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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04940624
Registration number
NCT04940624
Ethics application status
Date submitted
16/06/2021
Date registered
25/06/2021
Date last updated
1/05/2024
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
Experimental: Soticlestat - 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.
Placebo Comparator: Placebo - Soticlestat placebo-matching mini-tablets or tablets, orally or via G-tube or MIC-KEY button or J-tube, BID, up to 4 weeks 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.
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 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.
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Timepoint [1]
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Baseline up to Week 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 (EMA Region Specific)
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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 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.
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Timepoint [2]
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Baseline up to Week 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 are defined as those with =50% reduction from baseline in convulsive seizures during the Maintenance Period.
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Timepoint [1]
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Baseline up to Week 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 are defined as those with =50% reduction from baseline in convulsive seizures during the full Treatment Period.
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Timepoint [2]
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Baseline up to Week 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 in a Cumulative Response Curve
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Assessment method [3]
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Timepoint [3]
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Baseline up to Week 16
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Secondary outcome [4]
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Caregiver Global Impression of Improvement (Care GI-I) Score
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Assessment method [4]
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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 [4]
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Baseline up to Week 16
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Secondary outcome [5]
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Clinical Global Impression of Improvement (CGI-I) Score
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Assessment method [5]
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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 [5]
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Baseline up to Week 16
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Secondary outcome [6]
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CGI-I Nonseizure Symptoms Score
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Assessment method [6]
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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 [6]
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Baseline up to Week 16
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Secondary outcome [7]
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Change in Quality of Life Inventory-Disability (QI-Disability) Score
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Assessment method [7]
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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 [7]
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Baseline up to Week 16
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Secondary outcome [8]
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CGI-I Seizure Intensity and Duration Score
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Assessment method [8]
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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.
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Timepoint [8]
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Baseline up to 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 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.
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Timepoint [9]
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Baseline up to Week 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 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 [10]
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Baseline up to Week 16
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Secondary outcome [11]
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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 [11]
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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 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.
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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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Change from Baseline in Percentage of Convulsive Seizure-free Days
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Assessment method [12]
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Convulsive seizure-free days will be defined as number of days a participant remained convulsive seizure free after initiation of the treatment.
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Timepoint [12]
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Baseline up to Week 16
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Secondary outcome [13]
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Longest Convulsive Seizure-free Interval.
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Assessment method [13]
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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.
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Timepoint [13]
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Baseline up to Week 16
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Secondary outcome [14]
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Number of Days When Rescue Antiseizure Medication (ASM) is Used
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Assessment method [14]
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Timepoint [14]
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Baseline up to Week 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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Georgia
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Iowa
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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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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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Guangdong
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China
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Hubei
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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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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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Japan
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Nagasaki
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Niigata
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Osaka
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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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Netherlands
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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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Poznan
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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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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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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/ct2/show/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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Contact person for public queries
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04940624
Download to PDF