Clinical trial • Phase III • Neurology

BEXICASERIN HYDROCHLORIDE for Developmental and Epileptic Encephalopathies

Phase III trial of BEXICASERIN HYDROCHLORIDE for Developmental and Epileptic Encephalopathies. open-label. 135 participants.

Overview

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

Key dates

Initial CTIS Submission Date
04-03-2025
First CTIS Authorization Date
17-06-2025

Trial design

open-label Phase III trial across 40 sites in Belgium, France, Netherlands and others.

Open Label
Yes
Target Sample Size
135

Eligibility

Recruits 135 paediatric patients.

Vulnerable Population
Study includes children and other vulnerable participants (isVulnerablePopulationSelected = true). Consent approach: participants must be willing and able to provide written informed consent; where the participant is unable to provide consent, an appropriate legal representative must provide informed consent and the participant must assent (per local regulations) before participation. Study materials include age- and role-specific ICFs and assent forms (adult ICFs, caregiver ICFs, parent/LAR ICFs, pediatric assent for multiple age bands, picture books for young children, and pregnancy-specific materials) in multiple languages as provided per country.

Inclusion criteria

  • {"criterion_text":"- 1. The participant has satisfactorily completed Study LP352-301 or LP352-302 Visit 8, and who, in the opinion of the investigator, may benefit from continued LP352 administration."}
  • {"criterion_text":"- 2. Diagnosis of DEE that includes DS, LGS, or DEE Other (as defined and evaluated in Study LP352-301 or LP352-302)."}
  • {"criterion_text":"- 3. Has at least one reliable and consistent parent, legal guardian, or caregiver during the study."}
  • {"criterion_text":"- 4. 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 must provide informed consent and the participant will need to assent (as per local regulations) before participation in the study."}
  • {"criterion_text":"- 5. 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."}

Exclusion criteria

  • {"criterion_text":"- 1. Using exclusionary medications defined in protocol LP352-303"}
  • {"criterion_text":"- 2. Current use of any cannabis product or cannabidiol that is not in oral solution/capsule/tablet form, not obtained from a government-approved dispensary, or containing ≥50% THC."}
  • {"criterion_text":"- 3. 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."}
  • {"criterion_text":"- 4. Is unable or unwilling to comply with any of the study requirements or timelines."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- 1. Incidence and severity of TEAEs, including SAEs and AEs leading to discontinuation","definition_or_measurement_approach":"Collection and reporting of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events leading to discontinuation through safety monitoring and investigator reporting."}
  • {"endpoint_text":"- 2. Changes in safety laboratory parameters","definition_or_measurement_approach":"Laboratory safety tests (clinical chemistry, hematology, etc.) assessed over time to detect changes from baseline."}
  • {"endpoint_text":"- 3. Physical examination findings","definition_or_measurement_approach":"Periodic physical examinations with findings recorded and compared over treatment."}
  • {"endpoint_text":"- 4. Vital signs","definition_or_measurement_approach":"Measurement of vital signs (e.g., blood pressure, heart rate, respiratory rate, temperature) at scheduled visits."}
  • {"endpoint_text":"- 5. ECGs, and growth parameters (height and weight)","definition_or_measurement_approach":"Electrocardiograms performed per schedule; measurement of height and weight to assess growth parameters."}
  • {"endpoint_text":"- 6. Changes in suicidality or depression as rated by C-SSRS and PHQ-9 responses","definition_or_measurement_approach":"Assessment using Columbia-Suicide Severity Rating Scale (C-SSRS) and Patient Health Questionnaire-9 (PHQ-9) questionnaires."}
  • {"endpoint_text":"- 7. Changes in puberty development as rated by Tanner Staging","definition_or_measurement_approach":"Pubertal development assessed using Tanner staging at scheduled assessments."}

Secondary endpoints

  • {"endpoint_text":"- 1. Frequency percent change in countable motor seizures during Treatment (28-day average) compared to Baseline (28-day average) as assessed by seizure eDiary","definition_or_measurement_approach":"Percent change in frequency of countable motor seizures using 28-day averages, assessed from participant/caregiver seizure eDiary (seizure eDiary/eDiaries)."}
  • {"endpoint_text":"- 2. 50% responder rate (the percentage of participants with a ≥50% reduction in countable motor seizures) during Treatment (28-day average) compared to Baseline (28-day average) as assessed by seizure eDiary","definition_or_measurement_approach":"Proportion of participants achieving ≥50% reduction in countable motor seizures (28-day averages), assessed via seizure eDiary."}
  • {"endpoint_text":"- 3. Frequency percent change in countable motor seizures during Maintenance (28-day average) compared to Baseline (28-day average) as assessed by seizure eDiary","definition_or_measurement_approach":"Percent change in seizure frequency during maintenance period (28-day averages) versus baseline, assessed via seizure eDiary."}
  • {"endpoint_text":"- 4. 50% responder rate (the percentage of participants with a ≥50% reduction in countable motor seizures) during Maintenance (28-day average) compared to Baseline (28-day average) as assessed by seizure eDiary","definition_or_measurement_approach":"Proportion of participants with ≥50% reduction in countable motor seizures during maintenance (28-day averages) compared to baseline, assessed via seizure eDiary."}

Recruitment

Digital Remote Recruitment
Yes
Planned Sample Size
135
Recruitment Window Months
42
Consent Approach
Written informed consent required from the participant if able; where the participant is unable to provide consent, an appropriate legal representative (LAR) must provide informed consent and the participant must assent according to local regulations. Study documentation includes adult ICFs, caregiver ICFs, parent/LAR ICFs, pediatric assent forms for multiple age bands (e.g., 5-11, 6-11, 12-17), picture books for young children, pregnancy-specific ICFs/assent, and Study Information Sheets. Documents are provided in multiple languages across countries (English, Dutch, French, Spanish, Italian, Portuguese, German, Latvian, Russian and others as listed per-country documents).

Methods

  • Patient recruitment via third-party vendor Elligo Health Research Inc.: "Patient recruitment and website, promo items" (vendor listed in thirdParties).
  • eCOA/eDiary support via WCG Clinical Inc.: duty listed as "eCoa/eDiary".
  • Patient travel support and reimbursements via Scout Clinical: "Patient travel and reimbursements, stipends".
  • Courier and shipment logistics via Marken LLP: "Courier, DTP/DFP Shipment".
  • ECG and cardiac services via Eresearchtechnology Inc.: "ECG, cardiac services".
  • Study platform/URL support via Medidata Solutions Inc.: "URL" and other site/platform services.

Geography

Total Number Of Sites
40
Total Number Of Participants
181

Belgium

Earliest CTIS Part Ii Submission Date
19-03-2025
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
419
Number Of Sites
2
Number Of Participants
9

Sites

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

France

Earliest CTIS Part Ii Submission Date
03-06-2025
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
343
Number Of Sites
8
Number Of Participants
32

Sites

Site Name
Centre Hospitalier Universitaire De Rennes
Department Name
Pédiatrie
Principal Investigator Name
Silvia NAPURI PEIRANO
Principal Investigator Email
silvia.napuri@chu-rennes.fr
Contact Person Name
Silvia NAPURI PEIRANO
Contact Person Email
silvia.napuri@chu-rennes.fr
Site Name
Centre Hospitalier Universitaire De Lille
Department Name
Neuropédiatrie
Principal Investigator Name
Sylvie NGUYEN THE TICH
Principal Investigator Email
sylvie.nguyenthetich@chru-lille.fr
Contact Person Name
Sylvie NGUYEN THE TICH
Site Name
Centre Hospitalier Regional De Marseille
Department Name
Neurologie Pédiatrique et Epileptologie Pédiatrique
Principal Investigator Name
Mathieu MILH
Principal Investigator Email
mathieu.milh@ap-hm.fr
Contact Person Name
Mathieu MILH
Contact Person Email
mathieu.milh@ap-hm.fr
Site Name
Hopital Des Enfants
Department Name
Neurologie, Infectiologie Pédiatrique
Principal Investigator Name
Caroline HACHON LE CAMUS
Principal Investigator Email
hachonlecamus.c@chu-toulouse.fr
Contact Person Name
Caroline HACHON LE CAMUS
Site Name
Hospices Civils De Lyon
Department Name
Neurologie fonctionnelle et épileptologie
Principal Investigator Name
Sylvain RHEIMS
Principal Investigator Email
sylvain.rheims@chu-lyon.fr
Contact Person Name
Sylvain RHEIMS
Contact Person Email
sylvain.rheims@chu-lyon.fr
Site Name
Assistance Publique Hopitaux De Paris (48 Boulevard Serurier)
Department Name
Neurologie Pédiatrique
Principal Investigator Name
Stéphane AUVIN
Principal Investigator Email
stephane.auvin@aphp.fr
Contact Person Name
Stéphane AUVIN
Contact Person Email
stephane.auvin@aphp.fr
Site Name
Assistance Publique Hopitaux De Paris (149 Rue De Sevres)
Department Name
Neurologie pédiatrique
Principal Investigator Name
Rima EL NABBOUT-TARANTINO
Principal Investigator Email
Rima.Nabbout@aphp.fr
Contact Person Name
Rima EL NABBOUT-TARANTINO
Contact Person Email
Rima.Nabbout@aphp.fr
Site Name
Les Hopitaux Universitaires De Strasbourg
Department Name
Neuropédiatrie
Principal Investigator Name
Anne DE SAINT MARTIN
Principal Investigator Email
Anne.desaintmartin@chru-strasbourg.fr
Contact Person Name
Anne DE SAINT MARTIN

Netherlands

Earliest CTIS Part Ii Submission Date
02-06-2025
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
344
Number Of Sites
2
Number Of Participants
18

Sites

Site Name
Wilhelmina Childrens Hospital
Department Name
Pediatric Neurology
Principal Investigator Name
Floor Jansen
Principal Investigator Email
f.e.jansen@umcutrecht.nl
Contact Person Name
Floor Jansen
Contact Person Email
f.e.jansen@umcutrecht.nl
Site Name
Epilepsie Instellingen Nederland Stichting
Department Name
Epilepsy
Principal Investigator Name
Zwany Metting
Principal Investigator Email
trial@sein.nl
Contact Person Name
Zwany Metting
Contact Person Email
trial@sein.nl

Spain

Earliest CTIS Part Ii Submission Date
30-05-2025
Latest Decision Or Authorization Date
13-05-2026
Processing Time Days
348
Number Of Sites
10
Number Of Participants
46

Sites

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

Germany

Earliest CTIS Part Ii Submission Date
03-06-2025
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
343
Number Of Sites
7
Number Of Participants
31

Sites

Site Name
Universitaetsklinikum Frankfurt AöR
Department Name
ZNN – Epilepsy Center Rhine-Main
Principal Investigator Name
Adam Strzelczyk
Principal Investigator Email
strzelczyk@med.uni-frankfurt.de
Contact Person Name
Adam Strzelczyk
Site Name
Gesellschaft Fuer Epilepsieforschung
Department Name
Krankenhaus Mara
Principal Investigator Name
Christian Brandt
Principal Investigator Email
christian.brandt@mara.de
Contact Person Name
Christian Brandt
Contact Person Email
christian.brandt@mara.de
Site Name
Zentrum für Psychiatrie Südwürttemberg Epilepsy Centre Bodensee
Department Name
Department of Neurology and Epileptology
Principal Investigator Name
Christian Tilz
Principal Investigator Email
Christian.Tilz@ZfP-Zentrum.de
Contact Person Name
Christian Tilz
Contact Person Email
Christian.Tilz@ZfP-Zentrum.de
Site Name
Universitaetsklinikum Schleswig-Holstein AöR
Department Name
Klinik für Kinder- und Jugendmedizin II Abteilung für Neuropädiatrie und Sozialpädiatrie
Principal Investigator Name
Hiltrud Muhle
Principal Investigator Email
hiltrud.muhle@uksh.de
Contact Person Name
Hiltrud Muhle
Contact Person Email
hiltrud.muhle@uksh.de
Site Name
Schoen Klinik Vogtareuth SE & Co. KG
Principal Investigator Name
Milka Pringsheim
Principal Investigator Email
MPringsheim@schoen-klinik.de
Contact Person Name
Milka Pringsheim
Contact Person Email
MPringsheim@schoen-klinik.de
Site Name
Epilepsiezentrum Kleinwachau gGmbH
Department Name
Fachklinik für Neurologie
Principal Investigator Name
Thomas Mayer
Principal Investigator Email
t.mayer@kleinwachau.de
Contact Person Name
Thomas Mayer
Contact Person Email
t.mayer@kleinwachau.de
Site Name
Universitaetsklinikum Bonn AöR
Department Name
Neuropädiatrie und Sozialpädiatrisches Zentrum (SPZ)
Principal Investigator Name
Kerstin Alexandra Klotz
Principal Investigator Email
studie-kinderneurologie@ukbonn.de
Contact Person Name
Kerstin Alexandra Klotz

Italy

Earliest CTIS Part Ii Submission Date
02-06-2025
Latest Decision Or Authorization Date
13-05-2026
Processing Time Days
345
Number Of Sites
7
Number Of Participants
21

Sites

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

Latvia

Earliest CTIS Part Ii Submission Date
04-06-2025
Latest Decision Or Authorization Date
13-05-2026
Processing Time Days
343
Number Of Sites
1
Number Of Participants
6

Sites

Site Name
Bernu Kliniska Universitates Slimnica VSIA
Department Name
Epilepsy and Sleep Medicine Centre
Principal Investigator Name
Jurgis Strautmanis
Principal Investigator Email
jurgis.strautmanis@gmail.com
Contact Person Name
Jurgis Strautmanis
Contact Person Email
jurgis.strautmanis@gmail.com

Portugal

Earliest CTIS Part Ii Submission Date
30-05-2025
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
347
Number Of Sites
3
Number Of Participants
18

Sites

Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Paediatrics Department
Principal Investigator Name
Sofia Quintas
Principal Investigator Email
sofiamendesquintas@gmail.com
Contact Person Name
Sofia Quintas
Contact Person Email
sofiamendesquintas@gmail.com
Site Name
Unidade Local De Saude De Santo Antonio E.P.E.
Department Name
Paediatrics - NeuroPediatric Unit
Principal Investigator Name
Cristina Garrido
Principal Investigator Email
cristinagarrido@ulssa.min-saude.pt
Contact Person Name
Cristina Garrido
Site Name
Unidade Local De Saude De Coimbra E.P.E.
Department Name
Child development Center Service - Hospital Pediátrico - ULS Coimbra
Principal Investigator Name
Cristina Pereira
Principal Investigator Email
cristina.d.pereira@gmail.com
Contact Person Name
Cristina Pereira
Contact Person Email
cristina.d.pereira@gmail.com

Sponsor

Primary sponsor

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

Contract research organisations

Name
PPD Development LP
Responsibilities
codes: 1,11,12,13,14,2,4,5,6,7,8
Name
PPD International Holdings LLC
Responsibilities
code: 4
Name
Endpoint Clinical Inc.
Responsibilities
code: 3
Name
Marken LLP
Responsibilities
Courier, DTP/DFP Shipment
Name
WCG Clinical Inc.
Responsibilities
eCoa/eDiary

Third parties

  • {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"codes: 1,11,12,13,14,2,4,5,6,7,8","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"eCoa/eDiary; code: 7","organisation_type":"Pharmaceutical company"}
  • {"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":"Marken LLP","duties_or_roles":"Courier, DTP/DFP Shipment","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Mms Holdings Inc.","duties_or_roles":"codes: 5,6","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"code: 3","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"URL; code: 7","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":"Eresearchtechnology Inc.","duties_or_roles":"ECG, cardiac services","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 PEG tube
Authorisation Status
prodAuthStatus: 1
Maximum Dose
36 mg (max daily)
Investigational Product Name
LP352 (bexicaserin) Placebo Oral Solution
Modality
Other

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