Active, not recruiting
Phase 3
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathies (DEE)
Condition(s) studied
Developmental and Epileptic Encephalopathy
Investigational drug(s) / intervention(s)
LP352Placebo
LP352: LP352 will be administered orally or through G-tube/ percutaneous endoscopic gastrostomy (PEG) tube
Placebo: Participants will be administered with matching placebo orally or through G-tube/ PEG tube.
Study summary
This (DEEp OCEAN 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 DEE. The study consists of 3 main phases: Screening, Titration period, Maintenance period, followed by a Taper period and Follow-Up. The total duration of the study will be approximately 24 months.
Eligibility
Inclusion Criteria:
* Participants who are characterized as having Lennox-Gastaut Syndrome (LGS) must fulfill all of the following criteria:
* Onset of seizures at ≤8 years old
* History of tonic/tonic-atonic seizures plus at least 1 of the following seizure type(s): atypical absence, atonic, myoclonic, focal impaired awareness, generalized tonic-clonic, nonconvulsive status epilepticus, or epileptic spasms
* Presence of developmental plateauing or regression
* History of electroencephalogram (EEG) showing generalized slow (\<2.5 Hertz \[Hz\]) spike-and-wave complexes
* Participants who are characterized as having DEE (Other) must fulfill all of the following criteria:
* Does not meet criteria for LGS
* Onset of seizures at ≤5 years old
* Presence of developmental plateauing or regression
* History of multiple seizure types
* History of interictal EEG background showing diffuse or multifocal slowing (with or without epileptiform activity)
* 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 each of 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; in instances where the participant is unable to provide consent, an appropriate legal representative.
Exclusion Criteria:
* The participant has a diagnosis of Dravet Syndrome (DS) or has a mutation of the Sodium channel protein type 1 subunit alpha (SCN1A) gene consistent with DS.
* 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 is currently using 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 unable or unwilling to comply with any of the study requirements or timelines.
Primary outcome measure(s)
- Frequency Percent Change in Countable Motor Seizures During Treatment Compared to Baseline — Baseline and up to 15 Weeks
The percent change from Baseline in countable motor seizure frequency during Treatment will be calculated as countable motor seizure frequency during Treatment minus countable motor seizure frequency during Screening and divided by seizure frequency during Screening and multiplied by 100 where each seizure frequency will be based on number of seizures.
Trial sites (116)
| Facility | City | Region | Status |
| Arkansas Children's Hospital - Cardiology Clinic |
Little Rock |
Arkansas |
|
| Children's Hospital Los Angeles (CHLA) |
Los Angeles |
California |
|
| 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 |
|
| Children's National Hospital - Children's National Heart Center |
Washington D.C. |
District of Columbia |
|
| NW FL Clinical Research Group, LLC |
Gulf Breeze |
Florida |
|
| University of Miami Medical Group (UMMG) |
Miami |
Florida |
|
| Nicklaus Children's Hospital (Miami Children's Hospital) |
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 |
|
| Children's Healthcare of Atlanta (CHOA) |
Atlanta |
Georgia |
|
| Rare Disease Research, LLC. (RDR) |
Atlanta |
Georgia |
|
| Consultants in Epilepsy & Neurology, PLLC |
Boise |
Idaho |
|
| Northwestern Memorial Hospital |
Chicago |
Illinois |
|
| Northwestern University - Ann & Robert H. Lurie Children's Hospital of Chicago - Center for Cance... |
Chicago |
Illinois |
|
| University of Iowa Health Care Medical Center (University of Iowa Hospitals & Clinics) |
Iowa City |
Iowa |
|
| 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 |
|
| Le Bonheur Children's Hospital - Outpatient Center |
Memphis |
Tennessee |
|
| Child Neurology Consultants of Austin - Austin |
Austin |
Texas |
|
| Cook Children's Jane and John Justin Neurosciences Center |
Fort Worth |
Texas |
|
| The University of Texas Medical School at Houston - UT Gastroenterology |
Houston |
Texas |
|
| MultiCare Health System |
Tacoma |
Washington |
|
| Prince of Wales Hospital (POWH) |
Randwick |
New South Wales |
|
| 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 Alfred Hospital |
Melbourne |
Victoria |
|
| The Royal Children's Hospital (RCH) Melbourne |
Parkville |
Victoria |
|
| Universitair Ziekenhuis Antwerpen (UZA) |
Edegem |
Antwerp |
|
+ 76 more sites — see the full list on the official registry below.