a phase 3, placebo-controlled study to investigate lp352 in children and adults with dravet syndrome (ds)

Conditions: Dravet Syndrome

Summary: this (deep sea study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of lp352 in the treatment of seizures in children and adults with ds. the study consists of 3 main phases: screening, titration period, and maintenance period, followed by a taper period and follow-up. participants will be randomized to lp352 or placebo. the total duration of the study will be approximately 24 months.


Eligibility criteria:
inclusion criteria: * diagnosis of ds must fulfill all of the following criteria: 1. participants with seizure onset age \>1 and \<20 months 2. the participant has a history of at least 1 of the following seizure type(s): prolonged generalized tonic-clonic, hemiclonic, myoclonic, tonic, atonic, atypical absence, focal impaired awareness, nonconvulsive status epilepticus * the participant has a current occurrence of at least 1 of the following countable motor seizure types: generalized tonic-clonic, tonic (bilateral), clonic (bilateral), atonic (bilateral) with truncal/leg involvement, focal motor (including hemiclonic), and focal to bilateral tonic-clonic * the participant has demonstrated an average of at least 4 countable motor seizures per month for the 3 months prior to screening. * the participant has been taking 1 to 4 antiseizure medications (asms) at a stable dose for at least 4 weeks prior to screening. * the participant, parent, or caregiver is willing and able (in the judgment of the investigator) to comply with completion of the diaries throughout the study. * the participant must be willing and able to provide written informed consent. exclusion criteria: * the participant has a history of infantile/epileptic spasms. * the participant has been admitted to a medical facility for treatment of status epilepticus requiring mechanical ventilation within 3 months prior to screening. * the participant has a neurodegenerative disorder as indicated by magnetic resonance imaging or genetic testing. * the participant has an acquired lesion/injury unrelated to the primary etiology that could contribute as a secondary cause of seizures. * the participant is receiving exclusionary medications. * the participant has used any cannabis product or cannabidiol that is not in oral solution/capsule/tablet form, not obtained from a government-approved dispensary, or contains ≥50% delta-9-tetrahydrocannabinol (thc). * the participant has unstable, clinically significant neurologic (other than the disease being studied, eg, recurrent strokes), psychiatric, cardiovascular (eg, pulmonary arterial hypertension, cardiac valvulopathy, orthostatic hypotension/tachycardia), pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, or endocrine disease or other abnormality which may impact the ability of the participant to participate or potentially confound the study results. * the participant is unwilling to comply with any of the study requirements or timelines.

Keywords: antiseizure medication epilepsy neurodevelopmental disorders developmental and epileptic encephalopathy lp352 seizures deep sea bexicaserin