A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis
To determine if long-term topiramate therapy is safe and slows disease progression in patients with ALS.
Disease:Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALS
Study Type:Interventional Trial
Study Category:Drug Trial
Study Status:Not enrolling
Study Chair(s)/Principal Investigator(s):
Merit Cudkowicz, Massachusetts General Hospital
Clinicaltrials.gov ID (11 digit #):
Coordinating Center Contact Information
Full Study Summary:
Estimated Study Start Date:06/01/1999
Estimated Study Completion Date:08/31/2000
Posting Last Modified Date:05/09/2019
Date Study Added to alsconsortium.org:05/09/2019
Time since Symptom Onset:
Time since Diagnosis:
Can participants use Riluzole?
- Site Contact Information
Patients treated with topiramate showed a faster decrease in arm strength (33.3%) during 12 months (0.0997 vs 0.0748 unit decline/month, p = 0.012). Topiramate did not significantly alter the decline in FVC and ALSFRS or affect survival. Topiramate was associated with an increased frequency of anorexia, depression, diarrhea, ecchymosis, nausea, kidney calculus, paresthesia, taste perversion, thinking abnormalities, weight loss, and abnormal blood clotting (pulmonary embolism and deep venous thrombosis).
At the dose studied, topiramate did not have a beneficial effect for patients with ALS. High-dose topiramate treatment was associated with a faster rate of decline in muscle strength as measured by MVIC and with an increased risk for several adverse events in patients with ALS. Given the lack of efficacy and large number of adverse effects, further studies of topiramate at a dose of 800 mg or maximum tolerated dose up to 800 mg/day are not warranted.