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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03635073




Registration number
NCT03635073
Ethics application status
Date submitted
6/08/2018
Date registered
17/08/2018

Titles & IDs
Public title
A Study of Soticlestat in Adults and Children With Rare Epilepsies
Scientific title
A Phase 2, Prospective, Interventional, Open-Label, Multi-Site, Extension Study to Assess the Long-Term Safety and Tolerability of Soticlestat (TAK-935) as Adjunctive Therapy in Subjects With Developmental Epileptic Encephalopathies Including Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome (ENDYMION 1)
Secondary ID [1] 0 0
U1111-1218-5515
Secondary ID [2] 0 0
TAK-935-18-001
Universal Trial Number (UTN)
Trial acronym
Endymion 1
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Epilepsy 0 0
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 - Treatment: Soticlestat, tablets orally twice daily at optimized dose, titrated in up to 2 weeks of Dose Optimization Period, followed by Maintenance Period, which lasts until development is stopped by the sponsor, or the product is approved for marketing, or at any time at the discretion of the sponsor.


Treatment: Drugs: Soticlestat
Soticlestat tablets or mini-tablets.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants Who Experience At least one Adverse Event (AE)
Timepoint [1] 0 0
Up to 6 years
Primary outcome [2] 0 0
Change from Baseline in Behavioral and Adaptive Functional Measures Using the Vineland Adaptive Behavior Scale (VABS)
Timepoint [2] 0 0
Up to 6 years
Primary outcome [3] 0 0
Change from Baseline in Behavior Measures Using Total Scores and Subscale Scores of the Aberrant Behavior Checklist-Community Edition (ABC-C) for Participants =6 Years of age
Timepoint [3] 0 0
Up to 6 years
Primary outcome [4] 0 0
Change from Baseline in the Columbia-Suicide Severity Rating Scale (C-SSRS) Categorization Based on Columbia Classification Algorithm of Suicide Assessment Categories 1,2,3,4, and 5 for Participants =6 Years of age
Timepoint [4] 0 0
Up to 6 years
Secondary outcome [1] 0 0
Percent Change from Baseline in all Seizure 28-day Frequency
Timepoint [1] 0 0
Up to 6 years
Secondary outcome [2] 0 0
Percent Change from Baseline in Drop Seizure 28-day Frequency (Lennox-Gastaut syndrome [LGS] Participants)
Timepoint [2] 0 0
Up to 6 years
Secondary outcome [3] 0 0
Percent Change from Baseline in Convulsive Seizure 28-day Frequency (Dravet syndrome [DS] Participants)
Timepoint [3] 0 0
Up to 6 years
Secondary outcome [4] 0 0
Percent Change from Baseline in Motor Seizure 28-day Frequency
Timepoint [4] 0 0
Up to 6 years
Secondary outcome [5] 0 0
Change from Baseline in Clinician's Clinical Global Impression of Severity (CGI-S)
Timepoint [5] 0 0
Up to 6 years

Eligibility
Key inclusion criteria
1. Participants must have participated in a previous soticlestat study and meet one of the following conditions:

* Successfully completed a soticlestat clinical study.
* Received at least 10 weeks of treatment with the study drug in an antecedent placebo-controlled blinded soticlestat clinical study and the participant did not have a serious or severe AE that, in the investigator's or sponsor's opinion, was related to the study drug and would make it unsafe for the participant to continue receiving the study drug.
2. In the opinion of the investigator, the participant has the potential to benefit from the administration of soticlestat
Minimum age
2 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Clinically significant disease, that, in the investigator's opinion, precludes study participation.
2. Enrollment in any other clinical trial involving an investigational drug, device, or treatment in the past 90 days (with the exception of an antecedent study involving Soticlestat).
3. Participant is currently pregnant or breastfeeding or is planning to become pregnant during the study or within 30 days of the last study drug administration.
4. Suicide attempt within the last year, at significant risk of suicide (either in the opinion of the investigator or defined as 'yes' to suicidal ideation question 4 or 5 on the C-SSRS at Screening) or appearing suicidal per investigator judgment.

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
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 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/07/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
22/05/2026
Actual
Sample size
Target
Accrual to date
Final
156
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Monash Children's Hospital - Clayton
Recruitment hospital [2] 0 0
Austin Hospital - Heidelberg West
Recruitment hospital [3] 0 0
Austin Hospital - Heidelberg
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment postcode(s) [2] 0 0
3081 - Heidelberg West
Recruitment postcode(s) [3] 0 0
3084 - Heidelberg
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
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Kentucky
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Minnesota
Country [10] 0 0
United States of America
State/province [10] 0 0
Missouri
Country [11] 0 0
United States of America
State/province [11] 0 0
New Jersey
Country [12] 0 0
United States of America
State/province [12] 0 0
New York
Country [13] 0 0
United States of America
State/province [13] 0 0
North Carolina
Country [14] 0 0
United States of America
State/province [14] 0 0
South Carolina
Country [15] 0 0
United States of America
State/province [15] 0 0
Texas
Country [16] 0 0
Canada
State/province [16] 0 0
Ontario
Country [17] 0 0
China
State/province [17] 0 0
Guangdong
Country [18] 0 0
China
State/province [18] 0 0
Shanghai
Country [19] 0 0
China
State/province [19] 0 0
Beijing
Country [20] 0 0
China
State/province [20] 0 0
Changsha
Country [21] 0 0
Israel
State/province [21] 0 0
Tel-Aviv
Country [22] 0 0
Israel
State/province [22] 0 0
Be'er Sheva
Country [23] 0 0
Israel
State/province [23] 0 0
Beer Sheva
Country [24] 0 0
Israel
State/province [24] 0 0
Haifa
Country [25] 0 0
Israel
State/province [25] 0 0
Holon
Country [26] 0 0
Israel
State/province [26] 0 0
Petach Tikva
Country [27] 0 0
Israel
State/province [27] 0 0
Petah Tikva
Country [28] 0 0
Israel
State/province [28] 0 0
Ramat-Gan
Country [29] 0 0
Israel
State/province [29] 0 0
Tel Aviv
Country [30] 0 0
Poland
State/province [30] 0 0
Pomorskie
Country [31] 0 0
Poland
State/province [31] 0 0
Swietokrzyskie
Country [32] 0 0
Poland
State/province [32] 0 0
Wielkopolskie
Country [33] 0 0
Poland
State/province [33] 0 0
Krakow
Country [34] 0 0
Poland
State/province [34] 0 0
Warsaw
Country [35] 0 0
Poland
State/province [35] 0 0
Warszawa
Country [36] 0 0
Portugal
State/province [36] 0 0
Lisboa
Country [37] 0 0
Spain
State/province [37] 0 0
Madrid, Communidad Delaware
Country [38] 0 0
Spain
State/province [38] 0 0
Navarra
Country [39] 0 0
Spain
State/province [39] 0 0
Granada
Country [40] 0 0
Spain
State/province [40] 0 0
Madrid
Country [41] 0 0
Spain
State/province [41] 0 0
Valencia

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
Address 0 0
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/NCT03635073