RecruitingPhase 3NCT07322003

Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Amyotrophic Lateral Sclerosis

Sponsor

Prilenia

Enrollment

500 participants

Start Date

Feb 1, 2026

Study Type

INTERVENTIONAL

Conditions

Amyotrophic Lateral Sclerosis

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

Eligibility

Min Age: 18 YearsMax Age: 80 Years

Inclusion Criteria5

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.

Exclusion Criteria8

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.