RecruitingRecruiting
Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS
NCT07322003 · Prilenia
In plain English
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Official title
A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Amyotrophic Lateral Sclerosis
About this study
This is a Phase 3, randomized study consisting of a double-blind placebo-controlled (DBPC) period followed by an open-label extension (OLE) to evaluate the efficacy and safety of pridopidine administered orally at a dose of 45 mg twice a day in adult participants with early and rapidly progressing ALS. Standard of care treatments (e.g. riluzole, edaravone and Nuedexta) will be allowed as long as participants are on a stable dose for at least 4 weeks prior to dosing. In the DBPC period, participants will be randomized in a 3:2 ratio to the pridopidine and placebo arms.
In the DBPC period, participants will receive pridopidine or placebo for 48 weeks. In the OLE period, all participants will receive pridopidine for 48 weeks, while maintaining the blind to their original randomization for both the participant as well as the Investigator and other clinical staff. The total study duration per participant will be 102 weeks including screening and follow up.
Throughout the study, participants will be assessed through on-site clinic visits and virtual visits (via telephone).
Eligibility criteria
Key Inclusion Criteria:
* Definite ALS or Probable ALS using the El Escorial criteria.
* Symptom onset of ≤18 months at screening.
* Slow vital capacity (SVC) greater or equal to 60% predicted.
* 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.
* Able to swallow a capsule.
Key Exclusion Criteria:
* Presence of tracheostomy or permanent assisted ventilation.
* 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.
* 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.
* 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.
* Use of medications that prolong QT interval.
* Previous treatment with pridopidine, gene therapy, or antisense oligonucleotides.
* Confirmed mutation in the SOD1, FUS or C9orf72 gene.
* Pregnancy.
Study design
Enrollment target: 500 participants
Allocation: randomized
Masking: quadruple
Age groups: adult, older_adult
Timeline
Starts: 2026-02-01
Estimated completion: 2029-03
Last updated: 2026-07-14
Interventions
Drug: PridopidineDrug: Placebo
Primary outcomes
- • Change from baseline through Week 26 and Week 48 in the Revised ALS Functional Rating Scale (ALSFRS-R) total score adjusted for mortality (Baseline to Week 48)
Sponsor
Prilenia · industry
With: Ferrer Internacional S.A.
Contacts & investigators
ContactPrilenia Medical Information · contact · MedInfo@prilenia.com · 857-574-5755
All locations (36)
California Pacific Medical CenterRecruiting
San Francisco, California, United States
Mayo ClinicRecruiting
Jacksonville, Florida, United States
University of South FloridaRecruiting
Tampa, Florida, United States
Emory UniversityRecruiting
Atlanta, Georgia, United States
Northwestern UniversityRecruiting
Chicago, Illinois, United States
University of KansasRecruiting
Fairway, Kansas, United States
Sean M. Healey & AMG Center for ALSRecruiting
Boston, Massachusetts, United States
Washington UniversityRecruiting
St Louis, Missouri, United States
Somnos Clinical ResearchRecruiting
Lincoln, Nebraska, United States
Eleanor and Lou Gehrig ALS Center at Columbia UniversityRecruiting
New York, New York, United States
Neuroscience Department at LKSM at Temple UniversityRecruiting
Philadelphia, Pennsylvania, United States
Texas NeurologyRecruiting
Dallas, Texas, United States
Baylor College of Medicine; McNair Medical CampusRecruiting
Houston, Texas, United States
Swedish Medical CenterRecruiting
Seattle, Washington, United States
University of WashingtonRecruiting
Seattle, Washington, United States
UZ LeuvenRecruiting
Leuven, Belgium
Genge PartnersRecruiting
Montreal, Quebec, Canada
Hospitalier Pitié-SalpêtrièreRecruiting
Paris, France
ALS outpatient department at Charité - university medicine BerlinRecruiting
Berlin, Germany
ALS Clinic Bonn UniversityRecruiting
Bonn, Germany
University Hospital JenaRecruiting
Jena, Germany
University of UlmRecruiting
Ulm, Germany
Hadassah Medical CenterRecruiting
Jerusalem, Israel
Tel Aviv Sourasky Medical CenterRecruiting
Tel Aviv, Israel
Centro Clinico NEMO Fondazione Serena Onlus - ASST Niguarda HospitalRecruiting
Milan, Italy
Istituto Auxologico Italiano Ospedale San LucaRecruiting
Milan, Italy
UMC UtrechtRecruiting
Utrecht, Netherlands
Centrum Medyczne NeuromedRecruiting
Bydgoszcz, Poland
Linden sp. z o. o. sp. k.Recruiting
Krakow, Poland
City Clinic Research Sp. z o.o.Recruiting
Warsaw, Poland
Hospital Universitario La Paz - Carlos IIIRecruiting
Madrid, Spain
Clinical Hospital Santiago de CompostelaRecruiting
Santiago de Compostela, Spain
Hospital Universitari i Politècnic La FeRecruiting
Valencia, Spain
Studieenheten Akademiskt specialistcentrumRecruiting
Stockholm, Sweden
Umeå University HospitalRecruiting
Umeå, Sweden
Royal Stoke University HospitalRecruiting
Stoke-on-Trent, United Kingdom