Clinical trial • Phase III • Neurology|Rare Disease
PRIDOPIDINE HYDROCHLORIDE for Amyotrophic lateral sclerosis
Phase III trial of PRIDOPIDINE HYDROCHLORIDE for Amyotrophic lateral sclerosis.
Overview
- Trial Therapeutic Area
- Neurology|Rare Disease
- Trial Disease
- Amyotrophic lateral sclerosis
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 18-12-2025
- First CTIS Authorization Date
- 20-04-2026
Trial design
Randomised, pridopidine placebo (pridopidine placebo) — matching placebo control; dose/schedule for placebo not applicable / not specified in the record. Phase III trial in Belgium, France, Germany and others.
- Randomised
- Yes
- Comparator
- Pridopidine placebo (Pridopidine placebo) — matching placebo control; dose/schedule for placebo not applicable / not specified in the record.
- Target Sample Size
- 420
- Trial Duration For Participant
- 672
Eligibility
Recruits 420 isVulnerablePopulationSelected = true. Participants are adults aged 18–80. Exclusion criterion: "Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent and participate in the study" (i.e. participants unable to provide informed consent are excluded). Subject information sheets and informed consent forms (L1 SIS and ICF) are provided (multiple language versions available)..
- Vulnerable Population
- isVulnerablePopulationSelected = true. Participants are adults aged 18–80. Exclusion criterion: "Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent and participate in the study" (i.e. participants unable to provide informed consent are excluded). Subject information sheets and informed consent forms (L1 SIS and ICF) are provided (multiple language versions available).
Inclusion criteria
- {"criterion_text":"- Age 18 to 80 years (both inclusive), at the time of signing the informed consent"}
- {"criterion_text":"- Definite ALS or Probable ALS using the El Escorial criteria"}
- {"criterion_text":"- Symptom onset of ≤18 months at screening"}
- {"criterion_text":"- SVC greater or equal to 60% predicted"}
- {"criterion_text":"- Treatment Research Initiative to Cure ALS (TRICALS) Risk Profile Calculator score, based on the European Network for the Cure of ALS (ENCALS) survival prediction model in the range of -6 to -2, inclusive, at screening"}
Exclusion criteria
- {"criterion_text":"- Presence of tracheostomy or permanent assisted ventilation"}
- {"criterion_text":"- Clinically significant heart disease, clinically significant history of arrhythmia, symptomatic or uncontrolled atrial fibrillation despite treatment, or asymptomatic sustained ventricular tachycardia, or presence of left bundle branch block"}
- {"criterion_text":"- Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent and participate in the study"}
- {"criterion_text":"- Clinically significant and/or unstable medical condition (other than ALS) that may either pose a clinically meaningful risk to the participant and/or to study completion"}
- {"criterion_text":"- Clinically significant and/or unstable medical condition (other than ALS) that may either pose a clinically meaningful risk to the participant and/or to study completion"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Change from baseline through Week 26 in the ALSFRS-R total score adjusted for mortality.","definition_or_measurement_approach":"ALSFRS-R total score assessed at baseline and Week 26; analysis adjusted for mortality."}
- {"endpoint_text":"- Change from baseline through Week 48 in the ALSFRS-R total score adjusted for mortality.","definition_or_measurement_approach":"ALSFRS-R total score assessed at baseline and Week 48; analysis adjusted for mortality."}
Secondary endpoints
- {"endpoint_text":"- Overall survival at Week 96","definition_or_measurement_approach":"Overall survival assessed through Week 96 (time-to-event/survival outcome)."}
- {"endpoint_text":"- Change from baseline through Week 26, Week 48 in Speaking rate as measured by quantitative speech assessment in the clinic. Change from baseline through Week 48 in Intelligibility of speech as measured by quantitative speech assessment in the clinic.","definition_or_measurement_approach":"Quantitative speech assessments in clinic measuring speaking rate (Weeks 26 and 48) and intelligibility (through Week 48)."}
- {"endpoint_text":"- Change from baseline through Week 48 in percent predicted slow vital capacity","definition_or_measurement_approach":"Percent predicted slow vital capacity measured at baseline and Week 48."}
- {"endpoint_text":"- Change from baseline through Week 48 in the Bulbar subdomain of the ALSFRS-R","definition_or_measurement_approach":"Bulbar subdomain score of ALSFRS-R assessed at baseline and Week 48."}
- {"endpoint_text":"- Change from baseline through Week 48 in the ALSAQ-40","definition_or_measurement_approach":"ALSAQ-40 quality of life questionnaire score assessed at baseline and Week 48."}
- {"endpoint_text":"- Incidence, nature, and severity of treatment emergent adverse events, adverse events of special interest, and serious adverse events. Incidence and shifts of clinically significant abnormalities in ECG, laboratory tests, and vital signs. Change from baseline in C-SSRS. Tolerability.","definition_or_measurement_approach":"Safety assessments including AE reporting, ECG/laboratory/vital sign monitoring, C-SSRS suicidality scale changes, and tolerability measures."}
Recruitment
- Registry Or Advocacy Recruitment
- True, TRICALS Foundation Stichting (Treatment Research Initiative to Cure ALS / TRICALS) is named as providing patient recruitment and site training services
- Digital Remote Recruitment
- True — planned use of social media posts, online advertisements (banner ads), website content and online study materials to recruit participants; documents include social media/online advertisement materials and website content.
- Planned Sample Size
- 420
- Recruitment Window Months
- 34
- Consent Approach
- Informed consent is provided by the adult participant (eligible age 18–80). Subject information sheets and informed consent forms (L1_SIS and ICF) are available (multiple language versions listed, e.g. English, French, Dutch, Spanish, Polish). Pregnancy-specific ICFs / partner ICFs are present in documentation. Participants with cognitive impairment or other conditions that impair capacity to consent are excluded per criteria.
Methods
- Social media and online advertisements (documents: Social Media and Clinical Trial Posts; Online Advertisement Banners) — general public/patients
- Website content / UMCU website text concept — online information for potential participants
- Banner ads (online) — outreach to potential participants
- Patient brochures and participant study guides (patient-facing materials) — information for prospective participants
- Physician referral letters / physician referral materials — targeting clinicians to refer eligible patients
- Study brochures and 'About Clinical Trials' brochures — informational outreach to patients and clinicians
- Direct patient recruitment activities performed by TRICALS Foundation (patient recruitment and site training services)
Geography
- Total Number Of Sites
- 31
- Total Number Of Participants
- 420
Belgium
- Earliest CTIS Part Ii Submission Date
- 27-03-2026
- Latest Decision Or Authorization Date
- 27-04-2026
- Processing Time Days
- 31
- Number Of Sites
- 1
- Number Of Participants
- 25
Sites
- Site Name
- UZ Leuven
- Department Name
- Neurology
- Principal Investigator Name
- Philip Van Damme
- Principal Investigator Email
- Philip.vandamme@uzleuven.be
- Contact Person Name
- Philip Van Damme
- Contact Person Email
- Philip.vandamme@uzleuven.be
France
- Earliest CTIS Part Ii Submission Date
- 16-04-2026
- Latest Decision Or Authorization Date
- 24-04-2026
- Processing Time Days
- 8
- Number Of Sites
- 5
- Number Of Participants
- 60
Sites
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Neurologie
- Principal Investigator Name
- Gaelle BRUNETEAU
- Principal Investigator Email
- gaelle.bruneteau@aphp.fr
- Contact Person Name
- Gaelle BRUNETEAU
- Contact Person Email
- gaelle.bruneteau@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Neurologie
- Principal Investigator Name
- Veronique DANEL-BRUNAUD
- Principal Investigator Email
- veronique.danel@chu-lille.fr
- Contact Person Name
- Veronique DANEL-BRUNAUD
- Contact Person Email
- veronique.danel@chu-lille.fr
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Centre de Reference des Maladies Neuromusculaires rares et de la Sclérose Latérale Amyotrophique
- Principal Investigator Name
- Shahram ATTARIAN
- Principal Investigator Email
- shahram.attarian@ap-hm.fr
- Contact Person Name
- Shahram ATTARIAN
- Contact Person Email
- shahram.attarian@ap-hm.fr
- Site Name
- Centre Hospitalier Universitaire De Nice
- Department Name
- Neurologie
- Principal Investigator Name
- Marie-Hélène SORIANI
- Principal Investigator Email
- soriani.mh@chu-nice.fr
- Contact Person Name
- Marie-Hélène SORIANI
- Contact Person Email
- soriani.mh@chu-nice.fr
- Site Name
- Centre Hospitalier Regional Universitaire De Tours
- Department Name
- Neurologie
- Principal Investigator Name
- Philippe CORCIA
- Principal Investigator Email
- philippe.corcia@univ-tours.fr
- Contact Person Name
- Philippe CORCIA
- Contact Person Email
- philippe.corcia@univ-tours.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 13-04-2026
- Latest Decision Or Authorization Date
- 23-04-2026
- Processing Time Days
- 10
- Number Of Sites
- 6
- Number Of Participants
- 72
Sites
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Centrum für Neurologie, Neurochirurgie und Psychiatrie
- Principal Investigator Name
- Andre Maier
- Principal Investigator Email
- andre.maier@charite.de
- Contact Person Name
- Andre Maier
- Contact Person Email
- andre.maier@charite.de
- Site Name
- DIAKOVERE Krankenhaus gGmbH
- Department Name
- Klinik für Neurologie und Neurophysiologie
- Principal Investigator Name
- Susanne Petri
- Principal Investigator Email
- susanne.petri@diakovere.de
- Contact Person Name
- Susanne Petri
- Contact Person Email
- susanne.petri@diakovere.de
- Site Name
- Universitaetsklinikum Bonn AöR
- Department Name
- Klinik für Neurodegenerative Erkrankungen, Motoneuronambulanz
- Principal Investigator Name
- Patrick Weydt
- Principal Investigator Email
- patrick.weydt@ukbonn.de
- Contact Person Name
- Patrick Weydt
- Contact Person Email
- patrick.weydt@ukbonn.de
- Site Name
- Universitaetsklinikum Ulm AöR
- Department Name
- Klinik für Neurologie
- Principal Investigator Name
- Jochen Weishaupt
- Principal Investigator Email
- jochen.weishaupt@rku.de
- Contact Person Name
- Jochen Weishaupt
- Contact Person Email
- jochen.weishaupt@rku.de
- Site Name
- Universitaetsklinikum Schleswig-Holstein AöR
- Department Name
- Klinik für Neurologie, Neuromuskuläres Zentrum Schleswig-Holstein
- Principal Investigator Name
- Julian Großkreutz
- Principal Investigator Email
- julian.grosskreutz@uni-luebeck.de
- Contact Person Name
- Julian Großkreutz
- Contact Person Email
- julian.grosskreutz@uni-luebeck.de
- Site Name
- Universitaetsklinikum Jena KöR
- Department Name
- Klinik für Neurologie
- Principal Investigator Name
- Annekathrin Rödiger
- Principal Investigator Email
- annekathrin.roediger@med.uni-jena.de
- Contact Person Name
- Annekathrin Rödiger
- Contact Person Email
- annekathrin.roediger@med.uni-jena.de
Ireland
- Earliest CTIS Part Ii Submission Date
- 02-04-2026
- Latest Decision Or Authorization Date
- 24-04-2026
- Processing Time Days
- 22
- Number Of Sites
- 1
- Number Of Participants
- 25
Sites
- Site Name
- Beaumont Hospital
- Department Name
- Neurology
- Principal Investigator Name
- Orla Hardiman
- Principal Investigator Email
- HARDIMAO@tcd.ie
- Contact Person Name
- Orla Hardiman
- Contact Person Email
- HARDIMAO@tcd.ie
Italy
- Earliest CTIS Part Ii Submission Date
- 13-04-2026
- Latest Decision Or Authorization Date
- 20-04-2026
- Processing Time Days
- 7
- Number Of Sites
- 6
- Number Of Participants
- 72
Sites
- Site Name
- Azienda Ospedaliera di Padova
- Department Name
- Centro Regionale Malattie del Motoneurone, UOC Clinica Neurologica Padova
- Principal Investigator Name
- Gianni Sorarù
- Principal Investigator Email
- gianni.soraru@unipd.it
- Contact Person Name
- Gianni Sorarù
- Contact Person Email
- gianni.soraru@unipd.it
- Site Name
- Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
- Department Name
- SC Neurologia 1 U
- Principal Investigator Name
- Adriano Chiò
- Principal Investigator Email
- adriano.chio@unito.it
- Contact Person Name
- Adriano Chiò
- Contact Person Email
- adriano.chio@unito.it
- Site Name
- Azienda Ospedaliero Universitaria Di Modena
- Department Name
- Ospedale Civile di Baggiovara, Struttura Complessa di Neurologia
- Principal Investigator Name
- Jessica Mandrioli
- Principal Investigator Email
- jessica.mandrioli@unimore.it
- Contact Person Name
- Jessica Mandrioli
- Contact Person Email
- jessica.mandrioli@unimore.it
- Site Name
- Centro Clinico Nemo
- Department Name
- Centro Clinico NeMO di Milano
- Principal Investigator Name
- Federica Cerri
- Principal Investigator Email
- federica.cerri@centrocliniconemo.it
- Contact Person Name
- Federica Cerri
- Contact Person Email
- federica.cerri@centrocliniconemo.it
- Site Name
- Istituto Auxologico Italiano
- Department Name
- Ospedale San Luca, Neurology Unit
- Principal Investigator Name
- Nicola Ticozzi
- Principal Investigator Email
- n.ticozzi@auxologico.it
- Contact Person Name
- Nicola Ticozzi
- Contact Person Email
- n.ticozzi@auxologico.it
- Site Name
- Azienda Ospedaliera Universitaria Universita' Degli Studi Della Campania Luigi Vanvitelli
- Department Name
- Dipartimento Assistenziale di Medicina Interna, Geriatria e Neurologia
- Principal Investigator Name
- Francesca Trojsi
- Principal Investigator Email
- francesca.trojsi@unicampania.it
- Contact Person Name
- Francesca Trojsi
- Contact Person Email
- francesca.trojsi@unicampania.it
Spain
- Earliest CTIS Part Ii Submission Date
- 08-04-2026
- Latest Decision Or Authorization Date
- 22-04-2026
- Processing Time Days
- 14
- Number Of Sites
- 6
- Number Of Participants
- 60
Sites
- Site Name
- Bellvitge University Hospital
- Department Name
- Neurology
- Principal Investigator Name
- Monica Povedano Panades
- Principal Investigator Email
- mpovedano@bellvitgehospital.cat
- Contact Person Name
- Monica Povedano Panades
- Contact Person Email
- mpovedano@bellvitgehospital.cat
- Site Name
- Complexo Hospitalario Universitario De Santiago
- Department Name
- Neurology
- Principal Investigator Name
- Beatrice Canneti Heredia
- Principal Investigator Email
- canneti.heredia@gmail.com
- Contact Person Name
- Beatrice Canneti Heredia
- Contact Person Email
- canneti.heredia@gmail.com
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Neurology
- Principal Investigator Name
- Juan Francisco Vazquez Costa
- Principal Investigator Email
- vazquez_juacos@gva.es
- Contact Person Name
- Juan Francisco Vazquez Costa
- Contact Person Email
- vazquez_juacos@gva.es
- Site Name
- Hospital Del Mar
- Department Name
- Neurology
- Principal Investigator Name
- Miguel Angel Rubio Perez
- Principal Investigator Email
- marubio@psmar.cat
- Contact Person Name
- Miguel Angel Rubio Perez
- Contact Person Email
- marubio@psmar.cat
- Site Name
- Hospital Carlos III
- Department Name
- Neurology
- Principal Investigator Name
- Javier Pedro Mascías Cadavid
- Principal Investigator Email
- jmascias@yahoo.es
- Contact Person Name
- Javier Pedro Mascías Cadavid
- Contact Person Email
- jmascias@yahoo.es
- Site Name
- Hospital (additional listed site)
- Department Name
- Neurology
Sweden
- Earliest CTIS Part Ii Submission Date
- 31-03-2026
- Latest Decision Or Authorization Date
- 20-04-2026
- Processing Time Days
- 20
- Number Of Sites
- 2
- Number Of Participants
- 36
Sites
- Site Name
- Region Stockholm – SLSO
- Department Name
- ALS Clinic Karolinska University Hospital
- Principal Investigator Name
- Caroline Ingre
- Principal Investigator Email
- Caroline.Ingre@regionstockholm.se
- Contact Person Name
- Caroline Ingre
- Contact Person Email
- Caroline.Ingre@regionstockholm.se
- Site Name
- Region Vaesterbotten
- Department Name
- Department of Neurology A41
- Principal Investigator Name
- Peter Andersen
- Principal Investigator Email
- peter.andersen@umu.se
- Contact Person Name
- Peter Andersen
- Contact Person Email
- peter.andersen@umu.se
Netherlands
- Earliest CTIS Part Ii Submission Date
- 03-04-2026
- Latest Decision Or Authorization Date
- 20-04-2026
- Processing Time Days
- 17
- Number Of Sites
- 1
- Number Of Participants
- 25
Sites
- Site Name
- Universitair Medisch Centrum Utrecht
- Department Name
- Neurology
- Principal Investigator Name
- Henk-Jan Westeneng
- Principal Investigator Email
- h.j.westeneng@umcutrecht.nl
- Contact Person Name
- Henk-Jan Westeneng
- Contact Person Email
- h.j.westeneng@umcutrecht.nl
Poland
- Earliest CTIS Part Ii Submission Date
- 14-04-2026
- Latest Decision Or Authorization Date
- 23-04-2026
- Processing Time Days
- 9
- Number Of Sites
- 3
- Number Of Participants
- 45
Sites
- Site Name
- City Clinic Research Sp. z o.o.
- Principal Investigator Name
- Magdalena Kuźma-Kozakiewicz
- Principal Investigator Email
- magdalena.kuzma-kozakiewicz@wum.edu.pl
- Contact Person Name
- Magdalena Kuźma-Kozakiewicz
- Contact Person Email
- magdalena.kuzma-kozakiewicz@wum.edu.pl
- Site Name
- Linden Sp. z o.o. sp.k.
- Principal Investigator Name
- Jakub Antczak
- Principal Investigator Email
- jakub.antczak@uj.edu.pl
- Contact Person Name
- Jakub Antczak
- Contact Person Email
- jakub.antczak@uj.edu.pl
- Site Name
- Centrum Medyczne Neuromed Sp. z o.o.
- Principal Investigator Name
- Pawel Lisiewski
- Principal Investigator Email
- lisewski.p@gmail.com
- Contact Person Name
- Pawel Lisiewski
- Contact Person Email
- lisewski.p@gmail.com
Sponsor
Primary sponsor
- Full Name
- Ferrer Internacional S.A.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Spain
Contract research organisations
- Name
- Iqvia Biotech Limited
- Responsibilities
- Multiple sponsor duties including ECG and French patients reimbursement (various codes listed). Contact: Biotech-EUCTR-Support@iqvia.com
- Name
- Iqvia Laboratories Limited
- Responsibilities
- Laboratory activities (sponsor duty code 4). Contact: q2_eu_clinical_trials_information@q2labsolutions.com
- Name
- Greenphire LLC
- Responsibilities
- Patient reimbursement and travel arrangements
- Name
- Marken LLP
- Responsibilities
- Direct-to-home shipments
- Name
- Medidata Solutions Inc.
- Responsibilities
- Data/Medidata (sponsor duty code 7)
- Name
- Fisher Clinical Services GmbH
- Responsibilities
- Clinical services / logistics (code 14 indicated)
- Name
- Primevigilance Limited
- Responsibilities
- Pharmacovigilance
- Name
- Bioforum C.D.M.C Ltd.
- Responsibilities
- Biostatistics services
- Name
- Redenlab Pty Limited
- Responsibilities
- Speech assessment
- Name
- Ppd Inc.
- Responsibilities
- PK assessment
Third parties
- {"country":"United Kingdom","full_name":"Iqvia Biotech Limited","duties_or_roles":"codes: 1,11,12,13,15 (value: \"ECG, French patients reimbursement\"),2,5,6,8,9","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Iqvia Laboratories Limited","duties_or_roles":"code: 4","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient Reimbursement, Travel arrangements","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Marken LLP","duties_or_roles":"Direct to home shipments","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"code: 7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Germany","full_name":"Fisher Clinical Services GmbH","duties_or_roles":"code: 14","organisation_type":"Pharmaceutical company"}
- {"country":"Israel","full_name":"Bioforum C.D.M.C Ltd.","duties_or_roles":"Biostatistics services","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Primevigilance Limited","duties_or_roles":"Pharmacovigilance","organisation_type":"Pharmaceutical company"}
- {"country":"Australia","full_name":"Redenlab Pty Limited","duties_or_roles":"Speech assessment","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"TRICALS Foundation Stichting","duties_or_roles":"Patient Recruitment and Site Training services","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Ppd Inc.","duties_or_roles":"PK assessment","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Fisher Clinical Services GmbH (additional address)","duties_or_roles":"code: 14","organisation_type":"Pharmaceutical company"}
Co-sponsors
- Prilenia Therapeutics B.V.
Investigational products
- Investigational Product Name
- PRIDOPIDINE
- Active Substance
- PRIDOPIDINE HYDROCHLORIDE
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL USE
- Orphan Designation
- Yes
- Maximum Dose
- 90 mg
- Investigational Product Name
- Pridopidine placebo
- Modality
- Other
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