Journal article
A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis
Neurology, v 61(4), pp 456-464
26 Aug 2003
PMID: 12939417
Featured in Collection : UN Sustainable Development Goals @ Drexel
Abstract
OBJECTIVETo determine if long-term topiramate therapy is safe and slows disease progression in patients with ALS.METHODSA double-blind, placebo-controlled, multicenter randomized clinical trial was conducted. Participants with ALS (n = 296) were randomized (2:1) to receive topiramate (maximum tolerated dose up to 800 mg/day) or placebo for 12 months. The primary outcome measure was the rate of change in upper extremity motor function as measured by the maximum voluntary isometric contraction (MVIC) strength of eight arm muscle groups. Secondary endpoints included safety and the rate of decline of forced vital capacity (FVC), grip strength, ALS functional rating scale (ALSFRS), and survival.RESULTSPatients 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).CONCLUSIONSAt 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.
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Details
- Title
- A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis
- Creators
- M E Cudkowicz - Harvard University ,J M Shefner - SUNY Upstate Medical UniversityD A Schoenfeld - Harvard University ,R H Brown - Harvard University ,H Johnson - Harvard UniversityM Qureshi - Harvard University ,M Jacobs - Harvard UniversityJ D Rothstein - Johns Hopkins UniversityS H Appel - Baylor College of MedicineR M Pascuzzi - University of IndianapolisT D Heiman-Patterson - Drexel UniversityP D Donofrio - Wake Forest UniversityW S David - Hennepin County Medical CenterJ A Russell - Lahey Medical CenterR Tandan - University of VermontE P Pioro - Cleveland ClinicK J Felice - University of ConnecticutJ Rosenfeld - Carolinas Medical CenterR N Mandler - George Washington UniversityG M Sachs - Rhode Island HospitalW G Bradley - University of Miami*E M Raynor - Harvard University ,G D Baquis - Tufts UniversityJ M Belsh - Rutgers, The State University of New JerseyS Novella - Yale UniversityJ Goldstein - Yale UniversityJ Hulihan - Ortho-McNeil Pharmaceutical, Inc.NE ALS Consortium
- Publication Details
- Neurology, v 61(4), pp 456-464
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- Biology; Office of Research (and Innovation)
- Web of Science ID
- WOS:000184953900007
- Scopus ID
- 2-s2.0-10744222650
- Other Identifier
- 991019169801204721
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- Collaboration types
- Industry collaboration
- Domestic collaboration
- Web of Science research areas
- Clinical Neurology