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Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS
NCT07322003 · Prilenia
In plain English
Click the button to translate this study into plain language — what it is, who qualifies, and what participation looks like.
Official title
A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Amyotrophic Lateral Sclerosis
About this study
This is a Phase 3, randomized study consisting of a double-blind placebo-controlled (DBPC) period followed by an open-label extension (OLE) to evaluate the efficacy and safety of pridopidine administered orally at a dose of 45 mg twice a day in adult participants with early and rapidly progressing ALS. Standard of care treatments (e.g. riluzole, edaravone and Nuedexta) will be allowed as long as participants are on a stable dose for at least 4 weeks prior to dosing. In the DBPC period, participants will be randomized in a 3:2 ratio to the pridopidine and placebo arms.
In the DBPC period, participants will receive pridopidine or placebo for 48 weeks. In the OLE period, all participants will receive pridopidine for 48 weeks, while maintaining the blind to their original randomization for both the participant as well as the Investigator and other clinical staff. The total study duration per participant will be 102 weeks including screening and follow up.
Throughout the study, participants will be assessed through on-site clinic visits and virtual visits (via telephone).
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.
Study design
Enrollment target: 500 participants
Allocation: randomized
Masking: quadruple
Age groups: adult, older_adult
Timeline
Starts: 2026-02-01
Estimated completion: 2029-03
Last updated: 2026-02-27
Interventions
Drug: PridopidineDrug: Placebo
Primary outcomes
- • Change from baseline through Week 26 and Week 48 in the Revised ALS Functional Rating Scale (ALSFRS-R) total score adjusted for mortality (Baseline to Week 48)
Sponsor
Prilenia · industry
With: Ferrer Internacional S.A.
Contacts & investigators
ContactExecutive Director Clinical Operations · contact · MedInfo@prilenia.com · 857-574-5755
All locations (3)
Sean M. Healey & AMG Center for ALSRecruiting
Boston, Massachusetts, United States
Somnos Clinical ResearchRecruiting
Lincoln, Nebraska, United States
Texas NeurologyRecruiting
Dallas, Texas, United States