HEALEY ALS (CNM-Au8)
Trial question
What is the effect of CNM-Au8 in patients with ALS?
Study design
Multi-center
Double blinded
RCT
Population
Characteristics of study participants
35.0% female
65.0% male
N = 284
284 patients (98 female, 186 male).
Inclusion criteria: patients with ALS.
Key exclusion criteria: allergy to gold, gold salts, or colloidal gold preparations; clinically significant unstable medical condition; unstable psychiatric disease, cognitive impairment, dementia, or substance abuse; active cancer or history of cancer; pregnancy or lactation.
Interventions
N=120 CNM-Au8 (at a dose of 60 mg or 30 mg daily for 24 weeks).
N=164 placebo (matching placebo for 24 weeks).
Primary outcome
Reduction in disease severity through week 24, measured by Revised Amyotrophic Lateral Sclerosis Functional Rating Scale score and survival
1 points
1.03 points
1.0 points
0.8 points
0.5 points
0.3 points
0.0 points
CNM-Au8
Placebo
No significant
difference ↔
No significant difference in reduction in disease severity through week 24, measured by Revised ALS Functional Rating Scale score and survival (1 points vs. 1.03 points; DRR 0.97, 95% CI 0 to 1.94).
Secondary outcomes
No significant difference in mean reduction in Slow Vital Capacity over 24 weeks (9.32 PPN per month vs. 8.53 PPN per month; MD 0.78, 95% CI -2.68 to 4.25).
No significant difference in permanent assisted ventilation-free survival (4.2% vs. 5.6%; HR 0.46, 95% CI 0.12 to 1.49).
No significant difference in Combined Assessment of Function and Survival (140.5 vs. 143.9; MD -3.4, 95% CI -13.28 to 6.48).
Safety outcomes
No significant differences in ≥ 1 treatment-emergent adverse event, serious treatment-emergent adverse events.
Conclusion
In patients with ALS, CNM-Au8 was not superior to placebo with respect to reduction in disease severity through week 24, measured by Revised ALS Functional Rating Scale score and survival.
Reference
Writing Committee for the HEALEY ALS Platform Trial, James D Berry, Nicholas J Maragakis et al. CNM-Au8 in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial. JAMA. 2025 Feb 17:e2427643. Online ahead of print.
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