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Amifampridine phosphate (Firdapse ® ) is effective and safe in a phase 3 clinical trial in LEMS
Author(s) -
Oh Shin J,
Shcherbakova Natalya,
KosteraPruszczyk Anna,
Alsharabati Mohammad,
Dimachkie Mazen,
Blanco Jose Munoz,
Brannagan Thomas,
Lavrnić Dragana,
Shieh Perry B,
Vial Christophe,
Meisel Andreas,
Komoly Samuel,
Schoser Benedikt,
Sivakumar Kumaraswamy,
So Yuen
Publication year - 2016
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.25070
Subject(s) - placebo , lambert eaton myasthenic syndrome , clinical endpoint , myasthenia gravis , nausea , adverse effect , randomization , medicine , clinical trial , tolerability , phases of clinical research , phosphate , surrogate endpoint , alternative medicine , pathology , chemistry , organic chemistry
Objective We evaluated the efficacy and safety of amifampridine phosphate (Firdapse ® ) for symptomatic treatment in Lambert‐Eaton myasthenic syndrome (LEMS). Methods Phase 3, randomized, double‐blind, study. Patients were treated initially with amifampridine phosphate for 7–91 days, followed by randomization to continue amifampridine phosphate for 14 days or placebo (7‐day taper, 7‐day placebo). The primary efficacy endpoints were changes from baseline at day 14 in Quantitative Myasthenia Gravis and Subject Global Impression scores. Results The coprimary efficacy end points and 1 of the secondary efficacy end points were met, showing a significant benefit of aminfampridine phosphate over placebo at Day 14. All 5 primary, secondary, and tertiary endpoints achieved statistical significance at Day 8. Amifampridine phosphate was well tolerated; the most common adverse events were oral and digital paresthesias, nausea, and headache. Conclusions This study provides Class I evidence of efficacy of amifampridine phosphate as a symptomatic treatment for LEMS. Muscle Nerve 53 : 717–725, 2016