Empasiprubart Versus Placebo in Chronic Inflammatory Demyelinating Polyradiculoneuropathy: EMNERGIZE Phase Three Study Design
Simon Rinaldi1, Thomas Brannagan2, Pietro Emiliano Doneddu3, Karissa Gable4, Mark Stettner5, Luis Querol6, Reena Patel7, Hafedh Haddad7, Olivier Van de Steen7, Kevin Budding7, Inge Van de Walle7, Susan Ellor7, Martin Markov7, Jeffrey Allen8
1University of Oxford, 2Department of Neurology, Columbia University, 3Neuromuscular and Neuroimmunology Unit, IRCCS Humanitas Research Hospital, 4Neuromuscular Division, Department of Neurology, Duke University, 5Department of Neurology and Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Medicine Essen, 6Hospital de la Santa Creu i Sant Pau, 7argenx, 8Department of Neurology, University of Minnesota
Objective:
To describe the study design of the EMNERGIZE trial, which compares empasiprubart versus placebo in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).
Background:
CIDP is a rare, autoimmune neuropathy characterized by nerve damage, resulting in disability due to limb weakness, sensory disturbances, and fatigue. Nerve biopsies in CIDP feature complement deposition. Additionally, complement activation promotes macrophage-mediated demyelination. Empasiprubart binds complement C2, blocking activation of classical and lectin complement pathways.
Design/Methods:
This Phase 3, randomized, double-blinded, placebo-controlled study will randomize ~160 adults in a 2:1 ratio to receive either empasiprubart IV or placebo on Days 1 and 8 then once every 4 weeks in a 24-week double-blind treatment period (Part A), followed by a 4-week double-blind rollover and a 23-month open-label period (Part B), and a 15-month safety follow-up after the last dose.
Results:
The primary endpoint is a reduction of ≥1-point versus baseline in adjusted Inflammatory Neuropathy Cause and Treatment (aINCAT) score at Week 24. Key secondary endpoints include changes from baseline at Week 24 in Inflammatory Rasch-Built Overall Disability Scale score, Medical Research Council Sum score, dominant hand grip strength, and Timed Up and Go, and time to reduction of ≥1 point versus baseline in aINCAT score.
Conclusions:
This Phase 3 study will compare the efficacy and safety of empasiprubart versus placebo in patients with CIDP, focusing on functional ability, muscle strength/function, clinical status, gait impairment, and patient-reported outcomes.
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