GANGLIOSIDE GM(1) IN ACUTE ISCHEMIC STROKE - THE SASS TRIAL

Background and Purpose We sought to assess the safety and efficacy of ganglioside GM(1) in acute (less than or equal to 48 hours), anterior circulation ischemic stroke. Methods We screened more than 5000 patien ts at 13 centers in a randomized, double-blind, placebo-controlled, pa rallel-treatment, clinical trial and enrolled 287 patients. They recei ved 100 mg GM(1) or placebo intramuscularly daily for 28 days and were evaluated regularly for 84 days. Number of deaths, the Toronto Stroke Scale, and the Barthel Index were primary outcomes; improvements on t he Fugl-Meyer Scale and on a neuropsychological battery were secondary outcomes. Results The groups were balanced for severity, side of stro ke, age, sex, race, years of schooling, prior illness, and depression scores. Analyzable data were available on 275 patients; 217 patients c ompleted the trial. Protocol-specified primary and secondary outcome m easures showed no significant difference between treatment arms. Howev er, improve ment from baseline in the motor component of the Toronto S troke Scale favored the GM(1)-treated group at day 28 when GM(1) treat ment stopped (P=.020); at day 84, the difference still favored the GM( 1)-treated group (P=.057). All 10 components of the Barthel Index, the Fugl-Meyer Scale, and four of the five tests in the neuropsychologica l battery also favored the GM(1) group. Adverse experiences were simil ar in the two groups.Conclusions GM(1) is safe. However, since only ce rtain post hoc tests showed statistically significant differences or t rends favoring