Shin J Oh1, Natalya Shcherbakova2, Anna Kostera-Pruszczyk3, Mohammad Alsharabati1, Mazen Dimachkie4, Jose Munoz Blanco5, Thomas Brannagan6, Dragana Lavrnić7, Perry B Shieh8, Christophe Vial9, Andreas Meisel10, Samuel Komoly11, Benedikt Schoser12, Kumaraswamy Sivakumar13, Yuen So14. 1. Department of Neurology, University of Alabama at Birmingham, Birmingham, Alabama, USA. 2. Research Center of Neurology, Moscow, Russia. 3. Department of Neurology, Medical University of Warsaw, Poland. 4. University of Kansas Medical Center, Kansas City, Kansas, USA. 5. Gregorio Maranon Hospital, Madrid, Spain. 6. Columbia University Medical Center, New York, New York, USA. 7. Clinical Center of Serbia, Clinic of Neurology, Belgrade, Serbia. 8. Department of Neurology, University of California, Los Angeles, California, USA. 9. Hospital of Lyon, ENMG Service and Neuromuscular Pathology Hospital, Lyon, France. 10. Charite Universitatsmedizin Berlin-NeuroCure Clinical Research Center, Berlin, Germany. 11. University of Pécs, Department of Neurology, Pécs, Hungary. 12. Ludwig-Maximilians-University Munich Friedrich-Baur-Institute, Munich, Germany. 13. Neuromuscular Research Center, Phoenix, Arizona, USA. ksiva@nrcaz.com. 14. Stanford University, Stanford, California, USA.
Abstract
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.
RCT Entities:
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.
Authors: Donald B Sanders; Vern C Juel; Yadollah Harati; A Gordon Smith; Amanda C Peltier; Tessa Marburger; Jau-Shin Lou; Robert M Pascuzzi; David P Richman; Tai Xie; Valentin Demmel; Laura R Jacobus; Kathy L Aleš; David P Jacobus Journal: Muscle Nerve Date: 2018-02-02 Impact factor: 3.217
Authors: Wei-Hua Chiu; Lora Kovacheva; Ruth E Musgrove; Hadar Arien-Zakay; James B Koprich; Jonathan M Brotchie; Rami Yaka; Danny Ben-Zvi; Menachem Hanani; Jochen Roeper; Joshua A Goldberg Journal: Sci Adv Date: 2021-03-10 Impact factor: 14.136
Authors: Nilay Thakkar; Jeffrey T Guptill; Kathy Aleš; David Jacobus; Laura Jacobus; Charles Peloquin; Michael Cohen-Wolkowiez; Daniel Gonzalez Journal: CPT Pharmacometrics Syst Pharmacol Date: 2017-07-24