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.
Participants with seizure onset age >1 and <20 months
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 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.
Parallel
LP352
DrugLP352 will be administered orally or through G-tube/ percutaneous endoscopic gastrostomy (PEG) tube
Placebo
DrugParticipants will be administered with matching placebo orally or through G-tube/ PEG tube
Frequency Percent Change in Countable Motor Seizures During Treatment Compared to Baseline
The percent change from Baseline in countable motor seizure frequency during Treatment will be calculated as countable motor seizure frequency during Treatment minus countable motor seizure frequency during Screening and divided by seizure frequency during Screening and multiplied by 100 where each seizure frequency will be based on number of seizures.
Safety and Tolerability of LP352
Safety and tolerability as measured by incidence and severity of non-serious Treatment Emergent Adverse Events (TEAEs), Serious Adverse events (SAEs), AEs leading to discontinuation and clinically significant changes in laboratory parameters (hematology, serum chemistry and Urinalysis), physical examination findings, vital signs, growth parameters (height and weight), 12-lead electrocardiograms (ECGs), C-SSRS responses, and PHQ-9 total score and Question 9 score.
Percentage of participants with ≥ 50% Reduction in countable motor seizures during Treatment compared to Baseline
Frequency Percent Change in Countable Motor Seizures during Maintenance compared to Baseline
Click on the lead sponsor to view all of their trials.