Amifampridine Phosphate (Firdapse) Is Effective in a Confirmatory Phase 3 Clinical Trial in LEMS

Journal of Clinical Neuromuscular Disease
Perry ShiehShin J Oh

Abstract

To assess tolerability and efficacy of amifampridine phosphate versus placebo for symptomatic treatment of Lambert-Eaton Myasthenic Syndrome (LEMS). This phase 3 randomized, double-blind, placebo-controlled withdrawal trial in 26 adults with LEMS compared efficacy of amifampridine phosphate versus placebo over a 4-day period. The primary endpoints were quantitative myasthenia gravis score (QMG) and subject global impression, and the secondary endpoint was Clinical Global Impression-Improvement. The exploratory endpoints were 3TUG (timed up and go) test and QMG limb domain score. All participants had been receiving amifampridine phosphate (30-80 mg/d divided into 3 or 4 doses daily) in an expanded access protocol and had been titrated to the optimal dose and frequency for at least 1 week before randomization into the current study. After completion of assessments after 4 days of double-blind treatment, patients had the option to return to open-label amifampridine phosphate. The efficacy endpoints were mean changes from baseline in the various evaluation parameters. Amifampridine phosphate (n = 13) demonstrated significant benefit in QMG and subject global impression compared with placebo (n = 13) at 4 days. Other measures of eff...Continue Reading

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Citations

Sep 20, 2019·Expert Review of Clinical Immunology·Shin J Oh
Oct 23, 2019·Expert Review of Clinical Pharmacology·Renato Mantegazza
Oct 16, 2020·Neurodegenerative Disease Management·Laura De GiglioElena Maria Pennisi
Aug 28, 2021·Brain Sciences·Maria Pia GiannoccaroRocco Liguori

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