HEALEY ALS (verdiperstat)
Trial question
What is the role of verdiperstat, a myeloperoxidase inhibitor, in patients with ALS?
Study design
Multi-center
Double blinded
RCT
Population
Characteristics of study participants
35.0% female
65.0% male
N = 167
167 patients (59 female, 108 male).
Inclusion criteria: adult patients with ALS.
Key exclusion criteria: clinically significant unstable medical condition; unstable psychiatric disease, cognitive impairment, dementia, or substance abuse; active cancer or history of cancer; use of investigational treatments for ALS; exposure at any time to any gene therapies; pregnancy or lactation.
Interventions
N=126 verdiperstat (600 mg PO BID for 24 weeks).
N=41 placebo (matching placebo for 24 weeks).
Primary outcome
Improvement in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised total score at 24 weeks
-6.58 points
-6.69 points
0.0 points
-1.7 points
-3.3 points
-5.0 points
-6.7 points
Verdiperstat
Placebo
No significant
difference ↔
No significant difference in improvement in ALS Functional Rating Scale-Revised total score at 24 weeks (-6.58 points vs. -6.69 points; AD 0.11 points, 95% CI -2.08 to 2.3).
Secondary outcomes
No significant difference in improvement in hand-held dynamometer-upper limb at week 24 (-31.67% vs. -39.91%; AD 8.24%, 95% CI -3.88 to 20.36).
No significant difference in improvement in slow vital capacity at week 24 (-8.34% vs. -10.64%; AD 2.3%, 95% CI -3.01 to 7.61).
No significant difference in improvement in hand-held dynamometer-lower limb at week 24 (-19.77% vs. -21.57%; AD 1.8%, 95% CI -12.65 to 16.25).
Safety outcomes
No significant difference in treatment-emergent adverse events.
Conclusion
In adult patients with ALS, verdiperstat was not superior to placebo with respect to improvement in ALS Functional Rating Scale-Revised total score at 24 weeks.
Reference
Writing Committee for the HEALEY ALS Platform Trial, Jinsy Andrews, Sabrina Paganoni et al. https: / /pubmed.ncbi.nlm.nih.gov / 40067754 /. JAMA Neurol. 2025 Apr;82(4):333-343.
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