A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathies (DEE)

Launched by LONGBOARD PHARMACEUTICALS · Dec 2, 2024

Keywords

ClinConnect Summary

This clinical trial, called the DEEp OCEAN Study, is looking at a new medication called LP352 to see if it can help control seizures in children and adults who have developmental and epileptic encephalopathy (DEE), including a specific type known as Lennox-Gastaut Syndrome (LGS). The study will take about 24 months and has several phases, including screening, adjusting medication, and ongoing maintenance, followed by a tapering off period. Participants will be randomly assigned to receive either the medication or a placebo (a pill with no active ingredients) to compare the effects.

To be eligible for this trial, participants should be between the ages of 2 and 66, have a history of seizures starting at a young age, and experience multiple types of seizures. They should also have been on stable doses of anti-seizure medications for at least four weeks prior to joining the study. Throughout the trial, participants will need to keep track of their seizures and attend regular check-ins with the research team. It's important to note that certain conditions, like Dravet Syndrome or certain neurological disorders, would exclude someone from participating. This study aims to find out if LP352 is safe and effective in treating these challenging conditions, potentially offering new hope for those affected.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• * Participants who are characterized as having Lennox-Gastaut Syndrome (LGS) must fulfill all of the following criteria:
• • Onset of seizures at ≤8 years old
• • History of tonic/tonic-atonic seizures plus at least 1 of the following seizure type(s): atypical absence, atonic, myoclonic, focal impaired awareness, generalized tonic-clonic, nonconvulsive status epilepticus, or epileptic spasms
• • Presence of developmental plateauing or regression
• • History of electroencephalogram (EEG) showing generalized slow (\<2.5 Hertz \[Hz\]) spike-and-wave complexes
• * Participants who are characterized as having DEE (Other) must fulfill all of the following criteria:
• • Does not meet criteria for LGS
• • Onset of seizures at ≤5 years old
• • Presence of developmental plateauing or regression
• • History of multiple seizure types
• • History of interictal EEG background showing diffuse or multifocal slowing (with or without epileptiform activity)
• • 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 each of 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; in instances where the participant is unable to provide consent, an appropriate legal representative.
• Exclusion Criteria:
• • The participant has a diagnosis of Dravet Syndrome (DS) or has a mutation of the Sodium channel protein type 1 subunit alpha (SCN1A) gene consistent with DS.
• • 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 of 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 unable or unwilling to comply with any of the study requirements or timelines.