Open-label trial of riluzole 50 mg b.i.d. in treatment of amyotrophic lateral sclerosis (ALS)

The glutamate release inhibitor riluzole enjoys the reputation of being the only effective drug for prolonging life in amyotrophic lateral sclerosis ( ALS) patients. The aim of this multinational, uncontrolled, open treatment study was to provide a large number of ALS patients with riluzole until it received marketing approval and to obtain additional data on the safety of riluzole. Methods and results: Between 1995 and 1997 a total of 7916 ALS pa tients in 39 countries were given 100 mg riluzole per day for a mean of 202 .1 days (7.2 months). 919 of the patients were treated in 25 German centres . Of the 919 patients in Germany 162 (17.6%) died from the disease during t he course of the study. The most frequent adverse events were reduced lung function (7.3%) nausea (7.1%), asthenia (5.8%), pneumonia (2.5%) and abdomi nal pain (2.5%). Serious adverse events attributed to the study medication occurred in 16 patients (1.7%): most frequently these were changes in liver enzymes (0.9%) occurring during the first 3 treatment months. However, all liver enzyme changes were reversible and none were fatal. Conclusion: The results of the study allow the conclusion that riluzole is well tolerated. The majority of the observed adverse events were due to symptoms of the und erlying disease and not to riluzole. The incidence of adverse events in thi s multinational patient population is very much lower than the incidence ra tes reported in previous studies. Overall the safety profile found in the G erman centres was very similar to the profile seen in the total patient pop ulation.