Michael Weiss, MD, Department of Neurology, University of Washington Medical Center, discusses results from the RAISE clinical trial testing the safety and efficacy of zilucoplan in patients with myasthenia gravis (MG).
MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles that control the eye and eyelid, facial expressions, chewing, talking, and swallowing. Weakness tends to increase during periods of activity and improve after periods of rest. The condition results from a defect in the transmission of nerve impulses to muscles, which is due to the presence of antibodies against acetylcholine. The exact reason this occurs is not known.
RAISE Clinical Trial
The RAISE clinical trial (NCT04115293) is a phase 3, multicenter, double-blind, placebo-controlled study evaluating zilucoplan in adult patients with MG. The primary objectives of the study were to evaluate the effect of zilucoplan on the Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores in patients with generalized MG. Zilucoplan is a macrocyclic peptide complement component 5 inhibitor and was administered as a daily subcutaneous injection at a dose of 0.3 mg/kg for 12 weeks.
Results
At week 12, least-squares mean (LSM) change from baseline in MG-ADL total score was -4.39 for the zilucoplan treated group comopare to -2.30 for placebo. LSM change from baeline for QMG total score was -6.19 for zilucoplan and -3.25 for placebo. Mean change from baseline in AG-ADL subdomain scores in the zilucoplan versus placebo groups were: ocular, –1.5 vs –0.8 ; bulbar, –1.9 vs –1.1; respiratory, –0.4 vs –0.3; limb/axial, −1.2 vs –0.8. Mean CFB in QMG subdomain scores were: ocular, −2.0 vs −1.3; bulbar, −1.6 vs −1.1; respiratory, −0.6 vs −0.3; limb/axial, −2.9 vs −1.2.
These results further support zilucoplan’s ability to provide rapid and clinically meaningful improvements in MG-ADL and QMG scores compared to placebo.
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To learn more about MG and other rare neurological conditions, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/