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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-514974-39-00 | EU Trial (CTIS) Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LP352 | Experimental | Participants will be titrated up to highest tolerated dose of LP352 during the Titration period (Visit 1 - Visit 3), followed by maintenance period (Visit 4 - Visit 14) and then taper/down titration period (Visit 15 - Visit 17). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LP352 | Drug | LP352 will be administered orally or through G-tube/ percutaneous endoscopic gastrostomy (PEG) tube. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Up to 61 Weeks |
| Number of Participants With Clinically Significant Changes in Chemistry parameters | Up to 61 Weeks | |
| Number of Participants With Clinically Significant Changes in Hematology parameters | Up to 61 Weeks | |
| Number of Participants With Clinically Significant Changes in Urinalysis | Up to 61 Weeks | |
| Number of participants with clinically significant changes in vital signs | Up to 61 Weeks | |
| Number of participants with clinically significant changes in physical examinations | Up to 61 Weeks | |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital - PIN | Little Rock | Arkansas | 72202-3500 | United States | ||
| David Geffen School of Medicine at UCLA |
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| Number of participants with clinically significant changes in growth parameters |
| Up to 61 Weeks |
| Number of participants with clinically significant changes in electrocardiogram (ECG) parameters | 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. | 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. | Up to 61 Weeks |
| 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] | 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] | Baseline and up to 55 weeks |
| Los Angeles |
| California |
| 90095 |
| United States |
| NW FL Clinical Research Group, LLC | Gulf Breeze | Florida | 32561-4458 | United States |
| Research Institute of Orlando LLC | Orlando | Florida | 32806-5411 | United States |
| Pediatric Epilepsy and Neurology Specialists | Tampa | Florida | 33609-4181 | United States |
| Mid-Atlantic Epilepsy and Sleep Center | Bethesda | Maryland | 20817-1809 | United States |
| Institute of Neurology and Neurosurgery at Saint Barnabas, LLC | Livingston | New Jersey | 07039-5817 | United States |
| Northeast Regional Epilepsy Group - Morristown - 310 Madison Ave | Morristown | New Jersey | 07960 | United States |
| The University of Texas Medical School at Houston | Houston | Texas | 77030-3000 | United States |
| Austin Hospital | Heidelberg | Victoria | 3084 | Australia |
| AP-HP - Hôpital universitaire Robert-Debré | Paris | 75019 | France |
| Children's Clinical University Hospital | Riga | LV-1004 | Latvia |
| Mother and Child Health Care Institute of Serbia Dr Vukan Cupic | Belgrade | 11000 | Serbia |
| Hospital de La Santa Creu i Sant Pau | Barcelona | 8041 | Spain |
| Hospital Universitario Vithas Madrid La Milagrosa | Madrid | 28010 | Spain |
| Hospital Ruber Internacional (Grupo Quironsalud) | Madrid | 28034 | Spain |
| ID | Term |
|---|---|
| D012640 | Seizures |
| D004827 | Epilepsy |
| D065886 | Neurodevelopmental Disorders |
| D004831 | Epilepsies, Myoclonic |
| D065768 | Lennox Gastaut Syndrome |
| ID | Term |
|---|---|
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D001523 | Mental Disorders |
| D004829 | Epilepsy, Generalized |
| D000073376 | Epileptic Syndromes |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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