Clinical trial • Phase III • Neurology|Rare Disease

BEXICASERIN HYDROCHLORIDE for Developmental and Epileptic Encephalopathies

Phase III trial of BEXICASERIN HYDROCHLORIDE for Developmental and Epileptic Encephalopathies.

Overview

Trial Therapeutic Area
Neurology|Rare Disease
Trial Disease
Developmental and Epileptic Encephalopathies
Trial Stage
Phase III
Drug Modality
Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
14-02-2025
First CTIS Authorization Date
11-06-2025

Trial design

Randomised, placebo: lp352 (bexicaserin) placebo oral solution (oral). dose/schedule not specified in the provided ctis data.-controlled Phase III trial across 42 sites in France, Latvia, Spain and others.

Randomised
Yes
Comparator
Placebo: LP352 (bexicaserin) Placebo Oral Solution (oral). Dose/schedule not specified in the provided CTIS data.
Target Sample Size
100

Eligibility

Recruits 100 paediatric patients.

Vulnerable Population
The trial includes children and other vulnerable participants. If a participant is unable to provide informed consent an appropriate legal representative must provide informed consent and the participant must assent according to local regulations. Age-specific assent and information/consent forms are available (documents for ages <6, 6-10, 11-14, 15-17 and caregiver/legal guardian ICFs). The CTIS record indicates isVulnerablePopulationSelected = true and multiple pediatric assent/ICF documents are prepared.

Inclusion criteria

  • {"criterion_text":"- •\tParticipants who are characterized as having Lennox-Gastaut Syndrome (LGS) must fulfill all of the following criteria: o\tOnset of seizures at ≤8 years old o\tHistory 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 o\tPresence of developmental plateauing or regression\n- •\tParticipants who are characterized as having DEE (Other) must fulfill all of the following criteria: o\tDoes not meet criteria for LGS o\tOnset of seizures at ≤5 years old o\tPresence of developmental plateauing or regression o\tHistory of multiple seizure types\n- •\tAdditional specific main inclusion criteria include the following: o 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. o\tThe participant has demonstrated an average of at least 4 countable motor seizures per month for each of the 3 months prior to Screening. o\tThe participant has been taking 1 to 4 antiseizure medications (ASMs) at a stable dose for at least 4 weeks prior to Screening. o\tThe participant, parent, or caregiver is willing and able (in the judgment of the investigator) to comply with completion of the diaries throughout the study. o\tThe 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 must provide informed consent and the participant will need to assent (as per local regulations) before participation in the study."}

Exclusion criteria

  • {"criterion_text":"- •\tThe participant has a diagnosis of Dravet Syndrome (DS)\n- • The participant has been admitted to a medical facility for treatment of status epilepticus requiring mechanical ventilation within 3 months prior to Screening.\n- •\tThe participant has a neurodegenerative disorder as indicated by magnetic resonance imaging or genetic testing.\n- •\tThe participant has an acquired lesion/injury unrelated to the primary etiology that could contribute as a secondary cause of seizures.\n- •\tThe participant is receiving exclusionary medications.\n- •\tThe participant has used of 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).\n- •\tThe 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.\n- •\tThe participant is unable or unwilling to comply with any of the study requirements or timelines."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Frequency percent change in countable motor seizures during Maintenance compared to Baseline","definition_or_measurement_approach":"Percent change in frequency of countable motor seizures during the Maintenance period compared with Baseline, assessed using countable motor seizure counts (seizure diaries/recorded countable motor seizures)."}

Secondary endpoints

  • {"endpoint_text":"- 50% responder rate (the percentage of participants with a ≥50% reduction in countable motor seizures) during Maintenance compared to Baseline","definition_or_measurement_approach":"Responder rate defined as the percentage of participants achieving ≥50% reduction in countable motor seizures during Maintenance versus Baseline, assessed from seizure counts/diaries."}
  • {"endpoint_text":"- Frequency percent change in countable motor seizures during Treatment compared to Baseline","definition_or_measurement_approach":"Percent change in frequency of countable motor seizures during the overall Treatment period compared with Baseline, assessed using seizure counts/diaries."}

Recruitment

Digital Remote Recruitment
Yes
Planned Sample Size
100
Recruitment Window Months
11
Consent Approach
Informed consent is obtained in writing from the participant if capable; if the participant is unable to provide consent an appropriate legal representative must provide informed consent. Participant assent is required per local regulations. Age-specific assent and consent forms are prepared (multiple pediatric assent forms for age groups such as <6, 6-10, 11-14, 15-17, caregiver ICFs, parent/legal guardian ICFs). Pre-ICF telephone data consent is used in some countries. Consent/assent documentation exists in multiple languages (French, Spanish, Portuguese, German, Italian, Latvian, Russian, Dutch, English, Finnish as shown in the published documents).

Methods

  • Study/program websites (DEEp-Studies website and country-specific study websites) providing study information and recruitment materials (cookie/privacy policies and website content documents are present).
  • Patient brochures, flyers and recruitment brochures (country-specific patient flyers and brochures listed for multiple countries).
  • CRO-supported recruitment and patient outreach (vendors listed with roles including patient recruitment, website creation and promotional items: e.g. Elligo Health Research Inc., Scout Clinical).
  • Pre-ICF telephone contact and telephone data consent (Pre-ICF Telephone Data Consent documents available).
  • Country-specific recruitment arrangements and materials (K1/K2 recruitment-arrangements and recruitment brochures per country/language).

Geography

Total Number Of Sites
42
Total Number Of Participants
117

France

Earliest CTIS Part Ii Submission Date
12-05-2025
Latest Decision Or Authorization Date
16-02-2026
Processing Time Days
280
Number Of Sites
8
Number Of Participants
16

Sites

Site Name
Hopital Des Enfants
Department Name
Neurologie, Infectiologie Pédiatrique
Contact Person Name
Caroline HACHON LE CAMUS
Site Name
Centre Hospitalier Universitaire De Rennes
Department Name
Pédiatrie
Contact Person Name
Silvia NAPURI PEIRANO
Contact Person Email
silvia.napuri@chu-rennes.fr
Site Name
Hospices Civils De Lyon
Department Name
Neurologie fonctionnelle et épileptologie
Contact Person Name
Sylvain RHEIMS
Contact Person Email
sylvain.rheims@chu-lyon.fr
Site Name
Centre Hospitalier Universitaire De Lille
Department Name
Neuropédiatrie
Contact Person Name
Sylvie NGUYEN THE TICH
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Neurologie pédiatrique
Contact Person Name
Rima El Nabbout-Tarantino
Contact Person Email
Rima.Nabbout@aphp.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Neurologie Pédiatrique
Contact Person Name
Stéphane AUVIN
Contact Person Email
stephane.auvin@aphp.fr
Site Name
Les Hopitaux Universitaires De Strasbourg
Department Name
Neuropédiatrie
Contact Person Name
Anne DE SAINT MARTIN
Site Name
Centre Hospitalier Regional De Marseille
Department Name
Neurologie Pédiatrique et Epileptologie Pédiatrique
Contact Person Name
Mathieu MILH
Contact Person Email
mathieu.milh@ap-hm.fr

Latvia

Earliest CTIS Part Ii Submission Date
04-06-2025
Latest Decision Or Authorization Date
11-02-2026
Processing Time Days
252
Number Of Sites
1
Number Of Participants
4

Sites

Site Name
Bernu Kliniska Universitates Slimnica VSIA
Department Name
Bernu Kliniska Universitates Slimnica VSIA
Contact Person Name
Jurgis Strautmanis
Contact Person Email
jurgis.strautmanis@gmail.com

Spain

Earliest CTIS Part Ii Submission Date
30-05-2025
Latest Decision Or Authorization Date
11-02-2026
Processing Time Days
257
Number Of Sites
10
Number Of Participants
30

Sites

Site Name
Hospital Universitario Regional De Malaga
Department Name
Neurology
Contact Person Name
Pedro J. Serrano Castro
Contact Person Email
p.serrano.eecc@gmail.com
Site Name
Hospital Ruber Internacional
Department Name
Neurology
Contact Person Name
Antonio Gil-Nagel Rein
Site Name
Hospital Universitario De Navarra
Department Name
Pediatrics
Contact Person Name
Sergio Aguilera Albesa
Contact Person Email
saguilea@navarra.es
Site Name
Hospital Infantil Universitario Nino Jesus
Department Name
Neurology
Contact Person Name
Victor Soto Insuga
Contact Person Email
vsoto@salud.madrid.org
Site Name
Hospital Sant Joan De Deu Barcelona
Department Name
Neurology
Contact Person Name
Maria del Carmen Fons Estupiña
Contact Person Email
carmen.fons@sjd.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Neurology
Contact Person Name
Manuel Toledo Argany
Contact Person Email
manuel.toledo@vallhebron.cat
Site Name
Hospital De La Santa Creu I Sant Pau
Department Name
Neurology
Contact Person Name
Alba Sierra Marcos
Contact Person Email
asierram@santpau.cat
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Neurology
Contact Person Name
Vicente Villanueva Haba
Contact Person Email
vevillanuevah@yahoo.es
Site Name
Hospital Vithas Parque San Antonio
Department Name
Neurology
Contact Person Name
Pablo Cabezudo Garcia
Contact Person Email
pablocabezudogcp@gmail.com
Site Name
Hospital La Milagrosa S.A.
Department Name
Neurology
Contact Person Name
Mar Carreño Martinez
Contact Person Email
maria.carreno@ext.vithas.es

Germany

Earliest CTIS Part Ii Submission Date
27-05-2025
Latest Decision Or Authorization Date
11-02-2026
Processing Time Days
260
Number Of Sites
7
Number Of Participants
15

Sites

Site Name
Universitaetsklinikum Schleswig-Holstein AöR
Department Name
Klinik für Kinder- und Jugendmedizin II Abteilung für Neuropädiatrie und Sozialpädiatrie
Contact Person Name
Hiltrud Muhle
Contact Person Email
hiltrud.muhle@uksh.de
Site Name
Epilepsiezentrum Kleinwachau gGmbH
Contact Person Name
Thomas Mayer
Contact Person Email
t.mayer@kleinwachau.de
Site Name
Universitaetsklinikum Bonn AöR
Department Name
Department of Neuropediatrics
Contact Person Name
Alexandra Klotz
Site Name
Gesellschaft Fuer Epilepsieforschung
Contact Person Name
Christian Brandt
Contact Person Email
christian.brandt@mara.de
Site Name
Zentrum für Psychiatrie Südwürttemberg Epilepsie Zentrum Bodensee
Department Name
Klinik für Neurologie und Epileptologie
Contact Person Name
Christian Tilz
Contact Person Email
Christian.Tilz@ZfP-Zentrum.de
Site Name
Schoen Klinik Vogtareuth SE & Co. KG
Contact Person Name
Milka Pringsheim
Contact Person Email
MPringsheim@schoen-klinik.de
Site Name
Universitaetsklinikum Frankfurt AöR
Contact Person Name
Adam Strzelczyk

Italy

Earliest CTIS Part Ii Submission Date
19-05-2025
Latest Decision Or Authorization Date
12-02-2026
Processing Time Days
269
Number Of Sites
7
Number Of Participants
14

Sites

Site Name
IRCCS Foundation Istituto Neurologico Carlo Besta
Department Name
SC Neuropsichiatria Infantile 2, Epilettologia e Neurologia dello Sviluppo (NOS)
Contact Person Name
Francesca Ragona
Site Name
Azienda Ospedaliera Universitaria Integrata Verona
Department Name
UOC Neuropsichiatria infantile
Contact Person Name
Francesca Darra
Contact Person Email
francesca.darra@univr.it
Site Name
Fondazione Istituto Neurologico Nazionale Casimiro Mondino
Department Name
SC Neuropsichiatria Infantile
Contact Person Name
Valentina De Giorgis
Contact Person Email
valentina.degiorgis@mondino.it
Site Name
IRCCS Istituto Giannina Gaslini
Department Name
UOC Pediatric Neurology and Muscular Diseases
Contact Person Name
Pasquale Striano
Contact Person Email
pasqualestriano@gaslini.org
Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
U.O.C. Pediatric Neuropsychiatry
Contact Person Name
Domenica Immacolata Battaglia
Site Name
Ospedale Pediatrico Bambino Gesu
Department Name
UOC Neurologia dell'epilessia e disturbi del movimento
Contact Person Name
Nicola Specchio
Contact Person Email
nicola.specchio@opbg.net
Site Name
Azienda Ospedaliera Universitaria Meyer IRCCS
Department Name
Neuroscience and Medical Genetics Department
Contact Person Name
Renzo Guerrini
Contact Person Email
renzo.guerrini@meyer.it

Portugal

Earliest CTIS Part Ii Submission Date
19-05-2025
Latest Decision Or Authorization Date
13-02-2026
Processing Time Days
270
Number Of Sites
3
Number Of Participants
8

Sites

Site Name
Unidade Local De Saude De Santo Antonio E.P.E.
Department Name
Peadritics - NeuroPediatric Unit
Contact Person Name
Cristina Garrido
Site Name
Unidade Local De Saude De Coimbra E.P.E.
Department Name
Child development Center Service
Contact Person Name
Cristina Pereira
Contact Person Email
cristina.d.pereira@gmail.com
Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Paediatrics Department
Contact Person Name
Sofia Quintas
Contact Person Email
sofiamendesquintas@gmail.com

Belgium

Earliest CTIS Part Ii Submission Date
06-03-2025
Latest Decision Or Authorization Date
10-02-2026
Processing Time Days
341
Number Of Sites
2
Number Of Participants
7

Sites

Site Name
UZ Leuven
Department Name
Paediatric Neurology
Contact Person Name
Lieven Lagae
Contact Person Email
Lieven.lagae@uzleuven.be
Site Name
Universitair Ziekenhuis Antwerpen
Department Name
Pediatrics/ Pediatrics Neurology
Contact Person Name
An-Sofie Schoonjans
Contact Person Email
an-sofie.schoonjans@uza.be

Finland

Earliest CTIS Part Ii Submission Date
27-04-2026
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
15
Number Of Sites
2
Number Of Participants
15

Sites

Site Name
Pohjois-Savon hyvinvointialue
Department Name
Epilepsy Center
Contact Person Name
Reetta Kälviäinen
Site Name
HUS-yhtymae
Department Name
Pediatric Clinical Trial Unit
Contact Person Name
Eeva-Liisa Metsähonkala
Contact Person Email
eeva-liisa.metsahonkala@hus.fi

Netherlands

Earliest CTIS Part Ii Submission Date
12-05-2025
Latest Decision Or Authorization Date
25-03-2026
Processing Time Days
317
Number Of Sites
2
Number Of Participants
8

Sites

Site Name
Epilepsie Instellingen Nederland Stichting
Department Name
Epilepsy
Contact Person Name
Aysun Altinbas
Contact Person Email
polikliniek.heemstede@sein.nl
Site Name
Wilhelmina Childrens Hospital
Department Name
Pediatric Neurology
Contact Person Name
Floor Jansen
Contact Person Email
f.e.jansen@umcutrecht.nl

Sponsor

Primary sponsor

Full Name
Longboard Pharmaceuticals Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Endpoint Clinical Inc.
Name
Medidata Solutions Inc.
Responsibilities
URL
Name
Scout Clinical
Responsibilities
Patient travel and reimbursements, stipends
Name
Azenta US Inc.
Responsibilities
Longterm sample storage
Name
Marken LLP
Responsibilities
Courier, DTP/DFP Shipment
Name
Centogene GmbH
Name
Elligo Health Research Inc.
Responsibilities
Patient recruitment and website, promo items
Name
PPD Development LP
Name
Eresearchtechnology Inc.
Responsibilities
ECG, cardiac services
Name
WCG Clinical Inc.
Responsibilities
eCoa/eDiary
Name
PPD International Holdings LLC

Third parties

  • {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"URL","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patient travel and reimbursements, stipends","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Azenta US Inc.","duties_or_roles":"Longterm sample storage","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Marken LLP","duties_or_roles":"Courier, DTP/DFP Shipment","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"Centogene GmbH","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Elligo Health Research Inc.","duties_or_roles":"Patient recruitment and website, promo items","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ECG, cardiac services","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"eCoa/eDiary","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Bexicaserin
Active Substance
BEXICASERIN HYDROCHLORIDE
Modality
Small molecule
Routes Of Administration
ORAL, NASOGASTRIC TUBE OR PERCUTANEOUS ENDOSCOPIC GASTROSTOMY TUBE USE
Route
ORAL, NASOGASTRIC TUBE OR PERCUTANEOUS ENDOSCOPIC GASTROSTOMY TUBE USE
Authorisation Status
Not authorised (investigational)
Maximum Dose
36 mg (max daily dose amount provided in CTIS: 36 mg)
Investigational Product Name
LP352 (bexicaserin) Placebo Oral Solution
Modality
Other

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