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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04940624
Registration number
NCT04940624
Ethics application status
Date submitted
16/06/2021
Date registered
25/06/2021
Date last updated
25/03/2025
Titles & IDs
Public title
A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome
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Scientific 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)
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Secondary ID [1]
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jRCT2051210074
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Secondary ID [2]
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TAK-935-3001
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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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Condition category
Condition code
Neurological
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Epilepsy
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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
Treatment: Drugs - Placebo
Placebo comparator: Placebo - Soticlestat placebo-matching mini-tablets or tablets, orally or via gastrostomy tube (G-tube) or low-profile gastric tube (MIC-KEY) button or jejunostomy tube (J-tube), twice daily (BID), up to 4 weeks during titration. Participants continued to receive the soticlestat placebo-matching mini-tablets or tablets for 12 weeks during maintenance. The total duration of the treatment was up to 16 weeks (Full Treatment Period). Soticlestat matching tapering was done to maintain the blind if participants decided to discontinue the treatment.
Experimental: Soticlestat - Participants weighing \<45 kg: Soticlestat, mini-tablets, at the dose of 40 mg to 200 mg, orally or via G-tube or MIC-KEY button or J-tube, BID based on the body weight up to 4 weeks during titration. Participants continued to receive the dose that they were on at the end of the titration, for 12 weeks during maintenance. The total duration of the treatment was up to 16 weeks (Full Treatment Period) with dose tapered down if participants decided to discontinue the treatment.
Participants weighing =45 kg: Soticlestat mini-tablets or tablets with a starting dose of 100 mg BID followed by 200 mg BID and, then 300 mg BID, up to 4 weeks during titration. Participants continued to receive 300 mg BID for 12 weeks during maintenance. The total duration of the treatment was up to 16 weeks (Full Treatment Period) with dose tapered down if participants decided to discontinue the treatment.
Treatment: Drugs: Soticlestat
Soticlestat mini-tablets or tablets.
Treatment: Drugs: Placebo
Soticlestat placebo-matching 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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Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During the Full Treatment Period
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Assessment method [1]
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Convulsive seizure frequency per 28 days was defined as the total number of convulsive seizures reported during the period divided by the number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline was defined as (frequency of seizures per 28 days during the Full Treatment Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100.
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Timepoint [1]
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Baseline; Full Treatment Period: Weeks 1 to 16
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Primary outcome [2]
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Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During the Maintenance Period
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Assessment method [2]
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Convulsive seizure frequency per 28 days was defined as the 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 was 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.
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Timepoint [2]
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Baseline; Maintenance Period: Weeks 5 to 16
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Secondary outcome [1]
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Percentage of Responders During Maintenance Period
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Assessment method [1]
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Responders were defined as those with =50% reduction from Baseline in convulsive seizures during the Maintenance Period. Percentages were rounded off to the nearest single decimal place.
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Timepoint [1]
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Maintenance Period: Weeks 5 to 16
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Secondary outcome [2]
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Percentage of Responders During the Full Treatment Period
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Assessment method [2]
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Responders were defined as those with =50% reduction from Baseline in convulsive seizures during the Full Treatment Period. Percentages were rounded off to the nearest single decimal place.
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Timepoint [2]
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Full Treatment Period: Weeks 1 to 16
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Secondary outcome [3]
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Percentage of Participants With =0%, >0% to =25%, >25% to =50%, >50% to =75%, and >75% to =100% Reduction in Convulsive Seizures During the Full Treatment Period
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Assessment method [3]
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Percent reduction from Baseline (%) was defined as \[(Full Treatment Period Convulsive Seizure Frequency - Baseline Convulsive Seizure Frequency) divided by Baseline Convulsive Seizure Frequency\] multiplied by 100. Data was reported as reduction of =0%, \>0% to =25%, \>25% to =50%, \>50% to =75%, \>75% to =100% or more in seizures from Baseline. Percentages were rounded off to the nearest single decimal place.
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Timepoint [3]
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Full Treatment Period: Weeks 1 to 16
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Secondary outcome [4]
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Percentage of Participants With Caregiver Global Impression of Improvement (Care GI-I) Scale Responses as Per the Parent/Caregiver Reported Impression at Week 16
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Assessment method [4]
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The Care GI-I is a 7-point Likert scale that the caregiver used to rate improvement in overall seizure control, behavior, safety and tolerability after the initiation of study drug relative to Baseline (before treatment with the study drug). The participant was rated as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). The parent/caregiver completed the Care GI-I via interview. Lower scores indicate improvement. Percentages were rounded off to the nearest single decimal place.
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Timepoint [4]
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Week 16
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Secondary outcome [5]
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Percentage of Participants With Clinical Global Impression of Improvement (CGI-I) Scale Responses as Per the Investigator Reported Impression at Week 16
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Assessment method [5]
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The CGI-I (Clinician) is a 7-point Likert scale that the investigator used to rate a participant's change (improvement) in overall seizure control, behavior, safety and tolerability, after the initiation of study drug relative to Baseline (before treatment with the study drug). The participant was rated as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). The investigator or designee completed the CGI-I. Lower scores indicate improvement. Percentages were rounded off to the nearest single decimal place.
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Timepoint [5]
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Week 16
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Secondary outcome [6]
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Percentage of Participants With CGI-I Nonseizure Symptoms Instrument Responses for Each Domain as Per the Investigator Reported Impression at Week 16
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Assessment method [6]
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The CGI-I non-seizure symptoms instrument is a series of single-item assessments that the investigator used to rate improvement in the symptoms and impacts in select non-seizure domains (alertness, communication, and disruptive behaviors) since initiating the study drug. The participant was rated by the investigator for each domain as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). Lower scores indicated improvement. Data for percentage of participants categorized based on the responses for each domain are presented. Percentages were rounded off to the nearest single decimal place.
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Timepoint [6]
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Week 16
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Secondary outcome [7]
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Change From Baseline in Quality of Life Inventory-Disability (QI-Disability) Total Score at Week 16
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Assessment method [7]
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The QI-Disability tool is a parent/caregiver-reported questionnaire that evaluated the quality of life in children with intellectual disabilities. It contains 32 items covering 6 domains of quality of life: physical health, positive emotions, negative emotions, social interaction, leisure and the outdoors, and independence. Each QI-Disability item is rated on a Likert scale of: Never, Rarely, Sometimes, Often, and Very Often. Items were linearly transformed to a scale of 0 to 100, with higher scores representing better quality of life. Domain scores are calculated by averaging item scores. The domain scores are summed and divided by 6 to yield a total score. The total score ranges from 0 to 100, with higher scores indicating a better quality of life. A negative change from Baseline implies deteriorating quality of life. Mixed-effects model for repeated measures (MMRM) was used for analysis.
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Timepoint [7]
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Baseline, Week 16
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Secondary outcome [8]
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Percentage of Participants With CGI-I Seizure Intensity and Duration Instrument Responses as Per the Parent/Caregiver Reported Impression at Week 16
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Assessment method [8]
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The CGI-I seizure intensity and duration instrument was used by the parent/caregiver to rate changes in intensity and/or duration of the most impactful seizures from the first assessment. The participant's symptoms were rated 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). Lower scores indicate improvement. Percentages were rounded off to the nearest single decimal place.
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Timepoint [8]
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Week 16
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Secondary outcome [9]
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Percent Change From Baseline in Frequency of All Seizures Per 28 Days During the Maintenance Period
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Assessment method [9]
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Seizure frequency per 28 days was defined as the total number of seizures reported during the period divided by the number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline was defined as (frequency of seizures per 28 days during the Maintenance Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100.
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Timepoint [9]
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Baseline; Maintenance Period: Weeks 5 to 16
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Secondary outcome [10]
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Percent Change From Baseline in Frequency of All Seizures Per 28 Days During the Full Treatment Period
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Assessment method [10]
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Seizure frequency per 28 days was defined as the 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 was defined as (frequency of seizures per 28 days during the Full Treatment Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100.
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Timepoint [10]
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Baseline; Full Treatment Period: Weeks 1 to 16
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Secondary outcome [11]
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Change From Baseline in Percentage of Convulsive Seizure-free Days During the Full Treatment Period
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Assessment method [11]
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Convulsive seizure-free days was defined as number of days a participant remained convulsive seizure-free after initiation of the treatment. The change from baseline in percentage of convulsive seizure-free days, was defined as the percentage of seizure-free days during the Full Treatment Period minus the percentage of seizure-free days during the Baseline. A linear model with treatment group and age stratum as factors and baseline percentage as a covariate was used for analysis.
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Timepoint [11]
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Baseline up to Week 16
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Secondary outcome [12]
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Longest Convulsive Seizure-free Interval During the Full Treatment Period
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Assessment method [12]
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Longest convulsive seizure-free interval was defined as the longest time period that the participant remained convulsive seizure free after initiation of the treatment. A linear model with treatment group and age stratum as factors was used for analysis.
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Timepoint [12]
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Full Treatment Period: Weeks 1 to 16
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Secondary outcome [13]
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Number of Days When Rescue Antiseizure Medication (ASM) is Used During the Full Treatment Period
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Assessment method [13]
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Use of rescue ASM was recorded in the case report form (CRF) along with start and end date of medication. Based on the start and end dates for all rescue ASMs taken by a participant, the number of days during the Full Treatment Period when rescue ASM was used was derived.
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Timepoint [13]
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Full Treatment Period: Weeks 1 to 16
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Eligibility
Key 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.
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Minimum age
2
Years
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Maximum age
21
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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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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
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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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
Completed
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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
28/10/2021
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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
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Actual
11/04/2024
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Sample size
Target
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Accrual to date
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Final
144
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Recruitment in Australia
Recruitment state(s)
QLD
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Recruitment hospital [1]
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Queensland Childrens Hospital - South Brisbane
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Recruitment postcode(s) [1]
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4101 - South Brisbane
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Recruitment outside Australia
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United States of America
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Arizona
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California
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United States of America
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Georgia
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Iowa
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United States of America
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New York
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Ohio
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South Carolina
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Washington
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Brazil
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Parana
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Brazil
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Rio Grande Do Sul
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Brazil
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Sao Paulo
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Canada
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Alberta
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Canada
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British Columbia
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Canada
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Ontario
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China
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Beijing
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China
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Chongqing
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China
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Guangdong
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China
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Hunan
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China
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Jilin
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China
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Shanghai
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France
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Marseille
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France
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Paris
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Germany
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Bayern
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Germany
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Hessen
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Germany
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Nordrhein-Westfalen
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Greece
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Attiki
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Hungary
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Budapest
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Italy
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Lazio
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Italy
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Lombardia
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Italy
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Toscana
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Aiti
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Japan
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Kumamoto
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Nagasaki
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Niigata
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Osaka
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Japan
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Sizuoka
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Japan
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Tokyo
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Latvia
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Riga
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Noord-Brabant
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Overijssel
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Poland
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Malopolskie
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Mazowieckie
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Gdansk
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Russian Federation
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Moskva
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Russian Federation
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Krasnoyarsk
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Serbia
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Belgrade
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Serbia
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Nis
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Spain
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Barcelona
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Spain
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Malaga
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Spain
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Valencia
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Ukraine
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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 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.
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Trial website
https://clinicaltrials.gov/study/NCT04940624
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Study Director
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Address
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Takeda
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Country
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Phone
0
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Fax
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Email
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Contact person for public queries
Name
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Address
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Phone
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Fax
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Email
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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)
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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
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/24/NCT04940624/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/24/NCT04940624/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT04940624
Download to PDF