Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS

NCT07322003 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: PL101-ALS301 / FNP254-CT-2501
• Study Type: interventional
• Target: 500
• Locations: 13 countries
• Sites: 56

Brief Summary

The goal of this clinical trial is to learn if the drug pridopidine works to treat amyotrophic lateral sclerosis in adults. It will also help to learn about the safety of pridopidine. The main question it aims to answer is: Does pridopidine slow disease progression of ALS? Researchers will compare pridopidine to a placebo (a look-alike substance that contains no drug) to see if pridopidine works to treat ALS. Participants will: Take pridopidine or a placebo by mouth every day for 48 weeks. Afterwards, all participants will take pridopidine for another 48 weeks. Visit the clinic once every 1-3 months for checkups and tests

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Change from baseline through Week 26 and Week 48 in the Revised ALS Functional Rating Scale (ALSFRS-R) total score adjusted for mortality

  The ALSFRS-R encompasses 12 questions grouped into 4 domains to assess bulbar symptoms, limb and trunk functionality, respiratory symptoms, and the need for percutaneous endoscopic gastrostomy (PEG), non-invasive ventilation, or tracheostomy with invasive ventilation. Each function is scored from 0 (no ability) to 4 (normal), with a minimum total score of 0 and maximum total score of 48. Higher scores indicate better functioning.

  Baseline to Week 48

Secondary Outcomes (6)

• Overall survival at Week 96

  Baseline to Week 96

• Change from baseline through Week 26 and Week 48 in speaking rate as measured by quantitative speech assessment in the clinic

  Baseline to Week 48

• Change from baseline through Week 48 in intelligibility of speech by quantitative speech assessment in the clinic.

  Baseline to Week 48.

• Change from baseline through Week 48 in percent predicted slow vital capacity (SVC)

  Baseline to Week 48

• Change from baseline through Week 48 in the Bulbar subdomain of the ALSFRS-R

  Baseline to Week 48

Study Arms (2)

Pridopidine

EXPERIMENTAL

Pridopidine hard gelatin capsules of 45 mg strength. During titration period 1 capsule taken orally in the morning for 2 weeks. During the main treatment period, 1 capsule in the morning and 1 capsule in the afternoon taken orally. Total daily dose of 90 mg.

Drug: Pridopidine

Placebo

PLACEBO COMPARATOR

Placebo hard gelatin capsules. During titration period 1 capsule taken orally in the morning for 2 weeks. During the main treatment period, 1 capsule in the morning and 1 capsule in the afternoon taken orally.

Drug: Placebo

Interventions

Pridopidine DRUG

Pridopidine hard gelatin capsule.

Pridopidine

Placebo DRUG

Placebo hard gelatin capsule.

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Prilenia: lead
• Ferrer Internacional S.A.: collaborator

Study Sites (56)

Barrow Neurological Institute

Phoenix, Arizona, 85013, United States

RECRUITING

California Pacific Medical Center

San Francisco, California, 94109, United States

RECRUITING

University of Colorado CU Anschutz Neurology

Aurora, Colorado, 80045, United States

RECRUITING

Mayo Clinic

Jacksonville, Florida, 32224, United States

RECRUITING

University of South Florida

Tampa, Florida, 33612, United States

RECRUITING

Emory University

Atlanta, Georgia, 30322, United States

RECRUITING

Northwestern University

Chicago, Illinois, 60611, United States

RECRUITING

University of Kansas

Fairway, Kansas, 66205, United States

RECRUITING

Johns Hopkins University

Baltimore, Maryland, 21205, United States

RECRUITING

Sean M. Healey & AMG Center for ALS

Boston, Massachusetts, 02114, United States

RECRUITING

Washington University

St Louis, Missouri, 63110, United States

RECRUITING

Somnos Clinical Research

Lincoln, Nebraska, 68506, United States

RECRUITING

Eleanor and Lou Gehrig ALS Center at Columbia University

New York, New York, 10032, United States

RECRUITING

Neuroscience Department at LKSM at Temple University

Philadelphia, Pennsylvania, 19140, United States

RECRUITING

Texas Neurology

Dallas, Texas, 75206, United States

RECRUITING

Baylor College of Medicine; McNair Medical Campus

Houston, Texas, 77030, United States

RECRUITING

Swedish Medical Center

Seattle, Washington, 98122, United States

RECRUITING

University of Washington

Seattle, Washington, 98195, United States

RECRUITING

UZ Leuven

Leuven, 3000, Belgium

RECRUITING

Genge Partners

Montreal, Quebec, H4P 2N2, Canada

RECRUITING

University of Alberta

Edmonton, Canada

NOT YET RECRUITING

McMaster University Medical Centre

Hamilton, Canada

NOT YET RECRUITING

CHU Lille

Lille, France

NOT YET RECRUITING

Hôpitaux Universitaires de Marseille Timone

Marseille, France

NOT YET RECRUITING

Centre Hospitalier Universitaire de Nice

Nice, France

RECRUITING

Hospitalier Pitié-Salpêtrière

Paris, 75013, France

RECRUITING

Centre hospitalier régional universitaire de de Tours

Tours, France

RECRUITING

ALS outpatient department at Charité - university medicine Berlin

Berlin, 13353, Germany

RECRUITING

ALS outpatient department at Charité - university medicine Berlin

Berlin, Germany

RECRUITING

ALS Clinic Bonn University

Bonn, 53127, Germany

RECRUITING

University Hospital Jena

Jena, Germany

RECRUITING

Universitätsklinikum Schleswig-Holstein

München, Germany

RECRUITING

University of Ulm

Ulm, 89081, Germany

RECRUITING

Trinity College Dublin

Dublin, Ireland

NOT YET RECRUITING

Hadassah Medical Center

Jerusalem, IL 91120, Israel

RECRUITING

Tel Aviv Sourasky Medical Center

Tel Aviv, 6423906, Israel

RECRUITING

University of Campania "Luigi Vanvitelli"

Caserta, Italy

NOT YET RECRUITING

Centro Clinico NEMO Fondazione Serena Onlus - ASST Niguarda Hospital

Milan, 20126, Italy

RECRUITING

Istituto Auxologico Italiano Ospedale San Luca

Milan, Italy

RECRUITING

Azienda Ospedaliero Universitaria Di Modena

Modena, Italy

RECRUITING

University of Padova

Padova, Italy

NOT YET RECRUITING

University of Torino

Torino, Italy

NOT YET RECRUITING

UMC Utrecht

Utrecht, Netherlands

RECRUITING

Centrum Medyczne Neuromed

Bydgoszcz, 85-163, Poland

RECRUITING

Linden sp. z o. o. sp. k.

Krakow, 30-105, Poland

RECRUITING

City Clinic Research Sp. z o.o.

Warsaw, 02-473, Poland

RECRUITING

Hospital del Mar

Barcelona, Spain

RECRUITING

University Hospital of Bellvitge

Barcelona, Spain

RECRUITING

Hospital Universitario La Paz - Carlos III

Madrid, 28029, Spain

RECRUITING

Clinical Hospital Santiago de Compostela

Santiago de Compostela, 15706, Spain

RECRUITING

Hospital Universitari i Politècnic La Fe

Valencia, 46026, Spain

RECRUITING

Studieenheten Akademiskt specialistcentrum

Stockholm, 113 61, Sweden

RECRUITING

Umeå University Hospital

Umeå, 901 85, Sweden

RECRUITING

King's College Hospital

London, United Kingdom

RECRUITING

MDN Centre Queen Square Institute of Neurology

London, United Kingdom

NOT YET RECRUITING

Royal Stoke University Hospital

Stoke-on-Trent, ST4 6QG, United Kingdom

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

pridopidine

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Prilenia Medical Information

CONTACT

857-574-5755; MedInfo@prilenia.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Randomized (3:2), double-blind, placebo-controlled study followed by an open-label extension period. During the open-label extension, all participants will receive pridopidine.

Study Record Dates

• First Submitted: January 5, 2026
• First Posted: January 7, 2026
• Study Start: February 1, 2026
• Primary Completion (Estimated): March 1, 2028
• Study Completion (Estimated): March 1, 2029
• Last Updated: July 29, 2026

Record last verified: 2026-06

Locations

ES (5); IE (1); SE (2); FR (5); NL (1); US (18); BE (1); DE (6); GB (3); IT (6); IL (2); CA (3); PL (3)