Efficacy and Safety of Pridopidine in Participants with Amyotrophic Lateral Sclerosis: A Phase 3, Randomized, Double-blind, Placebo-controlled Study

The primary objective of this study is to evaluate the efficacy of pridopidine in modulating disease progression in participants with amyotrophic lateral sclerosis (5). Secondary objectives include the assessment of survival, speech, respiratory function, and bulbar function. Additionally, the study aims to evaluate the impact of the investigational product on quality of life and to monitor safety and tolerability (4).

The study involves 80 participants diagnosed with Amyotrophic Lateral Sclerosis. The study population consists of both male and female patients. Eligible individuals are between the ages of 18 and 80 years at the time of informed consent. Inclusion requires a diagnosis of definite ALS or probable ALS according to the El Escorial criteria. Participants must have a symptom onset of 18 months or less at the time of screening. Additional requirements include a forced vital capacity of 60% or greater of the predicted value and a specific score on the TRICALS Risk Profile Calculator, which is based on the ENCALS survival prediction model.

This Phase 3, randomized, double-blind, placebo-controlled study is designed to evaluate the efficacy and safety of pridopidine in participants diagnosed with amyotrophic lateral sclerosis. The investigation focuses on the impact of the study drug on disease progression. Participants will be assigned to receive either 90 mg of pridopidine hydrochloride in a hard capsule or a placebo via oral use. The methodology involves a screening visit to assess eligibility based on specific criteria, including age, clinical diagnosis, symptom onset, and respiratory function. Following screening, participants will undergo scheduled study visits to monitor primary and secondary endpoints, such as the ALSFRS-R total score, overall survival, and speech assessments. The study includes assessments of slow vital capacity, bulbar function, and tolerability. Clinical monitoring will track the incidence of adverse events and changes in electrocardiogram findings, laboratory tests, and vital signs.

The experimental treatment consists of pridopidine hydrochloride administered in a hard capsule pharmaceutical form. The prescribed dosage is 90 mg, which is delivered via the oral route.

The control group receives a placebo, designated as pridopidine placebo, to maintain the double-blind design of the study.

The efficacy of pridopidine in participants with Amyotrophic Lateral Sclerosis is evaluated through several primary and secondary endpoints. The primary assessment consists of the change from baseline in the ALSFRS-R total score, adjusted for mortality, at Week 26 and Week 48.

Secondary endpoints include:

• Overall survival at Week 96.
• Changes from baseline in speaking rate and intelligibility of speech, measured via quantitative speech assessment in a clinic setting at Week 26 and Week 48.
• Change from baseline in percent predicted slow vital capacity through Week 48.
• Change from baseline in the Bulbar subdomain of the ALSFRS-R through Week 48.
• Change from baseline in the ALSAQ-40 through Week 48.