email
13910150389@163.com
First Name & Middle Initial & Last Name & Degree
Fang Fang
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
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+862034152626
Email
wpliao@163.net
First Name & Middle Initial & Last Name & Degree
Weiping Liao
Facility Name
Guangzhou Women And Children's Medical Center
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510623
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+8618902268798
Email
gzchcwx@126.com
First Name & Middle Initial & Last Name & Degree
Wenxiong Chen
Facility Name
Shenzhen Children's Hospital
City
Shenzhen
State/Province
Guangdong
ZIP/Postal Code
518026
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+8675583936101
Email
liaojianxiang@vip.sina.com
First Name & Middle Initial & Last Name & Degree
Jianxiang Liao
Facility Name
Wuhan Childrens hospital
City
Wuhan
State/Province
Hubei
ZIP/Postal Code
430010
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+8613971616910
Email
bloveriver@163.com
First Name & Middle Initial & Last Name & Degree
Dan Sun
Facility Name
Xiangya Hospital Central South University
City
Changsha
State/Province
Hunan
ZIP/Postal Code
410008
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+8613548966986
Email
pengjing4346@163.com
First Name & Middle Initial & Last Name & Degree
Jing Peng
Facility Name
The First Hospital of Jilin University
City
Changchun
State/Province
Jilin
ZIP/Postal Code
130021
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+8643188782291
Email
jackyliangjm@163.com
First Name & Middle Initial & Last Name & Degree
Jianmin Liang
Facility Name
Children's Hospital of Shanghai
City
Shanghai
State/Province
Shanghai
ZIP/Postal Code
200040
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+8618930590051
Email
chenyc@shchildren.com.cn
First Name & Middle Initial & Last Name & Degree
Yucai Chen
Facility Name
Children's Hospital of Fudan University
City
Shanghai
State/Province
Shanghai
ZIP/Postal Code
201102
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+8618017591161
Email
szzhou@shmu.edu.cn
First Name & Middle Initial & Last Name & Degree
Shuizhen Zhou
Facility Name
CHRU Dijon Hopital General
City
Dijon
State/Province
Cote-d'Or
ZIP/Postal Code
21079
Country
France
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
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+33320446362
Email
sylvie.nguyenthetich@chru-lille.fr
First Name & Middle Initial & Last Name & Degree
Sylvie Nguyen
Facility Name
Hopitaux de La Timone
City
Marseille
ZIP/Postal Code
13386
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+33491385580
Email
mathieu.milh@ap-hm.fr
First Name & Middle Initial & Last Name & Degree
Milh Mathieu
Facility Name
Hopital Necker - Enfants Malades
City
Paris
ZIP/Postal Code
75015
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+33144494854
Email
rima.nabbout@aphp.fr
First Name & Middle Initial & Last Name & Degree
Rima El Nabbout-Tarantino
Facility Name
Hopital Robert Debre
City
Paris
ZIP/Postal Code
75019
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+33140033667
Email
stephane.auvin@aphp.fr
First Name & Middle Initial & Last Name & Degree
Stephane Auvin
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
4952177278893
Email
tilman.polster@mara.de
First Name & Middle Initial & Last Name & Degree
Tilman Polster
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
Childrens' Hospital of Athens 'P. and A. Kyriakou'
City
Athens
State/Province
Attiki
ZIP/Postal Code
115 27
Country
Greece
Individual Site Status
Active, not recruiting
Facility Name
Attikon University General Hospital
City
Chaidari
State/Province
Attiki
ZIP/Postal Code
124 62
Country
Greece
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+302105831269
Email
argidino@yahoo.com
First Name & Middle Initial & Last Name & Degree
Argirios Dinopoulos
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
Szent Janos Korhaz es Eszak-Budai Egyesitett Korhazak
City
Budapest
ZIP/Postal Code
1023
Country
Hungary
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+3614584500
Email
altmann.anna@yahoo.com
First Name & Middle Initial & Last Name & Degree
Anna Altmann
Facility Name
Orszagos Mentalis, Ideggyogyaszati es Idegsebeszeti Intezet
City
Budapest
ZIP/Postal Code
1145
Country
Hungary
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+36306894111
Email
fabo.daniel@gmail.com
First Name & Middle Initial & Last Name & Degree
Daniel Fabo
Facility Name
IRCCS Ospedale Pediatrico Bambino Gesu - INCIPIT - PIN
City
Roma
State/Province
Lazio
ZIP/Postal Code
164
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
390668592645
Email
nicola.specchio@opbg.net
First Name & Middle Initial & Last Name & Degree
Nicola Specchio
Facility Name
Fondazione Policlinico Universitario A Gemelli
City
Roma
State/Province
Lazio
ZIP/Postal Code
168
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
390630154901
Email
eeg@rm.unicatt.it
First Name & Middle Initial & Last Name & Degree
Domenica Battaglia
Facility Name
ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCS
City
Pavia
State/Province
Lombardia
ZIP/Postal Code
27100
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+390382380289
Email
valentina.degiorgis@mondino.it
First Name & Middle Initial & Last Name & Degree
Valentina De Giorgis
Facility Name
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
City
Firenze
State/Province
Toscana
ZIP/Postal Code
50139
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+390555662573
Email
renzo.guerrini@meyer.it
First Name & Middle Initial & Last Name & Degree
Renzo Guerrini
Facility Name
Aichi Medical University Hospital
City
Nagakute-Shi
State/Province
Aiti
ZIP/Postal Code
480-1195
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+81561623311
Email
okumura.akihisa.479@mail.aichi-med-u.ac.jp
First Name & Middle Initial & Last Name & Degree
Akihisa Okumura
Facility Name
Fukuoka Children's Hospital
City
Fukuoka-Shi
State/Province
Hukuoka
ZIP/Postal Code
813-0017
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+81926827000
Email
kira.r@fcho.jp
First Name & Middle Initial & Last Name & Degree
Ryutaro Kira
Facility Name
Kanagawa Prefectural Hospital Organization Kanagawa Children's Medical Center
City
Yokohama-Shi
State/Province
Kanagawa
ZIP/Postal Code
232-0066
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
81457112351
Email
tgoto@kcmc.jp
First Name & Middle Initial & Last Name & Degree
Tomohide Goto
Facility Name
Kumamoto-Ezuko Medical Center for The Severely Disabled
City
Kumamoto-Shi
State/Province
Kumamoto
ZIP/Postal Code
862-0947
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+81963700501
Email
T-ishitsu@ezuko.jp
First Name & Middle Initial & Last Name & Degree
Takateru Ishitsu
Facility Name
National Hospital Organization Nagasaki Medical Center
City
Omura-Shi
State/Province
Nagasaki
ZIP/Postal Code
856-0835
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+81957523121
Email
honda.ryoko.gu@mail.hosp.go.jp
First Name & Middle Initial & Last Name & Degree
Ryoko Honda
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
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+81252653171
Email
tohyama.jun.um@mail.hosp.go.jp
First Name & Middle Initial & Last Name & Degree
Jun Tohyama
Facility Name
Okayama University Hospital
City
Okayama-city
State/Province
Okayama
ZIP/Postal Code
700-8558
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+81862357991
Email
k_koba@md.okayama-u.ac.jp
First Name & Middle Initial & Last Name & Degree
Katsuhiro Kobayashi
Facility Name
Yasuhara Childrens Clinic
City
Neyagawa-Shi
State/Province
Osaka
ZIP/Postal Code
572-0085
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+81728322211
Email
akihiro-yasuhara@car.ocn.ne.jp
First Name & Middle Initial & Last Name & Degree
Akihiro Yasuhara
Facility Name
Osaka City General Hospital
City
Osaka-Shi
State/Province
Osaka
ZIP/Postal Code
534-0021
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+81669291221
Email
e2007kawawaki@yahoo.co.jp
First Name & Middle Initial & Last Name & Degree
Hisashi Kawawaki
Facility Name
Osaka University Hospital
City
Suita-Shi
State/Province
Osaka
ZIP/Postal Code
565-0871
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+81668795111
Email
nabatames@ped.med.osaka-u.ac.jp
First Name & Middle Initial & Last Name & Degree
Shin Nabatame
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
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+81542455446
Email
s.hizuokasan3@googlemail.com
First Name & Middle Initial & Last Name & Degree
Tokito Yamaguchi
Facility Name
Hokkaido University Hospital
City
Chuo-Ku
State/Province
Tokyo
ZIP/Postal Code
104-0045
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
81117067028
Email
siraisi@med.hokudai.ac.jp
First Name & Middle Initial & Last Name & Degree
Hideaki Shiraishi
Facility Name
National Center of Neurology and Psychiatry
City
Kodaira-Shi
State/Province
Tokyo
ZIP/Postal Code
187-0031
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+81423462190
Email
nakagawa@ncnp.go.jp
First Name & Middle Initial & Last Name & Degree
Eiji Nakagawa
Facility Name
Childrens University Hospital
City
Riga
ZIP/Postal Code
LV-1004
Country
Latvia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+37129535342
Email
dr.rozentals@inbox.lv
First Name & Middle Initial & Last Name & Degree
Guntis Rozentals
Facility Name
Kempenhaeghe - PPDS
City
Heeze
State/Province
Noord-Brabant
ZIP/Postal Code
5591 VE
Country
Netherlands
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+31402279203
Email
majoiem@kempenhaeghe.nl
First Name & Middle Initial & Last Name & Degree
Marian Majoie
Facility Name
Stichting Epilepsie Instellingen Nederland
City
Zwolle
State/Province
Overijssel
ZIP/Postal Code
8025 BV
Country
Netherlands
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+31683709913
Email
bgunning@sein.nl
First Name & Middle Initial & Last Name & Degree
Boudewijn Gunning
Facility Name
Centrum Medyczne Plejady
City
Krakow
State/Province
Malopolskie
ZIP/Postal Code
30-363
Country
Poland
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+48123535555
Email
marta.zolnowska@gmail.com
First Name & Middle Initial & Last Name & Degree
Marta Zolnowska
Facility Name
Neurosphera SP. Z O.O
City
Warszawa
State/Province
Mazowieckie
ZIP/Postal Code
02-952
Country
Poland
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
48602216032
Email
beata.m.zwolinska@gmail.com
First Name & Middle Initial & Last Name & Degree
Beata Zwolinska
Facility Name
Uniwersyteckie Centrum Kliniczne
City
Gdansk
ZIP/Postal Code
80-952
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
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+48618691613
Email
bstein@ump.edu.pl
First Name & Middle Initial & Last Name & Degree
Barbara Steinborn
Facility Name
Russian National Research Medical University n.a. N.I.Pirogov
City
Moscow
State/Province
Moskva
ZIP/Postal Code
117437
Country
Russian Federation
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
Krasnoyarsk State Medical University n.a. V.F. Voyno-Ysenetskiy
City
Krasnoyarsk
ZIP/Postal Code
660022
Country
Russian Federation
Individual Site Status
Active, not recruiting
Facility Name
Russian National Research Medical University n.a. N.I.Pirogov
City
Moscow
ZIP/Postal Code
125412
Country
Russian Federation
Individual Site Status
Active, not recruiting
Facility Name
Tyumen State Medical Academy
City
Tyumen'
ZIP/Postal Code
625023
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
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+381638679625
Email
milan.borkovic.npk@gmail.com
First Name & Middle Initial & Last Name & Degree
Milan Borkovic
Facility Name
Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
City
Belgrade
ZIP/Postal Code
11000
Country
Serbia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+381642612210
Email
ruzica.kravljanac@gmail.com
First Name & Middle Initial & Last Name & Degree
Ruzica Kravljanac
Facility Name
University Clinical Center Nis
City
Nis
ZIP/Postal Code
18 000
Country
Serbia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+381642572634
Email
tosic.tatjana@gmail.com
First Name & Middle Initial & Last Name & Degree
Tatjana Tosic
Facility Name
Children and Youth Health Care Institute of Vojvodina
City
Novi Sad
ZIP/Postal Code
21 000
Country
Serbia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+38121424385
Email
mkp.marija@gmail.com
First Name & Middle Initial & Last Name & Degree
Marija Knezevic-Pogancev
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
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+34934894257
Email
mtoledo@vhebron.net
First Name & Middle Initial & Last Name & Degree
Manuel Toledo
Facility Name
Hospital Regional Universitario de Malaga Hospital General
City
Malaga
ZIP/Postal Code
29010
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
34671562365
Email
pedro.serrano.c@gmail.com
First Name & Middle Initial & Last Name & Degree
Pedro Serrano
Facility Name
Centro de Neurologia Avanzada
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
34955277751
Email
uranganeuro@gmail.com
First Name & Middle Initial & Last Name & Degree
Juan Jesus Rodriguez Uranga
Facility Name
Hospital Universitari i Politecnic La Fe de Valencia
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+34961244161
Email
vevillanuevah@yahoo.es
First Name & Middle Initial & Last Name & Degree
Vicente Villanueva
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/60e40858ef0b71001e743e10
Description
To obtain more information on the study, click here/on this link

Learn more about this trial

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

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