A new phase 2/3 clinical trial testing nipocalimab in children aged 2 to 17 years with generalized myasthenia gravis has launched. 

Although the trial has not started recruiting participants yet, it aims to enroll an estimated 12 patients among 18 study locations in the United States, Canada, Japan, and the Netherlands.

The aim of the open-label, uncontrolled study is to evaluate the safety, pharmacokinetics, pharmacodynamics, and activity of nipocalimab. Participants will receive nipocalimab as an intravenous infusion once every 2 weeks for 24 weeks, after which they will have the option to enroll in a long-term extension study.

The primary outcome measures include change from baseline in total serum immunoglobulin G (IgG) levels, the number of participants with adverse events, abnormal clinical laboratory test results and vital signs, and the serum concentration, clearance, volume of distribution, and half-life of nipocalimab.

Secondary outcome measures include changes from baseline in the Myasthenia Gravis-Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG), European Quality of Life 5-Dimension Youth (EQ-5D-Y) Tool, and Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue scores, as well as the number of participants with antidrug antibodies, neutralizing antibodies to nipocalimab, and vaccine antibody titers to diphtheria or tetanus.

Read more about the etiology of myasthenia gravis

More information about the trial, including full eligibility criteria, can be found on the US National Library of Medicine website. The study is expected to be completed on March 19, 2025.

Myasthenia gravis is characterized by an IgG-mediated autoimmune response against the neuromuscular junction. 

Nipocalimab is a high-affinity, fully human IgG1 anti-neonatal Fc receptor (FcRn) monoclonal antibody. FcRn helps prevent the degradation of circulating IgG. Thus, by blocking the activity of FcRn, it is thought that nipocalimab can increase IgG1 degradation, thereby improving nerve-to-muscle signals and muscle function in patients with generalized myasthenia gravis.

Reference

A study of nipocalimab in children aged 2 to less than 18 years with generalized myasthenia gravis. ClinicalTrials.gov. March 3, 2022. Accessed March 8, 2022.