A Study of Soticlestat in Adults and Children With Rare Epilepsies
- Conditions
- Dravet Syndrome (DS)Lennox-Gastaut Syndrome (LGS)Epilepsy
- Interventions
- Registration Number
- NCT03635073
- Lead Sponsor
- Takeda
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 156
-
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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Soticlestat 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.
- Primary Outcome Measures
Name Time Method Percentage of Participants Who Experience At least one Adverse Event (AE) 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 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 Up to 6 years Change from Baseline in Behavioral and Adaptive Functional Measures Using the Vineland Adaptive Behavior Scale (VABS) 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 Up to 6 years
- Secondary Outcome Measures
Name Time Method Percent Change from Baseline in Convulsive Seizure 28-day Frequency (Dravet syndrome [DS] Participants) Up to 6 years Percent Change from Baseline in Drop Seizure 28-day Frequency (Lennox-Gastaut syndrome [LGS] Participants) Up to 6 years Percent Change from Baseline in all Seizure 28-day Frequency Up to 6 years Percent Change from Baseline in Motor Seizure 28-day Frequency Up to 6 years Change from Baseline in Clinician's Clinical Global Impression of Severity (CGI-S) 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).
Trial Locations
- Locations (48)
Hospital For Sick Children
🇨🇦Toronto, Ontario, Canada
Xenosciences Inc
🇺🇸Phoenix, Arizona, United States
Medsol Clinical Research Center Inc
🇺🇸Port Charlotte, Florida, United States
University of South Florida
🇺🇸Tampa, Florida, United States
Pediatric Neurology PA
🇺🇸Winter Park, Florida, United States
Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
David Geffen School of Medicine at UCLA
🇺🇸Los Angeles, California, United States
Colorado Children's Hospital
🇺🇸Aurora, Colorado, United States
Nicklaus Children's Hospital
🇺🇸Miami, Florida, United States
Center for Rare Neurological Diseases
🇺🇸Norcross, Georgia, United States
Ann and Robert H Lurie Childrens Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Bluegrass Epilepsy Research LLC
🇺🇸Lexington, Kentucky, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Mayo Clinic - PIN
🇺🇸Rochester, Minnesota, United States
Minnesota Epilepsy Group PA
🇺🇸Saint Paul, Minnesota, United States
Max Benzaquen, M.D., PC
🇺🇸Chesterfield, Missouri, United States
Northeast Regional Epilepsy Group
🇺🇸Hackensack, New Jersey, United States
Children's Hospital at Saint Peter's University Hospital
🇺🇸New Brunswick, New Jersey, United States
NYU - Ambulatory Care Center (ACC)
🇺🇸New York, New York, United States
Columbia University Medical Center - PIN
🇺🇸New York, New York, United States
Wake Forest Baptist Medical Center - PPDS
🇺🇸Winston-Salem, North Carolina, United States
Medical University of South Carolina Childrens Hospital - PIN
🇺🇸Charleston, South Carolina, United States
Cook Children's Medical Center - Jane and John Justin Neurosciences Center
🇺🇸Fort Worth, Texas, United States
Monash Children's Hospital
🇦🇺Clayton, Victoria, Australia
Austin Hospital
🇦🇺Heidelberg, Victoria, Australia
Shenzhen Children's Hospital
🇨🇳Shenzhen, Guangdong, China
Children's Hospital of Fudan University
🇨🇳Shanghai, Shanghai, China
Peking University First Hospital
🇨🇳Beijing, China
Beijing Children's Hospital,Capital Medical University
🇨🇳Beijing, China
Xiangya Hospital Central South University
🇨🇳Changsha, China
Xiangya Hospital of Central South University
🇨🇳Changsha, China
Tel Aviv Sourasky Medical Center PPDS
🇮🇱Tel Aviv, Israel
Soroka University Medical Centre
🇮🇱Beer Sheva, Israel
Bnai Zion Medical Center
🇮🇱Haifa, Israel
Edith Wolfson Medical Center
🇮🇱Holon, Israel
Schneider Children's Medical Center of Israel - Petah Tikvah - PIN
🇮🇱Petah Tikva, Israel
Sheba Medical Center - PPDS
🇮🇱Ramat-Gan, Israel
Uniwersyteckie Centrum Kliniczne
🇵🇱Gdansk, Pomorskie, Poland
NZOZ Centrum Neurologii Dzieciecej i Leczenia Padaczki
🇵🇱Kielce, Swietokrzyskie, Poland
Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
🇵🇱Poznan, Wielkopolskie, Poland
Centrum Medyczne Plejady
🇵🇱Krakow, Poland
Samodzielny Publiczny Dzieciecy Szpital Kliniczny w Warszawie
🇵🇱Warszawa, Poland
Centro Hospitalar Lisboa Central- Hospital Dona Estefania
🇵🇹Lisboa, Portugal
Centro Hospitalar Lisboa Norte, E.P.E. Hospital de Santa Maria
🇵🇹Lisboa, Portugal
Hospital Ruber Internacional (Grupo Quironsalud)
🇪🇸Madrid, Spain
Clinica Universidad Navarra
🇪🇸Pamplona, Navarra, Spain
Hospital Vithas La Salud
🇪🇸Granada, Spain
Hospital Universitari i Politecnic La Fe de Valencia
🇪🇸Valencia, Spain