Branched-chain amino acids and amyotrophic lateral sclerosis: a treatment failure? The Italian ALS Study Group.
We initiated a double-blind, placebo-controlled trial to test the efficacy and safety of branched-chain amino acids (BCAA) (L-leucine 12 g, L-isoleucine 6 g, and L-valine 6 g daily) in amyotrophic lateral sclerosis (ALS) patients. There was an excess mortality in subjects randomized to active treatment (24 BCAA, 13 placebo) when a total of 126 ALS patients had been recruited. This finding, associated with the lack of efficacy of BCAA (measured by comparing the disability scales in the two treatment groups), led the Data Monitoring Committee to require cessation of the trial.
D000597:Amino Acids, Branched-Chain / Q000009:adverse effects / Q000627:therapeutic use*; D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy* / Q000401:mortality / Q000503:physiopathology; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008297:Male; D015201:Meta-Analysis as Topic; D008875:Middle Aged; D015999:Multivariate Analysis; D010344:Patient Advocacy; D017751:Safety Management; D012720:Severity of Illness Index; D016019:Survival Analysis; D017211:Treatment Failure; D014797:Vital Capacity
D000597:Amino Acids, Branched-Chain