A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)

Официальное название

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Investigate the Efficacy, Safety, and Tolerability of LP352 in the Treatment of Seizures in Children and Adults With Dravet Syndrome

Обзор

This (DEEp SEA 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 DS. The study consists of 3 main phases: Screening, Titration period, and Maintenance period, followed by a Taper period and Follow-Up. Participants will be randomized to LP352 or placebo. The total duration of the study will be approximately 24 months.

Вмешательства

• Препарат LP352
  LP352 will be administered orally or through G-tube/ percutaneous endoscopic gastrostomy (PEG) tube
• Препарат Placebo
  Participants will be administered with matching placebo orally or through G-tube/ PEG tube

Первичные конечные точки

• Frequency Percent Change in Countable Motor Seizures During Treatment Compared to Baseline [Срок оценки: Baseline and up to 15 Weeks]

Вторичные конечные точки (3)

• Safety and Tolerability of LP352 [Срок оценки: Up to 21 Weeks]
• Percentage of participants with ≥ 50% Reduction in countable motor seizures during Treatment compared to Baseline [Срок оценки: Baseline and up to 15 Weeks]
• Frequency Percent Change in Countable Motor Seizures during Maintenance compared to Baseline [Срок оценки: Baseline and up to 15 Weeks]

Критерии участия

Критерии включения

• Diagnosis of DS must fulfill all of the following criteria:

• Participants with seizure onset age >1 and <20 months
• The participant has a history of at least 1 of the following seizure type(s): prolonged generalized tonic-clonic, hemiclonic, myoclonic, tonic, atonic, atypical absence, focal impaired awareness, nonconvulsive status epilepticus
• 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 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.

Критерии исключения

• The participant has a history of infantile/epileptic spasms.
• 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 unwilling to comply with any of the study requirements or timelines.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 25 центров

• Arkansas Children's Hospital - PIN — Little Rock
• Children's Hospital Los Angeles - PIN — Los Angeles
• David Geffen School of Medicine at UCLA — Los Angeles
• The Stanford Division of Child Neurology — Palo Alto
• UCSF Children's Hospital — San Francisco
• Children's Hospital Colorado. — Aurora
• Children's National Medical Center — Washington D.C.
• NW FL Clinical Research Group, LLC — Gulf Breeze
• … и ещё 17 центров

Япония · 10 центров

• IUHW Narita Hospital — Narita-shi
• Fukuoka Children's Hospital — Fukuoka
• Hokkaido University Hospital — Sapporo
• National Hospital Organization Nishi-Niigata Chuo National Hospital — Niigata
• Okayama University Hospital — Okayama
• Osaka City General Hospital — Osaka-Shi Miyakojima-Ku
• Saitama Prefectural Children's Medical Center — Saitama-shi
• National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders — Shizuoka
• … и ещё 2 центра

Австралия · 8 центров

• Prince of Wales Hospital — Randwick
• Sydney Children's Hospital — Randwick
• Children's Hospital at Westmead — Westmead
• Royal Brisbane & Women's Hospital — Herston
• Queensland Children's Hospital — South Brisbane
• Austin Hospital — Heidelberg
• Royal Children's Hospital Melbourne — Parkville
• The Alfred Hospital — Melbourne

Франция · 8 центров

• Hôpital Roger Salengro — Lille
• Hospices Civils de Lyon - Hôpital Pierre Wertheimer — Bron
• AP-HM- Hôpital de La Timone — Marseille
• Hôpital Necker - Enfants Malades — Paris
• AP-HP - Hôpital universitaire Robert-Debré — Paris
• CHU de Rennes - Hôpital Pontchaillou — Rennes
• CHU de Strasbourg - Hôpital de Hautepierre — Strasbourg
• Centre Hospitalier Universitaire de Toulouse — Toulouse

Испания · 8 центров

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Бразилия · 7 центров

• Instituto de Neurologia de Curitiba (INC) — Curitiba
• Hospital Moinhos de Vento — Porto Alegre
• Instituto do Cerebro do Rio Grande do Sul - InsCer — Porto Alegre
• Hospital das Clinicas - UNICAMP — Campinas
• Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP - PPDS — Ribeirão Preto
• Fundacao Faculdade Regional de Medicina de Sao Jose Do Rio Preto Hospital de Base - PPDS — São José do Rio Preto
• Instituto de Psiquiatria HC FMUSP — São Paulo

Китай · 6 центров

• Peking University First Hospital - Daxing Campus — Пекин
• The Second Affiliated Hospital of Guangzhou Medical University — Гуанчжоу
• Shenzhen Children's Hospital — Шэньчжэнь
• Wuhan Children's Hospital — Ухань
• Children's Hospital of Fudan University — Шанхай
• Beijing Children's Hospital, Capital Medical University - PIN — Пекин

Италия · 6 центров

• Fondazione Policlinico Universitario A Gemelli - Rome - PPDS — Rome
• Fondazione Istituto Neurologico Nazionale Casimiro Mondino IRCCS — Pavia
• Azienda Ospedaliero Universitaria A Meyer (Duplicate) — Florence
• Azienda Ospedaliera Universitaria Integrata Di Verona — Verona
• Istituto G Gaslini Ospedale Pediatrico IRCCS (Duplicate) — Genova
• IRCCS Ospedale Pediatrico Bambino Gesu — Roma

Германия · 5 центров

• Klinikum der Johann-Wolfgang Goethe-Universitat — Frankfurt am Main
• Universitätsklinikum Bonn — Bonn
• Gesellschaft für Epilepsieforschung e.V. — Bielefeld
• Universitätsklinikum Schleswig-Holstein - Campus Kiel — Kiel
• Epilepsie-Zentrum Bodensee — Ravensburg

Канада · 4 центра

• Children's and Women's Health Centre of British Columbia-4480 Oak St — Vancouver
• London Health Sciences Centre -800 Commissioners Rd E — London
• Hospital For Sick Children — Toronto
• Toronto Western Hospital — Toronto

Сербия · 4 центра

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Великобритания · 4 центра

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Португалия · 3 центра

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Бельгия · 2 центра

• UZ Leuven — Leuven
• UZ Antwerpen — Antwerp

Латвия · 1 центр

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Мексика · 1 центр

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Идентификаторы

NCT: NCT06660394 · LP352-302 · 2024-514937-39-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗