A Phase 2/3 Placebo-Controlled, Parallel Group, Randomized Withdrawal Study to Evaluate the Efficacy and Safety of Nipocalimab for Adults with Chronic Inflammatory Demyelinating Polyneuropathy: the ARISE study
Lisa Ford1, Janice Wong1, Eriene Youssef1, Robert Murray2, Pilar Lim1, Eduardo Nobile-Orazio3, Ingemar Merkies4, David Cornblath5, Hong Sun1
1Janssen Research & Development, LLC, Titusville, NJ, USA, 2Janssen Research & Development, High Wycombe, United Kingdom, 3Neuromuscular Diseases and Neuroimmunology Service, IRCCS Humanitas Clinical and Research Institute, Milan University, Rozzano, Italy, 4Department of Neurology, Maastricht University Medical Center, Maastricht, the Netherlands, 5No affiliation
Objective:
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare, chronic autoimmune disease of the peripheral nervous system characterized by progressive weakness and impaired sensation. The ARISE study (NCT05327114) is a Phase 2/3 multicenter, double-blind, placebo-controlled, randomized withdrawal study evaluating the efficacy and safety of nipocalimab in adults with CIDP.
Background:
Nipocalimab is a fully human aglycosylated immunoglobulin (Ig)G1 monoclonal antibody (mAb) designed to selectively bind, saturate, and block the IgG binding site on the endogenous neonatal Fc receptor. Nipocalimab is administered via intravenous infusion.
Design/Methods:
The study consists of the following periods: (1) Screening (≤4 weeks)/Run-in (≤12 weeks), which includes identification of patients with active CIDP; (2) Stage A (Open-label, 12 weeks), in which participants receive nipocalimab loading dose, then once every 2 weeks (q2w); (3) Stage B (Double-blind, placebo-controlled, ≤52 weeks), in which participants are randomized 1:1 to nipocalimab q2w versus placebo; (4) Open-label extension (variable duration), in which participants receive nipocalimab q2w. Key inclusion criteria include adults ≥18 years with CIDP, progressing/relapsing forms, confirmed by independent adjudication committee; CIDP disease activity score ≥3; and adjusted INCAT disability score 2-9. Patients with pure sensory CIDP or CISP, or other diagnoses that could better explain their clinical presentation are excluded. The primary endpoint is time to first occurrence of a relapse event in Stage B. Secondary efficacy endpoints include time to initial response to nipocalimab and percentage of nipocalimab responders in Stage A, and change from baseline in functional measures (e.g. grip strength, MRC sum score, etc.) in Stage B. Other secondary endpoints include safety/tolerability, pharmacokinetics, immunogenicity, and pharmacodynamics of nipocalimab.
Results:
The study is currently enrolling patients, targeting approximately 300 patients, with primary study completion date anticipated in 2026.
Conclusions:
This ongoing phase 2/3 study will assess the efficacy, safety, PK, and PD of nipocalimab in adults with CIDP.