Study to Evaluate NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)
- Conditions
- SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)MedDRA version: 20.0Level: PTClassification code 10077380Term: Epileptic encephalopathySystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2020-003140-83-BE
- Lead Sponsor
- eurocrine Biosciences, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 60
• 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
• Have previously been enrolled in this study and received blinded treatment.
• Have participated in an interventional clinical trial <30 days prior to screening.
• Have symptoms that would be more consistent with another epilepsy disorder such as Dravet syndrome (eg, fever-induced episodes of status epilepticus, frequent myoclonic seizures, worsening on sodium channel blockers, absence seizures with generalized spike-and-wave EEG as the sole seizure type).
• Are currently receiving cannabinoids or medical marijuana except Epidiolex/Epidyolex, unless approved by the Sponsor.
• Are currently taking systemic steroids (excluding inhaled medication for asthma treatments). If subject has received these medications in the past, must be off these medications for at least 3 months prior to the screening visit and these drugs may not be initiated during the duration of the study. Intermittent steroids to treat nonepilepsy related diseases (such as allergies or dermatological conditions) are not exclusionary.
• Have a history of moderate or severe head trauma or other neurological disorders or systemic medical diseases that are, in the investigator’s opinion, likely to affect nervous system functioning.
• Have a clinically significant medical condition or chronic disease that in the opinion of the investigator would preclude the subject from participating in and completing the study or that could confound interpretation of study outcome.
• Have clinically significant abnormal vital signs at the screening visit as determined by the investigator.
• Have one or more clinical laboratory test values outside the reference range, based on blood samples taken at the screening visit, that are of potential risk to the subject’s safety
• Have, at the screening visit, an ECG finding of a corrected QT interval using Fridericia’s formula (QTcF) >450 msec or presence of any significant cardiac abnormality.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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.<br>• To characterize the pharmacokinetics of NBI-921352 and determine the effect of NBI-921352 on plasma levels of concomitant ASMs and evaluated metabolites.<br>• 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;Main Objective: • To assess the efficacy of NBI-921352 as adjunctive therapy on the frequency of countable motor seizures <br>
- Secondary Outcome Measures
Name Time Method Secondary end point(s): • Treatment response of = 50% decrease for countable motor seizures <br>• Treatment response of = 25%, = 75%, or 100% decrease in countable motor seizures<br>• Clinical Global Impression of Change (CGIC) at each study visit <br>• Parent/Caregiver Global Impression of Change (GIC) at each study visit<br>• Change from Baseline in Clinical Global Impression of Severity (CGIS)<br>• Change from Baseline in Parent/Caregiver Global Impression of Severity (GIS)<br><br>Other secondary endpoints (maintenance period):<br>-Percentage Change from Baseline in 28-day Seizure Frequency for countable motor seizures<br>-Treatment response of = 25%, = 50%, = 75%, or 100% decrease for countable motor seizures <br>;Timepoint(s) of evaluation of this end point: Time Frame: Baseline, Treatment Period: Day 1 to Week 16.<br><br>Other secondary endpoints (maintenance period): <br>Timeframe: Maintenance Period: Week 7 to Week 16