Pridopidine for adults with early, rapidly progressing ALS

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

This trial will see if taking pridopidine 45 mg twice daily can slow disease progression in adults with early, rapidly progressing ALS.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 500 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Prilenia (Industry-sponsored)
• Locations: 5 sites (Fairway, Kansas and 4 other locations)
• Trial ID: NCT07322003 on ClinicalTrials.gov

What this trial studies

This Phase 3, randomized, double-blind, placebo-controlled study enrolls adults with early, rapidly progressing ALS who meet El Escorial criteria. Participants are randomized 3:2 to receive oral pridopidine 45 mg twice daily or placebo for 48 weeks, followed by a 48-week open-label extension where all participants receive pridopidine while the original blind is maintained. Standard ALS treatments (e.g., riluzole, edaravone, Nuedexta) are allowed if doses are stable, and participants will attend regular on-site visits and some virtual visits approximately every 1–3 months. Total participation is about 102 weeks including screening and follow-up.

Who should consider this trial

Good fit: Adults with definite or probable ALS by El Escorial criteria, symptom onset ≤18 months, SVC >=60% predicted, a TRICALS/ENCALS risk score between -6 and -2, able to swallow a capsule, and without tracheostomy or permanent assisted ventilation are ideal candidates.

Not a fit: Patients with advanced respiratory failure, tracheostomy or permanent assisted ventilation, significant uncontrolled cardiac arrhythmias, unstable psychiatric or cognitive impairment, or symptom onset beyond 18 months may be unlikely to benefit or are excluded.

Why it matters

Potential benefit: If successful, pridopidine could slow ALS progression and help preserve muscle function and independence for longer in affected adults.

How similar studies have performed: Preclinical data and early-stage clinical work have suggested potential neuroprotective effects of pridopidine, but its benefit in ALS has not yet been confirmed in a large Phase 3 trial.

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.

Where this trial is running

Fairway, Kansas and 4 other locations

• University of Kansas — Fairway, Kansas, United States (Recruiting)
• Sean M. Healey & AMG Center for ALS — Boston, Massachusetts, United States (Recruiting)
• Somnos Clinical Research — Lincoln, Nebraska, United States (Recruiting)
• Texas Neurology — Dallas, Texas, United States (Recruiting)
• Genge Partners — Montreal, Quebec, Canada (Recruiting)

Study contacts

• Study coordinator: Executive Director Clinical Operations

Conditions Amyotrophic Lateral Sclerosis