Pridopidine for adults with early, rapidly progressing ALS

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Barrow Neurological Institute
  Phoenix, Arizona, United States Recruiting
• California Pacific Medical Center
  San Francisco, California, United States Recruiting
• University of Colorado CU Anschutz Neurology
  Aurora, Colorado, United States Recruiting
• Mayo Clinic
  Jacksonville, Florida, United States Recruiting
• University of South Florida
  Tampa, Florida, United States Recruiting
• Emory University
  Atlanta, Georgia, United States Recruiting

+6 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Prilenia Medical Information
• Email: MedInfo@prilenia.com
• Phone: 857-574-5755

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.