Long-Term, Open-Label Extension Study to Evaluate the Safety and Tolerability of NBI-827104 in Pediatric Subjects With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep
Trial Snapshot
- Phase
- Phase 2
- Status
- Terminated
- Sponsor
- Neurocrine Biosciences
- Enrollment
- 19
- Locations
- 12
- Primary Endpoint
- The Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs)
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 4 Years to 12 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
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-CSWS
- •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).
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
- •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.
Arms & Interventions
NBI-827104
NBI-827104 administered orally
Intervention: NBI-827104 (Drug)
Outcomes
Primary Outcomes
The Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs)
Time Frame: 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.
Secondary Outcomes
No secondary outcomes reported