A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial of Dazucorilant, A Selective Glucocorticoid Receptor Modulator, in Amyotrophic Lateral Sclerosis

Summary

DAZALS did not achieve its primary endpoint of slowing functional decline as measured by the ALSFRS-R scale
Secondary and exploratory endpoints showed improvement in overall survival for participants who received dazucorilant 300 mg, especially in the subgroup treated for >24 weeks
Dazucorilant-treated participants discontinued at higher rates than placebo-treated participants due to abdominal pain, which occurred in the first 3–5 weeks
- Abdominal pain resolved following treatment interruption and with time in participants who continued treatment
Abdominal pain was less frequent in participants titrated from dazucorilant 150 mg to 300 mg versus those escalated directly from placebo to 300 mg, suggesting improved tolerability with dose titration
Dazucorilant-treated participants showed similar rates of serious and severe adverse events as the placebo group
These findings support further evaluation of dazucorilant in a phase 3 study in people with ALS