SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE) MedDRA version: 20.0 Level: PT Classification code 10077380 Term: Epileptic encephalopathy System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
Neurocrine Biosciences, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Be a male or female 12 to 21 years of age, inclusive. • Have a diagnosis of SCN8A-DEE supported by both clinical and genetic findings • Have on average at least 1 countable motor seizure per week and not be seizure-free for more than 20 consecutive days. • Being treated with at least 1 other ASM, but no more than 4 ASMs. • Have failed to achieve seizure freedom with at least 2 ASMs. • Must be using a nocturnal alerting system or practice consistent with standards of care at the time of screening and continue to use this for the duration of the study. • Must have an adequate rescue medication regimen per the investigator’s judgment in place at the time of screening and for the duration of the study. • Have a body weight of at least 10 kg • The subject's parent/caregiver is able to accurately identify seizure types, especially countable motor seizures and is able to complete seizure diary Are the trial subjects under 18? yes Number of subjects for this age range: 23 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 7 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Have previously been enrolled in this study and received blinded treatment. • Have participated in an interventional clinical trial 450 msec or presence of any significant cardiac abnormality.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To assess the efficacy of NBI-921352 as adjunctive therapy on the frequency of countable motor seizures ;Secondary Objective: • To evaluate the efficacy of NBI-921352 using the Clinical and Parent/Caregiver Global Impression of Change scales and the Clinical and Parent/Caregiver Global Impression of Severity scales. • To characterize the pharmacokinetics of NBI-921352 and determine the effect of NBI-921352 on plasma levels of concomitant ASMs and evaluated metabolites. • To evaluate the safety and tolerability of NBI-921352.;Primary end point(s): Percentage change from baseline in 28-day seizure frequency for countable motor seizures during the treatment period of the study.;Timepoint(s) of evaluation of this end point: Time Frame: Baseline, Treatment Period: Day 1 to Week 16 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Treatment response of = 50% decrease for countable motor seizures • Treatment response of = 25%, = 75%, or 100% decrease in countable motor seizures • Clinical Global Impression of Change (CGIC) at each study visit • Parent/Caregiver Global Impression of Change (GIC) at each study visit • Change from Baseline in Clinical Global Impression of Severity (CGIS) • Change from Baseline in Parent/Caregiver Global Impression of Severity (GIS) Other secondary endpoints (maintenance period): -Percentage Change from Baseline in 28-day Seizure Frequency for countable motor seizures -Treatment response of = 25%, = 50%, = 75%, or 100% decrease for countable motor seizures ;Timepoint(s) of evaluation of this end point: Time Frame: Baseline, Treatment Period: Day 1 to Week 16. Other secondary endpoints (maintenance period): Timeframe: Maintenance Period: Week 7 to Week 16 | — |
Countries
Australia, Belgium, Brazil, Canada, Czechia, Czech Republic, Denmark, France, Germany, Italy, Netherlands, New Zealand, Poland, Portugal, Spain, United Kingdom, United States
Contacts
Public ContactMedical Information Call Center
Neurocrine Biosciences, Inc.
Outcome results
None listed