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: 2 countries
• Sites: 5

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 (5)

University of Kansas

Fairway, Kansas, 66205, United States

RECRUITING

Sean M. Healey & AMG Center for ALS

Boston, Massachusetts, 02114, United States

RECRUITING

Somnos Clinical Research

Lincoln, Nebraska, 68506, United States

RECRUITING

Texas Neurology

Dallas, Texas, 75206, United States

RECRUITING

Genge Partners

Montreal, Quebec, H4P 2N2, Canada

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

Executive Director Clinical Operations

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: April 24, 2026

Record last verified: 2026-04

Locations

US (4); CA (1)