A phase 2b, randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of Usnoflast administered to adult subjects with Amyotrophic Lateral Sclerosis (ALS)

Condition studied

Amyotrophic Lateral Sclerosis (ALS)

Investigational medicinal product(s)

Eligibility

Primary endpoint

Change in disease progression from baseline through 36 weeks, as measured by ALSFRS-R total score and survival

Endpoint detail

1. Change in SVC from baseline to Week 36, 2. Change in serum levels of NfL protein from baseline to Week 36, 3. Change in ALSFRS-R total score and scores of various functional items/domains of the ALSFRS-R total score from baseline to Week 36, 4. Change in ALSAQ-40 score from baseline to Week 36, 5. Number of TEAEs and SAEs up to Week 36, 6. Assessment of PK in plasma (baseline, Week 16, and Week 36) and CSF (baseline, Week 16, and Week 36), 7. Change in CSF levels of NfL protein from baseline to Week 36, 8. Time from baseline to the occurrence of death or PAV (>22 hours daily for >7 days) (from baseline through Week 36)