A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Amyotrophic Lateral Sclerosis
Prilenia
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
Description
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, part…
Eligibility
- Age range
- 18–80 years
- Sex
- All
- Healthy volunteers
- No
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 his…
Interventions
- DrugPridopidine
Pridopidine hard gelatin capsule.
- DrugPlacebo
Placebo hard gelatin capsule.
Locations (5)
- University of KansasFairway, Kansas
- Sean M. Healey & AMG Center for ALSBoston, Massachusetts
- Somnos Clinical ResearchLincoln, Nebraska
- Texas NeurologyDallas, Texas
- Genge PartnersMontreal, Quebec