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
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).
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