Clinical trial • Phase III • Neurology

PRAX-628 for Focal epilepsy | Primary generalized tonic-clonic epilepsy

Phase III trial of PRAX-628 for Focal epilepsy | Primary generalized tonic-clonic epilepsy. open-label, none/not specified-controlled. 140 participants.

Overview

Trial Therapeutic Area
Neurology
Trial Disease
Focal epilepsy | Primary generalized tonic-clonic epilepsy
Trial Stage
Phase III
Drug Modality
Small molecule

Key dates

Initial CTIS Submission Date
18-06-2025
First CTIS Authorization Date
06-10-2025

Trial design

open-label, none/not specified-controlled Phase III trial in Germany, Spain, Poland and others.

Open Label
Yes
Comparator
None/Not specified
Target Sample Size
140

Eligibility

Recruits 140 No vulnerable populations selected. Informed consent must be signed by the participant and caregiver (if applicable) in accordance with ICH GCP (see inclusion criterion 1)..

Pregnancy Exclusion
3. Participant who is pregnant, lactating or planning to become pregnant.
Vulnerable Population
No vulnerable populations selected. Informed consent must be signed by the participant and caregiver (if applicable) in accordance with ICH GCP (see inclusion criterion 1).

Inclusion criteria

  • {"criterion_text":"- 1. Participant and caregiver (if applicable), is willing to sign an informed consent document in accordance with International Council for Harmonisation (ICH) Good Clinical Practice (GCP) guidelines, indicating that they understand the purpose of the clinical trial; understands and can perform, complete, and comply with all the procedures and assessments that are required during the clinical trial, including the seizure diary and contraception, and is willing to participate in the clinical trial.\n- 2. Participant who completed an eligible clinical trial (unless agreed by the sponsor medical monitor), including PRAX-628-212, PRAX-628-321 or other vormatrigine trial, or participant who has received vormatrigine outside of an eligible clinical trial (such as an expanded access program).\n- 3. Participant who had adequate seizure diary and vormatrigine adherence and had been otherwise adherent to the study procedures in the opinion of the investigator unless deemed unacceptable by sponsor."}

Exclusion criteria

  • {"criterion_text":"- 1. Participant who withdrew or discontinued treatment from the treatment period of an eligible clinical trial unless deemed acceptable by the sponsor.\n- 2. Participant who plans to enter another interventional investigational study or is on any prohibited medication listed in Table 7.\n- 3. Participant who is pregnant, lactating or planning to become pregnant.\n- 4. Participant who is ineligible for the extension clinical trial in the view of the investigator."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Incidence and severity of treatment-emergent adverse events (TEAEs), including discontinuation of study drug due to TEAEs.\n- Changes in vital sign measurements.\n- Changes in clinical laboratory results.\n- Changes in electrocardiogram parameters.\n- Changes in suicidality, as assessed by Columbia Suicide Severity Rating Scale (C-SSRS)","definition_or_measurement_approach":"Incidence and severity of TEAEs as reported during treatment; changes in vital signs measured at scheduled visits; changes in clinical laboratory results from laboratory testing; changes in ECG parameters from electrocardiogram assessments; suicidality assessed using the Columbia Suicide Severity Rating Scale (C-SSRS)."}

Secondary endpoints

  • {"endpoint_text":"- (EXPLORATORY) Percent change in monthly (28 days) focal or generalized seizure frequency from the Screening/Observation Period of the primary trial.","definition_or_measurement_approach":"Percent change in monthly (28-day) focal or generalized seizure frequency compared with the Screening/Observation Period of the primary trial, using seizure diary data."}
  • {"endpoint_text":"- (EXPLORATORY) Change from baseline over time in PGI-S and CGI-S.","definition_or_measurement_approach":"Change from baseline over time measured using the Patient Global Impression of Severity (PGI-S) and Clinician Global Impression of Severity (CGI-S) scales."}
  • {"endpoint_text":"- (EXPLORATORY) Vormatrigine plasma concentrations","definition_or_measurement_approach":"Plasma concentrations of vormatrigine measured at selected timepoints post-dose (PK sampling)."}
  • {"endpoint_text":"- (EXPLORATORY) PGI-C, CGI-C, and CaGI-C (if applicable) assessed overtime post-screening.","definition_or_measurement_approach":"Patient Global Impression of Change (PGI-C), Clinician Global Impression of Change (CGI-C), and Caregiver Global Impression of Change (CaGI-C, if applicable) assessed over time after screening."}

Recruitment

Digital Remote Recruitment
True, Home health/telehealth visits are planned or available (Emvenio Clinical Research LLC listed with role 'Home Health Vendor /Telehealth').
Planned Sample Size
140
Recruitment Window Months
24
Consent Approach
Informed consent must be signed by the participant and caregiver (if applicable) in accordance with ICH GCP (inclusion criterion 1). Subject information and informed consent forms are provided in multiple languages (documents available in English, German, Spanish, Italian, Polish as per listed ICF documents). A Pregnant Partner ICF is available.

Methods

  • Recruitment from participants who completed an eligible clinical trial (e.g., PRAX-628-212, PRAX-628-321) or received vormatrigine via expanded access (explicit in inclusion criterion 2).
  • Site-based recruitment at participating neurology hospitals/clinics across Germany, Spain, Italy and Poland (listed trial sites).
  • Home health / Telehealth visits provided by third parties (explicitly listed: Emvenio Clinical Research LLC noted as 'Home Health Vendor /Telehealth').
  • Patient reimbursement of travel/meal expenses provided by Scout Clinical (explicit duty 'Patient reimbursement of travel/meal expenses').

Geography

Total Number Of Sites
36
Total Number Of Participants
140

Germany

Earliest CTIS Part Ii Submission Date
18-09-2025
Latest Decision Or Authorization Date
11-02-2026
Processing Time Days
146
Number Of Sites
4
Number Of Participants
6

Sites

Site Name
Gesellschaft Fuer Epilepsieforschung
Department Name
Neurology
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
Universitaetsklinikum Tuebingen AöR
Department Name
Neurology
Principal Investigator Name
Josua Kegele
Principal Investigator Email
Josua.Kegele@med.uni-tuebingen.de
Contact Person Name
Josua Kegele
Site Name
Universitaetsklinikum Bonn AöR
Department Name
Epileptology
Principal Investigator Name
Rainer Surges
Principal Investigator Email
rainer.surges@ukbonn.de
Contact Person Name
Rainer Surges
Contact Person Email
rainer.surges@ukbonn.de
Site Name
Universitaetsklinikum Frankfurt AöR
Department Name
Neurology
Principal Investigator Name
Felix Rosenow
Principal Investigator Email
felix.rosenow@kgu.de
Contact Person Name
Felix Rosenow
Contact Person Email
felix.rosenow@kgu.de

Spain

Earliest CTIS Part Ii Submission Date
09-09-2025
Latest Decision Or Authorization Date
13-02-2026
Processing Time Days
157
Number Of Sites
15
Number Of Participants
20

Sites

Site Name
Hospital Universitari Vall D Hebron
Department Name
Neurology
Principal Investigator Name
Manuel Toledo Argany
Principal Investigator Email
mtoledo@vhebron.net
Contact Person Name
Manuel Toledo Argany
Contact Person Email
mtoledo@vhebron.net
Site Name
Hospital De La Santa Creu I Sant Pau
Department Name
Neurology
Principal Investigator Name
Alba Sierra Marcos
Principal Investigator Email
asierram@santpau.cat
Contact Person Name
Alba Sierra Marcos
Contact Person Email
asierram@santpau.cat
Site Name
Hospital Vithas La Salud
Department Name
Neurology
Principal Investigator Name
Juan Carlos Sánchez Álvarez
Principal Investigator Email
sanchezajc@vithas.es
Contact Person Name
Juan Carlos Sánchez Álvarez
Contact Person Email
sanchezajc@vithas.es
Site Name
Hospital Universitario Virgen De Las Nieves
Department Name
Neurology
Principal Investigator Name
Violeta Sanchez Sanchez
Principal Investigator Email
violetasanchezsanchez@gmail.com
Contact Person Name
Violeta Sanchez Sanchez
Site Name
Hospital Universitario Regional De Malaga
Department Name
Neurology
Principal Investigator Name
Pedro Jesús Serrano Castro
Principal Investigator Email
p.serrano.eecc@gmail.com
Contact Person Name
Pedro Jesús Serrano Castro
Contact Person Email
p.serrano.eecc@gmail.com
Site Name
Fundacio Assistencial De Mutua De Terrassa Fpc
Department Name
Neurology
Principal Investigator Name
Belen Díaz Fernández
Principal Investigator Email
bdiaz@mutuaterrassa.cat
Contact Person Name
Belen Díaz Fernández
Contact Person Email
bdiaz@mutuaterrassa.cat
Site Name
Hospital La Milagrosa S.A.
Department Name
Neurology
Principal Investigator Name
Mar Carreño Martínez
Principal Investigator Email
maria.carreno@ext.vithas.es
Contact Person Name
Mar Carreño Martínez
Contact Person Email
maria.carreno@ext.vithas.es
Site Name
Hospital Universitario Fundacion Jimenez Diaz
Department Name
Neurology
Principal Investigator Name
José María Serratosa Fernández
Principal Investigator Email
JMSerratosa@fjd.es
Contact Person Name
José María Serratosa Fernández
Contact Person Email
JMSerratosa@fjd.es
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Neurology
Principal Investigator Name
Vicente Enrique Villanueva Haba
Principal Investigator Email
villanueva_vichab@gva.es
Contact Person Name
Vicente Enrique Villanueva Haba
Contact Person Email
villanueva_vichab@gva.es
Site Name
Hospital Universitario De La Princesa
Department Name
Neurology
Principal Investigator Name
María de Toledo las Heras
Principal Investigator Email
mdetoledo@yahoo.com
Contact Person Name
María de Toledo las Heras
Contact Person Email
mdetoledo@yahoo.com
Site Name
Hospital Del Mar
Department Name
Neurology
Principal Investigator Name
Rodrigo Alberto Rocamora Zúñiga
Principal Investigator Email
RRocamora@parcdesalutmar.cat
Contact Person Name
Rodrigo Alberto Rocamora Zúñiga
Contact Person Email
RRocamora@parcdesalutmar.cat
Site Name
Hospital Clinico San Carlos
Department Name
Neurology
Principal Investigator Name
Irene García Morales
Principal Investigator Email
garciamorales2@gmail.com
Contact Person Name
Irene García Morales
Contact Person Email
garciamorales2@gmail.com
Site Name
Hospital Clinico Universitario De Valladolid
Department Name
Neurology
Principal Investigator Name
Dulce María Campos Blanco
Principal Investigator Email
dmcampos@saludcastillayleon.es
Contact Person Name
Dulce María Campos Blanco
Contact Person Email
dmcampos@saludcastillayleon.es
Site Name
Hospital Clinic De Barcelona
Department Name
Neurology
Principal Investigator Name
María Centeno Soladana
Principal Investigator Email
centeno@clinic.cat
Contact Person Name
María Centeno Soladana
Contact Person Email
centeno@clinic.cat
Site Name
Hospital Blua Sanitas Valdebebas
Department Name
Neurology
Principal Investigator Name
Angel Aledo Serrano
Principal Investigator Email
aledo.ensayos@gmail.com
Contact Person Name
Angel Aledo Serrano
Contact Person Email
aledo.ensayos@gmail.com

Poland

Earliest CTIS Part Ii Submission Date
12-09-2025
Latest Decision Or Authorization Date
16-02-2026
Processing Time Days
157
Number Of Sites
6
Number Of Participants
10

Sites

Site Name
Centrum Medyczne Neuromed Sp. z o.o.
Department Name
Neurology
Principal Investigator Name
Pawel Lisewski
Principal Investigator Email
lisewski.p@gmail.com
Contact Person Name
Pawel Lisewski
Contact Person Email
lisewski.p@gmail.com
Site Name
Niepubliczny Zaklad Opieki Zdrowotnej Wielospecjalistyczna Poradnia Lekarska Synapsis Lech Szczechowski
Department Name
Neurology
Principal Investigator Name
Lech Szczechowski
Principal Investigator Email
lszczechiwski@gmail.com
Contact Person Name
Lech Szczechowski
Contact Person Email
lszczechiwski@gmail.com
Site Name
Novo-Med Zielinski I Wspolnicy Sp. j.
Department Name
Neurology
Principal Investigator Name
Tomasz Zielinski
Principal Investigator Email
tzielinski@op.pl
Contact Person Name
Tomasz Zielinski
Contact Person Email
tzielinski@op.pl
Site Name
Futuremeds Sp. z o.o.
Department Name
Neurology
Principal Investigator Name
Anatol Mickielewicz
Principal Investigator Email
anatol.mickielewicz@futuremeds.com
Contact Person Name
Anatol Mickielewicz
Site Name
Mtz Clinical Research Powered By Pratia
Department Name
Neurology
Principal Investigator Name
Zygmunt Jamrozik
Principal Investigator Email
badacz@pratia.com
Contact Person Name
Zygmunt Jamrozik
Contact Person Email
badacz@pratia.com
Site Name
Niepubliczny Zaklad Opieki Zdrowotnej Neuromed M. I M. Nastaj. sp.p.
Department Name
Neurology
Principal Investigator Name
Marcin Nastaj
Principal Investigator Email
marcinnastaj@gmail.com
Contact Person Name
Marcin Nastaj
Contact Person Email
marcinnastaj@gmail.com

Italy

Earliest CTIS Part Ii Submission Date
08-09-2025
Latest Decision Or Authorization Date
09-02-2026
Processing Time Days
154
Number Of Sites
11
Number Of Participants
20

Sites

Site Name
Azienda Ospedaliera Di Rilievo Nazionale Antonio Cardarelli
Department Name
Clinical Neurophysiology Department
Principal Investigator Name
Marianna Pezzella
Principal Investigator Email
marianna.pezzella@aocardarelli.it
Contact Person Name
Marianna Pezzella
Site Name
IRCCS Foundation Istituto Neurologico Carlo Besta
Department Name
SC Neurologia 7
Principal Investigator Name
Laura Canafoglia
Principal Investigator Email
Laura.Canafoglia@istituto-besta.it
Contact Person Name
Laura Canafoglia
Site Name
Azienda Ospedaliero-Universitaria Policlinico Umberto I
Department Name
Neurology Department
Principal Investigator Name
Carlo Di Bonaventura
Principal Investigator Email
c.dibonaventura@policlinicoumberto1.it
Contact Person Name
Carlo Di Bonaventura
Site Name
IRCCS Eugenio Medea - Sezione scientifica dell'Associazione "La Nostra Famiglia"
Department Name
Neurology Department
Principal Investigator Name
Alberto Danieli
Principal Investigator Email
alberto.danieli@lanostrafamiglia.it
Contact Person Name
Alberto Danieli
Site Name
Azienda Ospedaliero Universitaria Pisana
Department Name
U.O Neurologia
Principal Investigator Name
Chiara Pizzanelli
Principal Investigator Email
c.pizzanelli@ao-pisa.toscana.it
Contact Person Name
Chiara Pizzanelli
Site Name
Azienda Sanitaria Locale Della Provincia Di Biella
Department Name
S.C. Neurologia
Principal Investigator Name
Mario Coletti Moja
Principal Investigator Email
mario.colettimoia@aslbi.piemonte.it
Contact Person Name
Mario Coletti Moja
Site Name
Fondazione Istituto Neurologico Nazionale Casimiro Mondino
Department Name
U.O. Medicina del Sonno ed Epilessia
Principal Investigator Name
Elena Tartara
Principal Investigator Email
elena.tartara@mondino.it
Contact Person Name
Elena Tartara
Contact Person Email
elena.tartara@mondino.it
Site Name
Azienda Ospedaliero-Universitaria Di Cagliari
Department Name
Neurology Department
Principal Investigator Name
Monica Puligheddu
Principal Investigator Email
puligheddu@unica.it
Contact Person Name
Monica Puligheddu
Contact Person Email
puligheddu@unica.it
Site Name
Fondazione Policlinico Universitario Campus Bio-medico
Department Name
U.O.C. Neurologia
Principal Investigator Name
Giovanni Assenza
Principal Investigator Email
g.assenza@policlinicocampus.it
Contact Person Name
Giovanni Assenza
Contact Person Email
g.assenza@policlinicocampus.it
Site Name
Istituto Neurologico Mediterraneo Neuromed S.p.A.
Department Name
U.O. Epilessia
Principal Investigator Name
Pier Paolo Quarato
Principal Investigator Email
spleen333@libero.it
Contact Person Name
Pier Paolo Quarato
Contact Person Email
spleen333@libero.it
Site Name
Azienda Unita Sanitaria Locale Di Bologna
Department Name
Neurology Department
Principal Investigator Name
Lidia Di Vito
Principal Investigator Email
lidia.divito@ausl.bologna.it
Contact Person Name
Lidia Di Vito
Contact Person Email
lidia.divito@ausl.bologna.it

Sponsor

Primary sponsor

Full Name
Praxis Precision Medicines Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Medrio Inc.
Responsibilities
code: 7
Name
Pivotal S.L.
Responsibilities
codes: 1,12,13,2,5
Name
Suvoda LLC
Responsibilities
code: 3
Name
PPD Global Central Labs
Responsibilities
code: 4
Name
Mms Holdings Inc.
Responsibilities
code: 8
Name
Scout Clinical
Responsibilities
code: 15; Patient reimbursement of travel/meal expenses
Name
Emvenio Clinical Research LLC
Responsibilities
code: 15; Home Health Vendor /Telehealth

Third parties

  • {"country":"United States","full_name":"Medrio Inc.","duties_or_roles":"codes: 7","organisation_type":"Pharmaceutical company"}
  • {"country":"Spain","full_name":"Pivotal S.L.","duties_or_roles":"codes: 1,12,13,2,5","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"codes: 3","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"codes: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Mms Holdings Inc.","duties_or_roles":"codes: 8","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"code: 15; value: Patient reimbursement of travel/meal expenses","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Emvenio Clinical Research LLC","duties_or_roles":"code: 15; value: Home Health Vendor /Telehealth","organisation_type":"Laboratory/Research/Testing facility"}

Investigational products

Investigational Product Name
PRAX-628 (CAPSULE)
Active Substance
PRAX-628
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
1
Investigational Product Name
PRAX-628 (TABLET)
Active Substance
VORMATRIGINE
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
1
Investigational Product Name
PRAX-628 (TABLET)
Active Substance
VORMATRIGINE
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
1
Investigational Product Name
PRAX-628 (TABLET)
Active Substance
VORMATRIGINE
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
1

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