Clinical trial NCT05301894: Extension Study to Evaluate NBI-827104 in Pediatric Participants With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep
Official title: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Epileptic Encephalopathy
Continuous Spike and Wave During Sleep
Keywords: Epilepsy
Epileptic Encephalopathy
Neurocrine
NBI-827104
Steamboat 2
Lead sponsor: Neurocrine Biosciences
Sponsor class: INDUSTRY
First posted: 2022-03-31
Enrollment: 19
Sex: ALL
Minimum age (years): 4
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility 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.

Primary outcomes:
measure: The Occurrence of Serious Treatment-emergent Adverse Events (TEAEs)
timeFrame: Day 1 up to 238 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Orange
country: United States
facility: Neurocrine Clinical Site
state: California
city: Aurora
country: United States
facility: Neurocrine Clinical Site
state: Colorado
city: Washington
country: United States
facility: Neurocrine Clinical Site
state: District of Columbia
city: Miami
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Rochester
country: United States
facility: Neurocrine Clinical Site
state: Minnesota
city: Durham
country: United States
facility: Neurocrine Clinical Site
state: North Carolina
city: Cleveland
country: United States
facility: Neurocrine Clinical Site
state: Ohio
city: Dianalund
country: Denmark
facility: Neurocrine Clinical Site
city: Barcelona
country: Spain
facility: Neurocrine Clinical Site
city: Madrid
country: Spain
facility: Neurocrine Clinical Site
city: Zürich
country: Switzerland
facility: Neurocrine Clinical Site
city: London
country: United Kingdom
facility: Neurocrine Clinical Site
Study officials:
affiliation: Neurocrine Biosciences
name: Clinical Development Lead
role: STUDY_DIRECTOR
References: