A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)
Condition(s) studied
Investigational drug(s) / intervention(s)
LP352: LP352 will be administered orally or through G-tube/ percutaneous endoscopic gastrostomy (PEG) tube.
Study summary
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.
Eligibility
Primary outcome measure(s)
- Number of participants reporting Treatment emergent adverse events (TEAEs) and serious adverse events (SAEs) and AEs leading to discontinuation — Up to 61 Weeks
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. - 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
- 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) — Up to 61 Weeks
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. - Number of participants with positive responses to Patient Health Questionnaire-9 (PHQ-9) and Question 9 — Up to 61 Weeks
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.
Trial sites (83)
| Facility | City | Region | Status |
|---|---|---|---|
| Arkansas Children's Hospital - Cardiology Clinic | Little Rock | Arkansas | |
| University of California Los Angeles (UCLA) - David Geffen School of Medicine - Global Health Pro... | Los Angeles | California | |
| Stanford University - Stanford School of Medicine - Pulmonary and Cystic Fibrosis Center | Palo Alto | California | |
| UCSF Benioff Children's Hospitals - San Francisco | San Francisco | California | |
| Children's Hospital Colorado - Heart Institute | Aurora | Colorado | |
| NW FL Clinical Research Group, LLC | Gulf Breeze | Florida | |
| University of Miami Medical Group (UMMG) | Miami | Florida | |
| Research Institute of Orlando LLC | Orlando | Florida | |
| Pediatric Epilepsy & Neurology Specialists (PENS) | Tampa | Florida | |
| University of South Florida (USF) - Tampa Campus | Tampa | Florida | |
| Consultants in Epilepsy & Neurology, PLLC | Boise | Idaho | |
| Mid-Atlantic Epilepsy and Sleep Center - Bethesda | Bethesda | Maryland | |
| Massachusetts General Hospital (MGH) | Boston | Massachusetts | |
| Mayo Clinic - Children's Center - Rochester | Rochester | Minnesota | |
| Barnabas Health - Institute of Neurology and Neurosurgery | Livingston | New Jersey | |
| Northeast Regional Epilepsy Group - Morristown | Morristown | New Jersey | |
| NYU Langone Health-NYU Comprehensive Epilepsy Center | New York | New York | |
| Oregon Health & Science University - Blood Brain Barrier and Neuro-Oncology Program | Portland | Oregon | |
| Child Neurology Consultants of Austin - Austin | Austin | Texas | |
| The University of Texas Medical School at Houston - UT Gastroenterology | Houston | Texas | |
| Sydney Children's Hospital - Randwick | Randwick | New South Wales | |
| The Children's Hospital-Westmead - Heart Centre for Children | Westmead | New South Wales | |
| Royal Brisbane and Women's Hospital (RBWH) | Brisbane | Queensland | |
| Queensland Children's Hospital (Lady Cilento Children's Hospital) | Brisbane | Queensland | |
| Austin Hospital | Heidelberg | Victoria | |
| The Royal Children's Hospital (RCH) Melbourne | Parkville | Victoria | |
| Universitair Ziekenhuis Antwerpen (UZA) | Edegem | Antwerp | |
| Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg | Leuven | Flemish Brabant | |
| Instituto de Neurologia de Curitiba (INC) | Curitiba | Paraná | |
| Universidade de Sao Paulo (USP) - Hospital das Clinicas da Faculdade de Medicina (HCFMUSP) - Inst... | São Paulo | Brazil | |
| Peking University First Hospital (Beida Hospital) | Beijing | Beijing Municipality | |
| Capital Medical University-Beijing Children's Hospital | Beijing | Beijing Municipality | |
| Guangzhou Medical University-The Second Affiliated Hospital | Guangzhou | Guangdong | |
| Wuhan Children's Hospital | Wuhan | Hubei | |
| Fudan University - Children's Hospital | Shanghai Shi | Shanghai Municipality | |
| Hôpital de Hautepierre - Les Hôpitaux Universitaires de Strasbourg | Strasbourg | Ain | |
| Hôpital Roger Salengro | Lille | Nord | |
| Centre Hospitalier Universitaire de Lyon - Hôpital Pierre Wertheimer | Bron | France | |
| AP-HM- Hôpital de La Timone | Marseille | France | |
| AP-HP - Hôpital universitaire Necker-Enfants malades | Paris | France |
+ 43 more sites — see the full list on the official registry below.
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06908226 on ClinicalTrials.gov ↗ ← All trials in Italy