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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
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)
Secondary ID [1] 0 0
2021-002482-17
Secondary ID [2] 0 0
TAK-935-3003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Dravet Syndrome (DS) 0 0
Lennox Gastaut Syndrome (LGS) 0 0
Condition category
Condition code
Neurological 0 0 0 0
Epilepsy
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
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

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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With At Least One Treatment Emergent Adverse Event (TEAE)
Timepoint [1] 0 0
Up to 4 years
Primary outcome [2] 0 0
Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS) Score
Timepoint [2] 0 0
Up to 4 years
Primary outcome [3] 0 0
Change from Baseline in Body Weight for All Age Groups
Timepoint [3] 0 0
Up to 4 years
Primary outcome [4] 0 0
Change from Baseline in Height for All Age Groups
Timepoint [4] 0 0
Up to 4 years
Primary outcome [5] 0 0
Absolute Value for Tanner Stage for Children 6 to 17 Years of Age During the Study
Timepoint [5] 0 0
Up to 4 years
Primary outcome [6] 0 0
Absolute Value for Insulin-like Growth Factor 1 (IGF-1) for Children 2 to 17 Years of Age During the Study
Timepoint [6] 0 0
Up to 4 years
Secondary outcome [1] 0 0
Percent Change from Baseline in Total Seizure Frequency per 28 Days for DS and LGS Participants
Timepoint [1] 0 0
Up to 4 years
Secondary outcome [2] 0 0
Percent Change from Baseline in Convulsive Seizure Frequency per 28 Days in DS Cohort
Timepoint [2] 0 0
Up to 4 years
Secondary outcome [3] 0 0
Percent Change from Baseline in Major Motor Drop (MMD) Seizure Frequency per 28 Days in LGS Cohort
Timepoint [3] 0 0
Up to 4 years
Secondary outcome [4] 0 0
Clinical Global Impression of Improvement (CGI-I) Score
Timepoint [4] 0 0
Up to 4 years
Secondary outcome [5] 0 0
Caregiver Global Impression of Improvement (Care GI-I) Score
Timepoint [5] 0 0
Up to 4 years
Secondary outcome [6] 0 0
CGI-I Seizure Intensity and Duration Score
Timepoint [6] 0 0
Up to 4 years
Secondary outcome [7] 0 0
CGI-I Nonseizure Symptoms Score
Timepoint [7] 0 0
Up to 4 years
Secondary outcome [8] 0 0
Change in Quality of Life Inventory-Disability (QI-Disability) Score
Timepoint [8] 0 0
Up to 4 years

Eligibility
Key inclusion criteria
1. Participant must have:

* Been previously enrolled in a phase 3 soticlestat clinical study.
Minimum age
2 Years
Maximum age
56 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.
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
4/03/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
22/05/2026
Actual
Sample size
Target
400
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [2] 0 0
Queensland Childrens Hospital - Brisbane
Recruitment hospital [3] 0 0
Austin Hospital - Heidelberg
Recruitment hospital [4] 0 0
Alfred Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
4101 - Brisbane
Recruitment postcode(s) [3] 0 0
3084 - Heidelberg
Recruitment postcode(s) [4] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
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United States of America
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California
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Colorado
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Florida
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United States of America
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Georgia
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United States of America
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Iowa
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United States of America
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Maryland
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Minnesota
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New Jersey
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New York
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Ohio
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Oregon
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Pennsylvania
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South Carolina
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Texas
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Utah
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United States of America
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Washington
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Belgium
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Antwerpen
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Belgium
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Brabant Wallon
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Belgium
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Brussels
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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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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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Hubei
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China
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Hunan
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China
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Jiangxi
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China
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Jilin
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China
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Shanghai
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Paris
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Germany
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Baden-Wurttemberg
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Germany
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Hessen
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Nordrhein-Westfalen
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Germany
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Sachsen
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Greece
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Attiki
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Greece
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Larisa
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Greece
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Thessaloniki
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Hungary
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Baranya
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Hungary
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Budapest
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Italy
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Lazio
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Lombardia
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Toscana
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Japan
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Aiti
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Hukuoka
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Kanagawa
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Kumamoto
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Japan
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Nagasaki
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Japan
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Niigata
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Okayama
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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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Mexico
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Jalisco
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Netherlands
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Noord-Brabant
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Netherlands
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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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Poland
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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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Ekaterinburg
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Russian Federation
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Krasnoyarsk
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Russian Federation
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Moscow
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Russian Federation
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Tyumen'
Country [73] 0 0
Serbia
State/province [73] 0 0
Belgrade
Country [74] 0 0
Serbia
State/province [74] 0 0
Nis
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Serbia
State/province [75] 0 0
Novi Sad
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Spain
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Navarra
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Spain
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Almeria
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Spain
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Barcelona
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Spain
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Granada
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Spain
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Sevilla
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Spain
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Valencia
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Ukraine
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Dnipropetrovs'ka Oblast
Country [83] 0 0
Ukraine
State/province [83] 0 0
Ivano-Frankivsk
Country [84] 0 0
Ukraine
State/province [84] 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
Takeda Contact
Address 0 0
Country 0 0
Phone 0 0
+1-877-825-3327
Fax 0 0
Email 0 0
medinfoUS@takeda.com
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/NCT05163314