A Study of Soticlestat in Adults and Children With Rare Epilepsies (Endymion 1)
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|ClinicalTrials.gov Identifier: NCT03635073|
Recruitment Status : Active, not recruiting
First Posted : August 17, 2018
Last Update Posted : May 13, 2022
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The main aim is to assess the long-term safety and tolerability of soticlestat when used along with other anti-seizure treatment.
Participants will receive soticlestat twice a day. Participants will visit the study clinic every 2-6 months throughout the study.
Study treatments may continue as long as the participant is receiving benefit from it.
|Condition or disease||Intervention/treatment||Phase|
|Epilepsy Dravet Syndrome (DS) Lennox-Gastaut Syndrome (LGS)||Drug: Soticlestat||Phase 2|
The drug being tested in this study is called soticlestat (TAK-935). This global, open-label extension (OLE) study will assess the long-term safety and tolerability of soticlestat in participants with developmental and epileptic encephalopathy (DEE) who participated in previous short-term efficacy/safety studies of soticlestat. All participants will receive Soticlestat treatment.
Participants who rollover from previous blinded study will undergo up to 2 weeks of Dose Optimization Period (depending on the previous study) followed by Maintenance Period. Participants who rollover from an open-label study will continue on their current dose until development is stopped by the sponsor, or the product is approved for marketing, or at any time at the discretion of the sponsor. There will be a 4-week Safety Follow-up Period after the last dose in Maintenance Period, including a 2-week dose Tapering Period.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||156 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official 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)|
|Actual Study Start Date :||July 19, 2018|
|Estimated Primary Completion Date :||November 21, 2024|
|Estimated Study Completion Date :||November 21, 2024|
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.
Soticlestat tablets or mini-tablets.
Other Name: TAK-935
- Percentage of Participants Who Experience At least one Adverse Event (AE) [ Time Frame: Up to 6 years ]An Adverse Event (AE) is defined any untoward medical occurrence in a subject or clinical study subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable or unintended sign (for example, an abnormal laboratory finding, vital sign or ECG evaluation), symptom, or disease temporally associated with the use of a medicinal product, whether considered related to this medicinal product.
- Change from Baseline in Behavioral and Adaptive Functional Measures Using the Vineland Adaptive Behavior Scale (VABS) [ Time Frame: Up to 6 years ]The VABS, 3rd edition, Parent Caregiver Form (VABS-3 Parent Caregiver Form), is a parent-report questionnaire of adaptive functioning or how an individual behaves in their day-to-day life at home and in the community.
- 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 [ Time Frame: Up to 6 years ]
- 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 [ Time Frame: Up to 6 years ]
- Percent Change from Baseline in all Seizure 28-day Frequency [ Time Frame: Up to 6 years ]
- Percent Change from Baseline in Drop Seizure 28-day Frequency (Lennox-Gastaut syndrome [LGS] Participants) [ Time Frame: Up to 6 years ]
- Percent Change from Baseline in Convulsive Seizure 28-day Frequency (Dravet syndrome [DS] Participants) [ Time Frame: Up to 6 years ]
- Percent Change from Baseline in Motor Seizure 28-day Frequency [ Time Frame: Up to 6 years ]
- Change from Baseline in Clinician's Clinical Global Impression of Severity (CGI-S) [ Time Frame: Up to 6 years ]CGI-S is used to obtain an assessment of symptom severity, focusing on clinicians' observations of the subject's current cognitive, functional, and behavioral performance. The CGI-S is rated on a 7-point scale, with the severity of illness scale using a range of responses from 1 (normal) through to 7 (among the most severely ill participants).
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|Ages Eligible for Study:||2 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
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.
- In the opinion of the investigator, the participant has the potential to benefit from the administration of soticlestat
- Clinically significant disease, that, in the investigator's opinion, precludes study participation.
- 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).
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03635073
|Other Study ID Numbers:||
U1111-1218-5515 ( Other Identifier: WHO )
2018-002485-39 ( EudraCT Number )
|First Posted:||August 17, 2018 Key Record Dates|
|Last Update Posted:||May 13, 2022|
|Last Verified:||May 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Yes|
|Plan Description:||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.|
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
|Access Criteria:||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.|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
|Product Manufactured in and Exported from the U.S.:||No|
Lennox Gastaut Syndrome
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn