Efficacy and Safety Evaluation of Nipocalimab in Adults with Active Idiopathic Inflammatory Myopathies: A Phase 2, Randomized, Double-blind, Placebo-controlled Study

The primary objective of this study is to evaluate the **efficacy** of **nipocalimab** compared to placebo in participants with active **Idiopathic Inflammatory Myopathies (IIM)**. This is clinically relevant as it aims to determine the therapeutic potential of nipocalimab in managing symptoms and progression of IIM, a group of disorders characterized by muscle inflammation and weakness, which can significantly impact patients' quality of life. The study is designed as a Phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel-group trial, ensuring robust and reliable results.

The clinical trial involves a total of **129 participants** diagnosed with **Active Idiopathic Inflammatory Myopathies (IIM)**. The study population includes both male and female subjects, with an age range corresponding to categories 3 and 4, indicating a broad age spectrum. Participants were selected based on specific diagnostic criteria, including the presence of myositis-specific antibodies, as per the 2017 EULAR/ACR classification criteria for adult IIM. The trial includes a vulnerable population, ensuring careful consideration of ethical standards. Participants' general health status is characterized by the presence of active IIM, and they may be on stable doses of low potency topical glucocorticoids or topical tacrolimus for skin lesions. The selection process ensures that participants meet the necessary antibody positivity criteria, which is crucial for the study's focus on evaluating the efficacy of nipocalimab versus placebo.

The clinical trial is a **Phase 2**, multicenter, randomized, double-blind, placebo-controlled, parallel-group study designed to evaluate the efficacy and safety of **nipocalimab** in participants with active **idiopathic inflammatory myopathies** (IIM). The trial aims to compare the effects of nipocalimab against a placebo in achieving at least a minimal improvement in the International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS TIS) at Week 52, while participants are on a reduced dose of oral prednisone or its equivalent. The study is expected to last until March 2027, with recruitment having commenced in January 2023.

Participants will be randomly assigned to receive either the investigational product, JNJ-80202135, or a placebo, both administered as a **solution for infusion** via **intravenous use**. The maximum treatment period is 52 weeks. The trial includes several key visits: an initial screening visit to confirm eligibility based on the 2017 EULAR/ACR classification criteria for adult IIM, followed by regular follow-up visits to monitor safety and efficacy, and a final end-of-study visit to assess the primary and secondary endpoints.

The expected duration of participant involvement is approximately 52 weeks, with conditions for early termination including failure to meet inclusion criteria, adverse events, or withdrawal of consent. Participants must have stable doses of any allowed topical treatments for skin lesions for at least four weeks prior to the first administration of the study intervention and maintain these doses throughout the study. The trial's primary endpoint is the proportion of participants achieving a ≥20-point improvement in IMACS TIS at Week 52, while secondary endpoints will be evaluated as per the study protocol.

The clinical trial involves the administration of **nipocalimab**, an investigational medication, under the product name JNJ-80202135. Nipocalimab is provided in two pharmaceutical forms: a **solution for infusion** and a **solution for injection**. The active substance, nipocalimab, is a protein classified as a human monoclonal antibody targeting the neonatal Fc receptor. The medication is administered via the **intravenous route**. The dosing schedule and frequency are determined by the study protocol, with a maximum treatment period of 52 weeks. The trial aims to evaluate the efficacy and safety of nipocalimab in participants with active idiopathic inflammatory myopathies.

In addition to the experimental treatment, a **placebo** is used as a comparator in this double-blind, placebo-controlled study. The placebo consists of a saline solution, specifically a 0.9% sodium chloride solution for injection. This non-experimental treatment serves as a control to assess the effects of the investigational drug. The administration of the placebo follows the same route and schedule as the experimental medication to maintain the study's blinding and integrity.

The efficacy of the investigational product, **nipocalimab**, will be assessed in a Phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel-group study involving participants with active idiopathic inflammatory myopathies (IIM). The primary endpoint for evaluating efficacy is the proportion of participants who achieve at least minimal improvement, defined as a ≥ 20-point improvement in the International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS TIS) at Week 52. Additionally, participants must be on ≤ 5 mg/day of oral prednisone (or equivalent) from Week 44 through Week 52 to meet this endpoint.