Continuous Spike and Wave During Sleep, Epileptic Encephalopathy
Conditions
Keywords
Epilepsy, Epileptic Encephalopathy, Neurocrine, NBI-827104, Steamboat 2
Brief summary
The primary objective for this study is to evaluate the long-term safety and tolerability of NBI-827104 in pediatric participants with epileptic encephalopathy with continuous spike-and-wave during sleep (EECSWS).
Detailed description
This study will enroll participants who completed treatment in the Phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel-group Study NBI-827104-CSWS2010 (NCT04625101). Participants who did not participate in Study NBI-827104-CSWS2010 may also be eligible for enrollment.
Interventions
DRUGNBI-827104
T-type calcium channel blocker.
Sponsors
Neurocrine Biosciences
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
4 Years to 12 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: For participants who enroll directly following the completion of the Study NBI-827104-CSWS2010: * Completed 12 weeks of treatment in Study NBI-827104-CSWS2010. For Participants Who Do Not Enroll Directly from or Did Not Participate in Study NBI-827104-CSWS2010: * Have diagnosis of EECSWS confirmed by the Diagnosis Confirmation Panel (DCP). Key
Exclusion criteria
For participants who enroll directly following the completion of the Study NBI-827104-CSWS2010: * Have developed any other disorder for which the treatment takes priority over treatment of EECSWS or is likely to interfere with study treatment or impair treatment compliance. For Participants Who Do Not Enroll Directly from or Did Not Participate in Study NBI-827104-CSWS2010: * Body weight \<15 kg at Day 1. * Clinically relevant findings related to cardiovascular or laboratory parameters at screening as determined by the investigator. * Presence of relevant neurological disorders other than EECSWS and its underlying conditions as judged by the investigator. Symptomatic conditions underlying EECSWS (for example, neonatal strokes) have to be stable for at least 1 year prior to screening. * Planned surgical intervention related to structural abnormalities of the brain from screening through the Week 6 Visit. * Used any active investigational drug other than NBI-827104 in the context of a clinical study within 30 days or 5 half-lives (whichever is longer) before screening or plans to use such an investigational drug (other than NBI-827104) during the study. * Have developed any other disorder for which the treatment takes priority over treatment of EECSWS or is likely to interfere with study treatment or impair treatment compliance.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs) | Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days) | A TEAE is an adverse event (AE) that was not present prior to the initiation of study treatment or was an already present condition that worsened either in intensity or frequency following the initiation of study treatment. |
Countries
Denmark, Spain, Switzerland, United Kingdom, United States
Contacts
STUDY_DIRECTORClinical Development Lead
Neurocrine Biosciences
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 9.04 years STANDARD_DEVIATION 1.78 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 17 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race/Ethnicity, Customized Race Other | 1 Participants |
| Race/Ethnicity, Customized Race White | 18 Participants |
| Sex: Female, Male Female | 9 Participants |
| Sex: Female, Male Male | 10 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 19 |
| other Total, other adverse events | 17 / 19 |
| serious Total, serious adverse events | 2 / 19 |
Outcome results
None listed