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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05163314
Registration number
NCT05163314
Ethics application status
Date submitted
15/12/2021
Date registered
20/12/2021
Titles & IDs
Public title
A Study of Soticlestat as an Add-on Therapy in Children and Adults With Dravet Syndrome or Lennox-Gastaut Syndrome
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Scientific title
A Phase 3, Prospective, Open-Label, Multisite, Extension of Phase 3 Studies To Assess the Long-Term Safety and Tolerability of Soticlestat as Adjunctive Therapy in Subjects With Dravet Syndrome or Lennox-Gastaut Syndrome (ENDYMION 2)
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Secondary ID [1]
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2021-002482-17
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Secondary ID [2]
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TAK-935-3003
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Dravet Syndrome (DS)
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Lennox Gastaut Syndrome (LGS)
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Condition category
Condition code
Neurological
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Epilepsy
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Soticlestat
Experimental: Soticlestat - Participants with DS and LGS will receive:Participants weighing \<45kg:Soticlestat,mini-tablets,titrated from lower dose level(60mg to 140mg) to higher dose(100mg to 200mg) twice daily(BID),based on body weight,orally/via enteral feeding tubes including but not limited to nasogastric(NG)-tube,gastrostomy tube(G-tube),MIC-KEY button,upto 2 weeks in Titration Period. Will continue to receive dose they are on at end of Titration Period,for approximately 4 years in Maintenance Period.Dose will be tapered down to lower dose(not less than lowest dose level based on weight)every 3 days until study drug is discontinued(upto 1week) in Taper Period.Participants weighing =45kg/adults:Soticlestat mini-tablets/tablets with starting dose of 200mg BID followed by 300mg BID,up to 2 weeks in Titration Period.Will continue to receive 300mg BID for approximately 4 years in Maintenance Period.Dose will be tapered down upto 100mg every 3 days until study drug is discontinued(up to 1 week) in Taper Period.
Treatment: Drugs: Soticlestat
Soticlestat mini-tablets or tablets
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Participants With At Least One Treatment Emergent Adverse Event (TEAE)
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Assessment method [1]
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An Adverse Event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A TEAE is any AE either reported for the first time or worsening of a pre-existing event after first dose of study drug and within 30 days of the last administration of study drug.
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Timepoint [1]
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Up to 4 years
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Primary outcome [2]
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Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS) Score
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Assessment method [2]
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C-SSRS systematically tracks suicidal ideation and behavior. The total score range is 0 (no ideation is present) to 5 (active suicidal ideation with specific plan and intent). The higher the score, the greater one's suicidal ideation.
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Timepoint [2]
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Up to 4 years
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Primary outcome [3]
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Change from Baseline in Body Weight for All Age Groups
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Assessment method [3]
0
0
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Timepoint [3]
0
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Up to 4 years
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Primary outcome [4]
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Change from Baseline in Height for All Age Groups
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Assessment method [4]
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0
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Timepoint [4]
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Up to 4 years
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Primary outcome [5]
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Absolute Value for Tanner Stage for Children 6 to 17 Years of Age During the Study
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Assessment method [5]
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Tanner assessment score is used to document the stage of development of puberty by assessing the secondary sexual characteristics, rated in 5 stages: stage 1 (no development) to 5 (adult-like development in quantity and size).
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Timepoint [5]
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Up to 4 years
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Primary outcome [6]
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Absolute Value for Insulin-like Growth Factor 1 (IGF-1) for Children 2 to 17 Years of Age During the Study
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Assessment method [6]
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Timepoint [6]
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Up to 4 years
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Secondary outcome [1]
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Percent Change from Baseline in Total Seizure Frequency per 28 Days for DS and LGS Participants
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Assessment method [1]
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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.
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Timepoint [1]
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Up to 4 years
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Secondary outcome [2]
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Percent Change from Baseline in Convulsive Seizure Frequency per 28 Days in DS Cohort
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Assessment method [2]
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Convulsive seizure frequency per 28 days is defined as total number of convulsive seizures reported during the period divided by number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline will be defined as (frequency of convulsive seizures per 28 days during Treatment Period - frequency of convulsive seizures per 28 days at Baseline) divided by the frequency of convulsive seizures per 28 days at Baseline multiplied by 100.
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Timepoint [2]
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Up to 4 years
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Secondary outcome [3]
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Percent Change from Baseline in Major Motor Drop (MMD) Seizure Frequency per 28 Days in LGS Cohort
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Assessment method [3]
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MMD seizure frequency per 28 days is defined as total number of MMD seizures reported during the period divided by number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline will be defined as (frequency of MMD seizures per 28 days during the Treatment Period - frequency of MMD seizures per 28 days at Baseline) divided by the frequency of MMD seizures per 28 days at Baseline multiplied by 100.
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Timepoint [3]
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Up to 4 years
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Secondary outcome [4]
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Clinical Global Impression of Improvement (CGI-I) Score
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Assessment method [4]
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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.
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Timepoint [4]
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Up to 4 years
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Secondary outcome [5]
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Caregiver Global Impression of Improvement (Care GI-I) Score
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Assessment method [5]
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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.
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Timepoint [5]
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Up to 4 years
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Secondary outcome [6]
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CGI-I Seizure Intensity and Duration Score
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Assessment method [6]
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The CGI-I seizure intensity and duration instrument is used by the parent/caregiver to rate improvement in intensity and duration of convulsive seizures (DS Cohort) or MMD seizures (LGS Cohort) from Baseline. The participant's symptoms will be rated on 7-point scale as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). Higher score will indicate worse symptoms.
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Timepoint [6]
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Up to 4 years
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Secondary outcome [7]
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CGI-I Nonseizure Symptoms Score
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Assessment method [7]
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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.
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Timepoint [7]
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Up to 4 years
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Secondary outcome [8]
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Change in Quality of Life Inventory-Disability (QI-Disability) Score
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Assessment method [8]
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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.
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Timepoint [8]
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Up to 4 years
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Eligibility
Key inclusion criteria
1. Participant must have:
* Been previously enrolled in a phase 3 soticlestat clinical study.
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Minimum age
2
Years
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Maximum age
56
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Unstable, clinically significant neurologic (other than the disease being studied), psychiatric, cardiovascular, ophthalmologic, pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, endocrine disease, malignancy including progressive tumors, or other abnormality that may impact the ability to participate in the study or that may potentially confound the study results. It is the responsibility of the investigator to assess the clinical significance; however, consultation with the medical monitor may be warranted.
2. Abnormal and clinically significant ECG abnormality at Visit 1 including QT interval with Fridericia correction method (QTcF) >450 milliseconds (ms) confirmed with a repeat ECG using manual measurement of QTcF.
3. Participant is considered by the investigator to be at imminent risk of suicide or injury to self, others, or property. Participants who have positive answers on item numbers 4 or 5 on the CSSRS before dosing are excluded. This scale will only be administered to participants aged =6 years.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
4/03/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
22/05/2026
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Actual
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Sample size
Target
400
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
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Recruitment hospital [1]
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Sydney Children's Hospital - Randwick
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Queensland Childrens Hospital - Brisbane
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Austin Hospital - Heidelberg
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Alfred Hospital - Melbourne
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2031 - Randwick
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Recruitment postcode(s) [2]
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4101 - Brisbane
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3084 - Heidelberg
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Recruitment postcode(s) [4]
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3004 - Melbourne
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Recruitment outside Australia
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Valencia
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Dnipropetrovs'ka Oblast
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Ukraine
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Ivano-Frankivsk
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Ukraine
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Kyiv
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Takeda
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The main aim of the study is to learn if soticlestat, when given as an add-on therapy, reduces the number of seizures in children and adults with Dravet Syndrome (DS) or Lennox-Gastaut Syndrome (LGS). Participants will receive their standard anti-seizure therapy, plus tablets of soticlestat. There will be scheduled visits and follow-up phone calls throughout the study.
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Trial website
https://clinicaltrials.gov/study/NCT05163314
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Study Director
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Address
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Takeda
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Contact person for public queries
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
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.
Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR)
Query!
When will data be available (start and end dates)?
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Available to whom?
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.
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/takeda/
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT05163314