A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)

Longboard Pharmaceuticals

Status and phase

Enrolling

Phase 3

Conditions

Dravet Syndrome

Treatments

Drug: Placebo

Drug: LP352

Study type

Interventional

Funder types

Industry

Identifiers

NCT06660394

2024-514937-39-00 (EU Trial (CTIS) Number)

LP352-302

Details and patient eligibility

About

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.

Enrollment

160 estimated patients

Sex

All

Ages

2 to 65 years old

Volunteers

No Healthy Volunteers

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.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Quadruple Blind

160 participants in 2 patient groups, including a placebo group

LP352

Experimental group

Description:

Participants will be titrated up to highest tolerated dose of LP352 during the Titration period (Visit 2 - Visit 5), followed by maintenance period (Visit 5 - Visit 8) and then taper/down titration period.

Treatment:

Drug: LP352

Placebo

Placebo Comparator group

Description:

Placebo for LP352

Treatment:

Drug: Placebo