Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies
- Conditions
- Developmental and Epileptic EncephalopathyLennox Gastaut SyndromeDravet Syndrome
- Interventions
- Drug: Placebo
- Registration Number
- NCT05364021
- Lead Sponsor
- Longboard Pharmaceuticals
- Brief Summary
The objective of this study is to assess the safety, tolerability, efficacy, and pharmacokinetics of adjunctive therapy of LP352 in adults and adolescents with developmental and epileptic encephalopathies.
- Detailed Description
This is a randomized, double-blind, parallel-group, dose-escalation, placebo-controlled study of LP352 in adults and adolescents with developmental and epileptic encephalopathies (DEE) with an average of ≥ 4 observed/countable motor seizures per 4-week period during the 12 weeks before screening while on stable antiseizure medicine (ASM).
Subjects will be randomized 4:1 to LP352 or placebo. The study will have a baseline period of 28 days, followed by a 15 day up-titration period during which time subjects will titrate up to their highest tolerated doses, and a 60-day maintenance period. After Day 75, subjects will be tapered down over a period of up to 15 days, with a follow-up visit 30 days after last dose. Enrolled subjects will be allowed to continue treatment with up to 4 concomitant ASMs at a stable dose.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 52
- Male or non-pregnant, non-lactating female, age 12 to 65 years
- Diagnosis of Dravet syndrome, Lennox-Gastaut syndrome, or other developmental and epileptic encephalopathy
- Has a minimum number of seizures per 4-week period while taking 1 to 4 anti-seizure medications
- All medications and epilepsy interventions must be stable for 4 weeks before screening and are expected to remain stable during the study
- The patient/parent/caregiver is able and willing to attend study visits, complete the diary and take study drug as instructed
Key
- Current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction, stroke, pulmonary arterial hypertension or abnormal blood pressure
- Has glaucoma, renal impairment, liver disease or any other medical condition that would affect study participation or pose a risk to the subject
- Current or recent history of moderate or severe depression, anorexia nervosa, bulimia or at risk of suicidal behavior
- Currently taking anorectic agents, monoamine oxidase inhibitors; serotonin agonists or antagonists including fenfluramine, atomoxetine, vortioxetine, or other medications for weight loss
- Positive test result on the drug screen, except tetrahydrocannabinol (THC) for patients taking prescribed cannabidiol
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Placebo for LP352 LP352 LP352 Subjects will be titrated up to highest tolerated dose of LP352 during a 15-day period, followed by a 60-day maintenance period and a 15-day taper/down titration period.
- Primary Outcome Measures
Name Time Method Columbia-Suicide Severity Rating Scale (C-SSRS) Response Baseline up to Day 75 Type of Suicidal Ideation, Intensity (1 - 5, with 5 being most severe), Suicidal Behavior
Percent Change from Baseline in Observed Countable Motor Seizure Frequency (per 28 Days) During the Maintenance Period Baseline up to Day 75 Treatment-emergent Adverse Events Baseline up to Day 75 Incidence and severity of adverse events, including serious adverse events and adverse events leading to study discontinuation and clinically significant changes in vital signs, physical examination endpoints, clinical safety laboratory values and ECGs
Patient Health Questionnaire-9 Total Score and Question 9 Score Baseline up to Day 75 Severity Rating Scale: 0 - 27; higher scores indicate greater severity of depressive disorder
Percent Change from Baseline in Observed Countable Motor Seizure Frequency (per 28 Days) During the Treatment Period Baseline up to Day 75
- Secondary Outcome Measures
Name Time Method Observed Plasma Concentrations of LP352 by Time and Dose Baseline up to Day 75 Modeled Estimate of Average Plasma Concentration Baseline up to Day 75 Modeled Estimate of Observed Plasma Concentration Just Prior to Dosing Baseline up to Day 75 Correlation of Plasma Concentration with Incidence of Treatment-emergent Adverse Events Baseline up to Day 75 Correlation of Plasma Concentration with Seizure Frequency Baseline up to Day 75 Observed and Change from Baseline Prolactin Concentration During the Treatment Period Baseline up to Day 75
Related Research Topics
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Trial Locations
- Locations (34)
University of Miami
🇺🇸Miami, Florida, United States
Consultants in Epilepsy and Neurology
🇺🇸Boise, Idaho, United States
Royal Brisbane Women's Hospital
🇦🇺Herston, Queensland, Australia
Northwestern University Feinberg School of Medicine
🇺🇸Chicago, Illinois, United States
Northeast Regional Epilepsy Group
🇺🇸Staten Island, New York, United States
Northwest Florida Clinical Research Group
🇺🇸Gulf Breeze, Florida, United States
Mid-Atlantic Epilepsy and Sleep Center
🇺🇸Bethesda, Maryland, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
Boston Children's Health Physicians LLP
🇺🇸Hawthorne, New York, United States
Research Institute of Orlando
🇺🇸Orlando, Florida, United States
University of Arizona - Health Sciences Center
🇺🇸Tucson, Arizona, United States
Northwell Health
🇺🇸New York, New York, United States
Hawaii Pacific Neuroscience
🇺🇸Honolulu, Hawaii, United States
Child Neurology Consultants of Austin
🇺🇸Austin, Texas, United States
Austin Health
🇦🇺Heidelberg, Victoria, Australia
Queensland Children's Hospital
🇦🇺South Brisbane, Queensland, Australia
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Children's Hospital of Orange County
🇺🇸Orange, California, United States
University of California San Francisco
🇺🇸San Francisco, California, United States
Austin Epilepsy Care Center
🇺🇸Austin, Texas, United States
Rancho Los Amigos National Rehabilitation Center (RLANRC)
🇺🇸Downey, California, United States
Advent Health Orlando
🇺🇸Orlando, Florida, United States
University of South Florida
🇺🇸Tampa, Florida, United States
New York University Langone Hospital - Long Island
🇺🇸Mineola, New York, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
OnSite Clinical Solutions LLC
🇺🇸Charlotte, North Carolina, United States
Wake Forest University School of Medicine
🇺🇸Winston-Salem, North Carolina, United States
Providence Neurological Specialties-East
🇺🇸Portland, Oregon, United States
University of Washington Valley Medical Center
🇺🇸Renton, Washington, United States
Spectrum Health
🇺🇸Grand Rapids, Michigan, United States
Alfred Health
🇦🇺Melbourne, Victoria, Australia
Monash Children's Hospital, Monash Health
🇦🇺Clayton, Victoria, Australia
University Hospitals Cleveland Medical Center
🇺🇸Cleveland, Ohio, United States