Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS

Condition(s) studied

Investigational drug(s) / intervention(s)

Pridopidine: Pridopidine hard gelatin capsule.

Placebo: Placebo hard gelatin capsule.

Study summary

The goal of this clinical trial is to learn if the drug pridopidine works to treat amyotrophic lateral sclerosis in adults. It will also help to learn about the safety of pridopidine. The main question it aims to answer is:

Does pridopidine slow disease progression of ALS?

Researchers will compare pridopidine to a placebo (a look-alike substance that contains no drug) to see if pridopidine works to treat ALS.

Participants will:

Take pridopidine or a placebo by mouth every day for 48 weeks. Afterwards, all participants will take pridopidine for another 48 weeks.

Visit the clinic once every 1-3 months for checkups and tests

Eligibility

Primary outcome measure(s)

• 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
  The ALSFRS-R encompasses 12 questions grouped into 4 domains to assess bulbar symptoms, limb and trunk functionality, respiratory symptoms, and the need for percutaneous endoscopic gastrostomy (PEG), non-invasive ventilation, or tracheostomy with invasive ventilation. Each function is scored from 0 (no ability) to 4 (normal), with a minimum total score of 0 and maximum total score of 48. Higher scores indicate better functioning.

Trial sites (55)

+ 15 more sites — see the full list on the official registry below.