This (DEEp OLE Study) is a multicentre, open-label study to investigate the long-term safety, efficacy, tolerability, and pharmacokinetics (PK) of LP352 in the treatment of seizures in children and adults with DEE who completed Study LP352-301 or LP352-302. The study consists of 3 main phases: Screening, Titration period and Maintenance period, followed by a Taper period and Follow-Up. The total duration of the study will be approximately 14 months.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
324
LP352 will be administered orally or through G-tube/ percutaneous endoscopic gastrostomy (PEG) tube.
Arkansas Children's Hospital - PIN
Little Rock, Arkansas, United States
David Geffen School of Medicine at UCLA
Los Angeles, California, United States
Number of participants reporting Treatment emergent adverse events (TEAEs) and serious adverse events (SAEs) and AEs leading to discontinuation
An AE is defined as any untoward medical occurrence in a participant enrolled into this study, regardless of its causal relationship to the study drug. A treatment-emergent AE is defined as any event that is not present before exposure to study drug or any event or condition that is already present that worsens in either intensity or frequency after exposure to study drug. An SAE is defined as any event that: Results in death; Is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization. "Inpatient hospitalization" includes admission to an emergency room for observation and/or treatment that would have been insufficient in an outpatient setting; results in persistent or significant disability/incapacity; Is a congenital anomaly/birth defect or is an important medical event. An adverse event of special interest (AESI) is an AE or SAE is defined as an AE or SAE of scientific or medical concern specific to the sponsor's product or program.
Time frame: Up to 61 Weeks
Number of Participants With Clinically Significant Changes in Chemistry parameters
Time frame: Up to 61 Weeks
Number of Participants With Clinically Significant Changes in Hematology parameters
Time frame: Up to 61 Weeks
Number of Participants With Clinically Significant Changes in Urinalysis
Time frame: Up to 61 Weeks
Number of participants with clinically significant changes in vital signs
Time frame: Up to 61 Weeks
Number of participants with clinically significant changes in physical examinations
Time frame: Up to 61 Weeks
Number of participants with clinically significant changes in growth parameters
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NW FL Clinical Research Group, LLC
Gulf Breeze, Florida, United States
Research Institute of Orlando LLC
Orlando, Florida, United States
Pediatric Epilepsy and Neurology Specialists
Tampa, Florida, United States
Mid-Atlantic Epilepsy and Sleep Center
Bethesda, Maryland, United States
Institute of Neurology and Neurosurgery at Saint Barnabas, LLC
Livingston, New Jersey, United States
Northeast Regional Epilepsy Group - Morristown - 310 Madison Ave
Morristown, New Jersey, United States
The University of Texas Medical School at Houston
Houston, Texas, United States
Austin Hospital
Heidelberg, Victoria, Australia
...and 6 more locations
Time frame: Up to 61 Weeks
Number of participants with clinically significant changes in electrocardiogram (ECG) parameters
Time frame: Up to 61 Weeks
Number of participants with postive responses to Columbia-Suicide Severity Rating Scale (C-SSRS)
C-SSRS was developed by researchers at Columbia University as a tool to help systematically assess suicidal ideation and behavior. It is composed of questions addressing suicidal ideation and suicidal behavior, as well as self-injurious behavior without suicidal intent. The tool will be administered by a trained operator/interviewer (investigator or designee) via interview with the participant at the study time points. If the participant is unable to complete the C-SSRS due to developmental status, the participant's legally acceptable representative may not complete the C SSRS. In these cases, the investigator may use clinical judgment to assess both the participant's status regarding suicidality and ability to complete the scale, both of which must then be documented in the source document.
Time frame: Up to 61 Weeks
Number of participants with positive responses to Patient Health Questionnaire-9 (PHQ-9) and Question 9
The PHQ-9 is a multipurpose instrument for Screening, diagnosing, monitoring, and measuring the severity of depression. The scale is an easy-to-use participant questionnaire that is a self-administered version of the Primary Care Evaluation of Mental Disorders diagnostic instrument for common mental disorders. The PHQ-9 is the depression module, which scores each of the 9 Diagnostic and Statistical Manual of Mental Disorders IV, Text Revision criteria as "0" (not at all) to "3" (nearly every day). It has been validated for use in primary care. Participants with a depression score of greater than 9 (mild) on the PHQ-9 scale or a positive response to Question 9 should be excluded from the study. When there is a positive response to PHQ-9 Question 9 post randomization, the investigator should determine whether an AE has occurred.
Time frame: Up to 61 Weeks
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) - (countable motor seizure frequency during Baseline \[Visit 1 of LP352-301 and LP352-302\]) ÷ seizure frequency during Baseline \[Visit 1 of LP352-301 and LP352-302\]) × 100
Time frame: Baseline and up to 55 weeks
Percentage of participants with ≥ 50% Reduction in countable motor seizures during Treatment compared to Baseline [Visit 1 of LP352-301 and LP352-302]
Time frame: Baseline and up to 55 weeks
Frequency Percent Change in Countable Motor Seizures during Maintenance compared to Baseline [Visit 1 of LP352-301 and LP352-302]
Time frame: Baseline and up to 55 weeks