Clinical Research Alert: Phase 3 Study of Pridopidine in Individuals with ALS

Researchers at participating clinical research sites are seeking individuals with amyotrophic lateral sclerosis (ALS) for a phase 3 study (PREVAiLS). This study will evaluate the safety and efficacy of the investigational drug pridopidine to treat ALS. Pridopidine, which is being developed by Prilenia Therapeutics and Ferrer, has shown neuroprotective effects in multiple preclinical studies, as well as beneficial effects across secondary and exploratory endpoints in specific patient types in a Phase 2 clinical trial. It is being studied for the ability to help maintain proper neuronal function and delay neurodegeneration in people with ALS.

The study

This is a randomized, double-blind, placebo-controlled study with an open-label extension (OLE). This means that participants will be randomly assigned to receive either pridopidine or an inactive placebo control during the double-blind part of the study (48 weeks). The randomization ratio is 3:2, meaning that for every 3 participants assigned to active treatment 2 participants will be assigned to placebo. Following this part of the trial, all participants will be given the active study drug during the OLE (another 48 weeks). During the double-blind period, participants will visit the clinic 7 times (including screening and end of study visits/transition to OLE). Five phone visits will be conducted between clinic visits. During the OLE period, participants will visit the clinic an additional 4 times over the course of the year, with an additional 4 phone visits.

The drug/placebo will be administered orally (by mouth) twice daily. The effect of pridopidine will be evaluated by a number of tests and assessments including, but not limited to: ALS functional rating assessment (ALSFRS-R), speech recording, respiratory function testing, and quality of life. Participants will also have regular physical examinations, recording of vital signs, ECGs, and blood testing.

Study criteria

To be eligible, individuals must meet the following inclusion criteria:
• 18-80 years of age
• Diagnosis of definite or probable ALS (by El Escorial Criteria)
• 18 months or less from onset of symptoms
• SVC (slow vital capacity, a respiratory function test) >=60%
• Currently able to swallow capsules

Individuals may not be eligible to participate if they meet the following exclusion criteria:
• Presence of tracheostomy or permanent assisted ventilation (PAV)
• Certain mutations confirmed by genetic analysis
• Prior treatment with pridopidine, gene therapy or antisense oligonucleotides (ASOs)

Participants may continue using certain approved ALS medications, such as riluzole, edaravone and dextromethorphan/quinidine.

Interested in participating?

To learn more or inquire about participation, visit the sponsor’s website.