Study of NBI-827104 versus placebo in pediatric subjects with EECSWS
Research type
Research Study
Full title
Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-827104 in Pediatric Subjects with Epileptic Encephalopathy with Continuous Spike-and-Wave During Sleep
IRAS ID
301382
Contact name
Suresh Pujar
Contact email
Sponsor organisation
Neurocrine Biosciences, Inc.
Eudract number
2020-003141-11
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 8 months, 9 days
Research summary
Summary of Research
Epileptic Encephalopathy with Continuous Spike-and-Wave During Sleep (EECSWS) is a spectrum of epileptic conditions that include electroclinical seizures, sleep potentiation of epileptiform activity, i.e. an abnormal spike and wave activity on the EEG with sleep, developmental delay/regression, and an age-related evolution of condition with onset in early childhood and spontaneous improvement before puberty. Although spontaneous improvement before puberty is observed with EECSWS, neurocognitive regression can remain. The potentiation of epileptiform activity during sleep leads to an electroencephalogram (EEG) pattern of electrical status epilepticus during sleep (ESES), which consists of abnormal spike-waves during non-rapid eye movement (NREM) sleep and has been suggested to interfere with normal recuperation functions of sleep, thereby adversely affecting learning abilities, language, memory, and other cognitive domains. Abnormal T-type calcium channel oscillations have been implicated in the pathophysiology of idiopathic generalized epilepsy (IGE) and spike-wave discharges, i.e. abnormal activity at a channel located on neurons resulting in a higher likelihood of developing those two conditions. NBI-827104 is a novel selective and orally available T-type calcium channel blocker which effectively crosses the blood-brain barrier and is being evaluated for the treatment of EECSWS.
NBI-827104-CSWS2010 is a Phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel-group study designed to assess the efficacy, safety, tolerability, and pharmacokinetics of NBI-827104 versus placebo administered once daily in approximately 24 pediatric subjects aged 4 to 12 years (inclusive) with EECSWS.NBI-827104 is being evaluated as an add-on therapy and all subjects, regardless of treatment randomization, are required to be receiving at least 1 anti-epileptic drug (AED) throughout the study.Summary of Results
Study results are posted on clinicaltrialsregister.eu
REC name
London - West London & GTAC Research Ethics Committee
REC reference
21/LO/0644
Date of REC Opinion
16 Nov 2021
REC opinion
Further Information Favourable Opinion