Topiramate (Topamax) - North Eastern ALS Consortium

Study Results and Conclusions

A 22-site trial of topiramate titled, "Clinical Trial of Topiramate in ALS" is now completed. The purpose of the study was to evaluate the safety and effectiveness of topiramate in the treatment of ALS.

In this study, 800 milligrams per day of topiramate did not demonstrate a benefit for people with ALS. There were no significant differences in the following outcome measures between people in the treatment group and people in the placebo group.

• Arm strength
• Grip strength
• Forced vital capacity - measurement of respiratory muscle strength
• ALS Functional Rating Scale of ability to perform various activities of daily living

Results of this study that included eight ALSA certified centers and 298 people with ALS were presented at the International Symposium of ALS/MND in November, 2002.

There were a number of patients who dropped out of the study due to side effects of the medication, disease progression, study travel requirements and other personal reasons. Side effects included anorexia, nausea, diarrhea, depression, skin problems, kidney problems and blood clots. The trial was stopped due to serious side effects in some of the people in the study. There were 162 patients who completed the study.

Background

The objective of this 12-month study was to determine whether topiramate would slow the progressive deterioration of motor function in people with ALS. The study was a double-blind, randomized, placebo-controlled trial of the safety and efficacy of topiramate.