NCT07325591

Efficacy and Safety of Tazbentetol in ALS Participants

A Phase 2B/3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Tazbentetol in Participants With Amyotrophic Lateral Sclerosis (ALS)

• Status: Not Yet Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 430 (estimated)
• Sponsor: Spinogenix · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

The objectives of this study are to examine the effects of tazbentetol on clinical measures of ALS, patient reported outcomes (PROs), long-term safety and tolerability.

Detailed description

This is a Phase 2B/3 adaptive design randomized, double-blind, placebo-controlled (DBPC) study to evaluate the efficacy, safety and tolerability of tazbentetol administered orally in participants with ALS. The study consists of 2 parts, as follows Phase 2 double-blind, placebo controlled (DBPC): Randomized, double-blind, placebo-controlled study. Participants will be randomized to receive tazbentetol or placebo for 36 weeks. Phase 3 double blind, placebo controlled (DBPC): Randomized, double-blind, placebo-controlled study. Participants will be randomized to receive the dose determined from Phase 2 or placebo for 36 weeks. Open-label extension: Eligible participants who complete 36 weeks in the DBPC of either Phase 2 or Phase 3 will be offered to enroll into the OLE, starting at the corresponding DBPC Week 36 visit, and receive tazbentetol for 36 weeks. The dose for this extension will be based on data from DBPC phases.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	Tazbentetol	Participants in both Phase 2B and Phase 3 will be randomized to received study drug tazbentetol or placebo tablets. Participants in the open-label extension phase will receive the dose determined from Phase 2.
DRUG	placebo	participants in double blind placebo controlled phase will be randomized to received placebo tablets

Timeline

• Start date: 2026-03-01
• Primary completion: 2027-12-31
• Completion: 2028-03-01
• First posted: 2026-01-08
• Last updated: 2026-03-02

Regulatory

• FDA-regulated drug study