Independent Adjudication Committee Helps Facilitate Accurate Enrollment of Phase 2/3 ARISE Trial of Nipocalimab

A newly presented analysis from the ongoing ARISE phase 2/3 trial (NCT05327114) of nipocalimab (Imaavy; Johnson & Johnson) showed that use of an independent adjudication committee to confirm diagnoses in patients with chronic inflammatory demyelinating polyneuropathy (CIDP) improved the accuracy of patient selection prior to enrollment, underscoring the challenges of achieving diagnostic precision in this neuromuscular disorder.¹

In the first 110 patients screened (median age, 57 years; men, n = 74), an independent CIDP adjudication committee of 4 members confirmed that 72.7% of patients (n = 80) met diagnostic eligibility based on the European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) 2021 criteria. Lead author Jonathan Katz, MD, director of the Forbes Norris ALS Clinic at California Pacific Medical Center, presented these findings at the 2025 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) meeting, held October 29 to November 1, in San Francisco, California.

ARISE is a phase 2/3 multicenter, randomized, double-blind, placebo-controlled withdrawal study designed to evaluate nipocalimab, a neonatal Fc receptor blocker therapy, as a potential treatment for adults with CIDP. Since the diagnosis of CIDP can be challenging, researchers used a CIDP adjudication committee to potentially improve case selection for enrollment into the trial. Thus, this analysis focused on diagnostic confirmation and patient characteristics prior to assessing efficacy or safety outcomes. Authors noted that the eligible patients in the study would be reflective of the real-world demographics and distribution of CIDP types.

Under the study protocol, site principal investigators first submitted deidentified diagnostic data for review by a CIDP adjudication committee member. Agreement between the first CIDP adjudication committee reviewer and the site principal investigators confirmed patient eligibility. In cases of disagreement, the data were assigned to a second CIDP adjudication committee reviewer. Agreement between the second reviewer and the site principal investigators allowed the patient to proceed with enrollment, whereas persistent disagreement results led to exclusion.

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All told, agreement between the first CIDP adjudication committee reviewer and the site principal investigator adjudicated 69 patients (62.7%) as eligible (typical CIDP, n = 52; variant CIDP, n = 17). The remaining 41 patients (37.3%) were assigned to a second CIDP adjudication committee reviewer. Agreement between the second reviewer and the site PI adjudicated 11 additional patients as eligible (typical CIDP, n= 10; variant CIDP, n= 1), whereas the remaining 30 patients were adjudicated as ineligible. Overall, findings showed that the use of a second CIDP adjudication committee reviewer increased the number of eligible patients by 10%.

In April 2025, the FDA approved nipocalimab for adults and pediatric patients aged 12 years and older with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor or anti-muscle-specific kinase antibody positive. Findings from the ongoing phase 3 VIVACITY-MG3 study (NCT04951622) served as the basis for the approval, showing a 4.70-point improvement in Myasthenia Gravis-Activities of Daily Living score for those treated with nipocalimab plus standard-of-care (SOC). Notably, those on nipocalimab plus SOC demonstrated a significant improvement in Quantitative Myasthenia Gravis score over placebo through 22 and 24 weeks (P <.001).

In an interview with NeurologyLive^®, Nicholas J. Silvestri, MD, FAAN, a professor of neurology at the University of Buffalo Jacobs School of Medicine and Biomedical Sciences, discussed the significance of the approved gMG therapy. In the discussion, he highlighted its clinical benefits demonstrated in the phase 3 VIVACITY-MG3 trial and noted its unique advantage in treating adolescents, filling an unmet need in that population. During the conversation, Silvestri also addressed important considerations such as infection risk, long-term safety, and vaccine administration during treatment, underlining the importance of individualized care and expanding therapeutic choices.

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REFERENCES
1. Katz J, Gorson K, Harbo T, et al. Nipocalimab in Chronic Inflammatory Demyelinating Polyneuropathy: Diagnostic Adjudication of the First 110 Patients of the Phase 2/3 ARISE Study. Presented at: 2025 AANEM; October 29 to November 1; San Francisco, California. Abstract 177.
2. Johnson & Johnson receives FDA approval for IMAAVY™ (nipocalimab-aahu), a new FcRn blocker offering long-lasting disease control in the broadest population of people living with generalized myasthenia gravis (gMG). News release. Johnson & Johnson. April 30, 2025. Accessed October 28, 2025. https://www.prnewswire.com/news-releases/johnson--johnson-receives-fda-approval-for-imaavy-nipocalimab-aahu-a-new-fcrn-blocker-offering-long-lasting-disease-control-in-the-broadest-population-of-people-living-with-generalized-myasthenia-gravis-gmg-302442650.html