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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

Titles & IDs
Public title
A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome
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)
Secondary ID [1] 0 0
jRCT2051210074
Secondary ID [2] 0 0
TAK-935-3001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Dravet Syndrome (DS) 0 0
Condition category
Condition code
Neurological 0 0 0 0
Epilepsy
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percent Change From Baseline in Convulsive Seizure Frequency per 28 days During the Full Treatment Period
Timepoint [1] 0 0
Baseline up to Week 16
Primary outcome [2] 0 0
Percent Change From Baseline in Convulsive Seizure Frequency per 28 days During the Maintenance Period (EMA Region Specific)
Timepoint [2] 0 0
Baseline up to Week 16
Secondary outcome [1] 0 0
Percentage of Responders During Maintenance Period
Timepoint [1] 0 0
Baseline up to Week 16
Secondary outcome [2] 0 0
Percentage of Responders During the Full Treatment Period
Timepoint [2] 0 0
Baseline up to Week 16
Secondary outcome [3] 0 0
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
Timepoint [3] 0 0
Baseline up to Week 16
Secondary outcome [4] 0 0
Caregiver Global Impression of Improvement (Care GI-I) Score
Timepoint [4] 0 0
Baseline up to Week 16
Secondary outcome [5] 0 0
Clinical Global Impression of Improvement (CGI-I) Score
Timepoint [5] 0 0
Baseline up to Week 16
Secondary outcome [6] 0 0
CGI-I Nonseizure Symptoms Score
Timepoint [6] 0 0
Baseline up to Week 16
Secondary outcome [7] 0 0
Change in Quality of Life Inventory-Disability (QI-Disability) Score
Timepoint [7] 0 0
Baseline up to Week 16
Secondary outcome [8] 0 0
CGI-I Seizure Intensity and Duration Score
Timepoint [8] 0 0
Baseline up to Week 16
Secondary outcome [9] 0 0
Percent Change From Baseline in Frequency of all Seizures per 28 Days During the Maintenance Period
Timepoint [9] 0 0
Baseline up to Week 16
Secondary outcome [10] 0 0
Percent Change From Baseline in Frequency of all Seizures per 28 Days During the Full Treatment Period
Timepoint [10] 0 0
Baseline up to Week 16
Secondary outcome [11] 0 0
Percent Change From Baseline in Convulsive Seizure Frequency per 28 days During the Maintenance Period
Timepoint [11] 0 0
Baseline up to Week 16
Secondary outcome [12] 0 0
Change from Baseline in Percentage of Convulsive Seizure-free Days
Timepoint [12] 0 0
Baseline up to Week 16
Secondary outcome [13] 0 0
Longest Convulsive Seizure-free Interval.
Timepoint [13] 0 0
Baseline up to Week 16
Secondary outcome [14] 0 0
Number of Days When Rescue Antiseizure Medication (ASM) is Used
Timepoint [14] 0 0
Baseline up to Week 16

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.
Minimum age
2 Years
Maximum age
21 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
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
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/10/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
11/04/2024
Sample size
Target
Accrual to date
Final
144
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Queensland Childrens Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
4101 - South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Iowa
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
United States of America
State/province [7] 0 0
South Carolina
Country [8] 0 0
United States of America
State/province [8] 0 0
Washington
Country [9] 0 0
Brazil
State/province [9] 0 0
Parana
Country [10] 0 0
Brazil
State/province [10] 0 0
Rio Grande Do Sul
Country [11] 0 0
Brazil
State/province [11] 0 0
Sao Paulo
Country [12] 0 0
Canada
State/province [12] 0 0
Alberta
Country [13] 0 0
Canada
State/province [13] 0 0
British Columbia
Country [14] 0 0
Canada
State/province [14] 0 0
Ontario
Country [15] 0 0
China
State/province [15] 0 0
Beijing
Country [16] 0 0
China
State/province [16] 0 0
Chongqing
Country [17] 0 0
China
State/province [17] 0 0
Guangdong
Country [18] 0 0
China
State/province [18] 0 0
Hubei
Country [19] 0 0
China
State/province [19] 0 0
Hunan
Country [20] 0 0
China
State/province [20] 0 0
Jilin
Country [21] 0 0
China
State/province [21] 0 0
Shanghai
Country [22] 0 0
France
State/province [22] 0 0
Marseille
Country [23] 0 0
France
State/province [23] 0 0
Paris
Country [24] 0 0
Germany
State/province [24] 0 0
Bayern
Country [25] 0 0
Germany
State/province [25] 0 0
Hessen
Country [26] 0 0
Germany
State/province [26] 0 0
Nordrhein-Westfalen
Country [27] 0 0
Greece
State/province [27] 0 0
Attiki
Country [28] 0 0
Hungary
State/province [28] 0 0
Budapest
Country [29] 0 0
Italy
State/province [29] 0 0
Lazio
Country [30] 0 0
Italy
State/province [30] 0 0
Lombardia
Country [31] 0 0
Italy
State/province [31] 0 0
Toscana
Country [32] 0 0
Japan
State/province [32] 0 0
Aiti
Country [33] 0 0
Japan
State/province [33] 0 0
Kumamoto
Country [34] 0 0
Japan
State/province [34] 0 0
Nagasaki
Country [35] 0 0
Japan
State/province [35] 0 0
Niigata
Country [36] 0 0
Japan
State/province [36] 0 0
Osaka
Country [37] 0 0
Japan
State/province [37] 0 0
Sizuoka
Country [38] 0 0
Japan
State/province [38] 0 0
Tokyo
Country [39] 0 0
Latvia
State/province [39] 0 0
Riga
Country [40] 0 0
Netherlands
State/province [40] 0 0
Noord-Brabant
Country [41] 0 0
Netherlands
State/province [41] 0 0
Overijssel
Country [42] 0 0
Poland
State/province [42] 0 0
Malopolskie
Country [43] 0 0
Poland
State/province [43] 0 0
Mazowieckie
Country [44] 0 0
Poland
State/province [44] 0 0
Gdansk
Country [45] 0 0
Poland
State/province [45] 0 0
Poznan
Country [46] 0 0
Russian Federation
State/province [46] 0 0
Moskva
Country [47] 0 0
Russian Federation
State/province [47] 0 0
Krasnoyarsk
Country [48] 0 0
Serbia
State/province [48] 0 0
Belgrade
Country [49] 0 0
Serbia
State/province [49] 0 0
Nis
Country [50] 0 0
Spain
State/province [50] 0 0
Barcelona
Country [51] 0 0
Spain
State/province [51] 0 0
Malaga
Country [52] 0 0
Spain
State/province [52] 0 0
Valencia
Country [53] 0 0
Ukraine
State/province [53] 0 0
Dnipropetrovs'ka Oblast
Country [54] 0 0
Ukraine
State/province [54] 0 0
Ivano-Frankivsk
Country [55] 0 0
Ukraine
State/province [55] 0 0
Kyiv

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Takeda
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Takeda
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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)
When will data be available (start and end dates)?
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.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/takeda/


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/NCT04940624