Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS)
Conditions
Interventions
Soticlestat will be available as yellow-red colored, film-coated tablets and
mini-tablets. Soticlestat (tablets/mini-tablets) can be swallowed whole or can
be crushed and mixed well in applesauce or
Sponsors
Takeda
Eligibility
Age
2 Years to 64 Years
Inclusion criteria
Inclusion criteria: - Pediatric and adult subjects with DS or LGS from antecedent soticlestat phase 3 clinical studies; - received at least 12 weeks of treatment (combined titration and Maintenance Period) with soticlestat or placebo in the antecedent study; - did not have a serious or severe adverse event (AE) that, in the investigator*s or sponsor*s opinion, was related to the study drug and would make it unsafe for the subject to continue receiving the study drug; and - in the opinion of the investigator, have the potential to benefit from the administration of soticlestat.
Exclusion criteria
Exclusion criteria: - Unstable, clinically significant neurologic (other than DS or LGS), 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. - Abnormal and clinically significant electrocardiogram (ECG) abnormality at Visit 1, including QT interval with Fridericia correction method (QTcF) >450 ms. - Currently pregnant or breastfeeding or is planning to become pregnant within 30 days of the last dose of study drug. - Considered by the investigator to be at imminent risk of suicide or injury to self, others, or property, or has positive answers on item numbers 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS) at Visit 1.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoints are for safety and include the following: - Incidence of treatment-emergent AEs. - Incidence of abnormal values for clinical laboratory tests and ECG evaluations. - Change from baseline in clinical laboratory test values, vital signs, C-SSRS, and ECG parameters. - Change from baseline in height and weight for all age groups. - Absolute value for Tanner stage for children 6 to 17 years of age during the study. - Absolute values for IGF-1 for children 2 to 17 years of age during the study. | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary endpoints include the following: - Percent change from baseline in total seizure frequency per 28 days (DS and LGS) cohort. - Percent change from baseline in convulsive seizure frequency (DS) per 28 days. - Percent change from baseline in MMD seizure frequency (LGS) per 28 days. - Effect on the CGI-I and Care GI-I. - Effect on CGI-I Seizure Intensity and Duration. - Effect on the CGI-I Nonseizure Symptoms completed by clinician with input from the caregivers. - Effect on QI-Disability. | — |
Countries
Netherlands
Outcome results
None listed