Clinical Evaluation of Pharmacokinetics, Efficacy, and Safety of a 10% Immunoglobulin in Primary Immunodeficiency Disease (CARES10 Study)

INTRODUCTION: Differential tolerability and side effects of intravenous immunoglobulin G (IVIg) therapy to treat primary immunodeficiency (PI) have resulted in an unmet need for alternative treatment options. This study was conducted to evaluate the safety and efficacy of a newly formulated IVIg, Kedrion IVIg 10% (KIg10). METHODS: The CARES10 (Clinical Assessment of phaRmacokinetics, Efficacy, and Safety of 10% IVIg in PID patients) study was a phase III, open-label, prospective, single-arm, historically controlled, multicenter study of KIg10 (200-800 mg/kg) in previously treated patients with PI. RESULTS: A total of 47 patients with PI were enrolled in the study and received infusions of KIg10 on a 21- or 28-day dosing schedule for a period of 48 weeks. All study participants completed the trial, and 44 of the 47 reached the maximum allowed infusion rate for the study. No acute serious bacterial infections (SBI) were reported. All patients had post-baseline IgG trough levels of ≥ 6 g/L at all study visits, except for one patient at a single visit, and all patients maintained IgG subclass distributions comparable to those in healthy subjects. KIg10 pharmacokinetics were measured in a subset of patients (n = 23). The primary adverse events observed were similar in frequency to other IVIg products and did not raise any safety concerns. CONCLUSION: KIg10 was well tolerated and effective for treating patients with PI, and presents a potential additional therapeutic option for treating patients with PI. TRIAL REGISTRATION: ClinicalTrials.gov identifier, NCT03961009.