To determine the safety profile of Arimoclomol in patients with Amyotrophic Lateral Sclerosis (ALS).

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease characterized by muscle weakness and respiratory failure. Arimoclomol, a co-inducer of heat-shock protein-70 (HSP70), has shown neuroprotective effects in animal models of ALS by promoting the clearance of protein aggregates, a key pathological feature of both sporadic and familial ALS.

Three randomized clinical trials pooling 319 patients were included in this meta-analysis. Arimoclomol was comparable to placebo regarding the serious adverse events (RR=0.75; 95%CI:[0.41,1.37]; p=0.35; I²=44%), respiratory (RR=1.36; 95%CI:[0.47,3.92]; p=0.57; I²=70%), upper GI  (RR=1.87; 95%CI:[0.24,14.36]; p=0.55; I²=93%), and lower GI adverse events (RR=1.51; 95%CI:[0.89,2.54]; p=0.12; I²=0%). The was also no significant difference in terms of CV (RR=1.64; 95%CI:[0.57,4.72]; p=0.35; I²=19%), MSK adverse events (RR=1.82; 95%CI:[0.81,4.05]; p=0.14; I²=45%) and mood symptoms (RR=0.88; 95%CI:[0.26,2.95]; p=0.83; I²=81%) between the treatment and the control group in ALS patients.

The meta-analysis concluded that Arimoclomol appears to have a comparable safety profile to placebo in patients with ALS, with no significant differences in the incidence of serious adverse events, including respiratory, gastrointestinal, cardiac, musculoskeletal, and mood-related symptoms. The findings suggest that Arimoclomol is generally well-tolerated in ALS patients, though further studies may be needed to confirm these results.