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 patients at 13 centers in a randomized, double-blind, placebo-controlled, parallel-treatment, clinical trial and enrolled 287 patients. They received 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 the Fugl-Meyer Scale and on a neuropsychological battery were secondary outcomes. Results The groups were balanced for severity, side of stroke, age, sex, race, years of schooling, prior illness, and depression scores. Analyzable data were available on 275 patients; 217 patients completed the trial. Protocol-specified primary and secondary outcome measures showed no significant difference between treatment arms. However, improve ment from baseline in the motor component of the Toronto Stroke Scale favored the GM(1)-treated group at day 28 when GM(1) treatment 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 neuropsychological battery also favored the GM(1) group. Adverse experiences were similar in the two groups. Conclusions GM(1) is safe. However, since only certain post hoc tests showed statistically significant differences or trends favoring