Clinical trials

This is a multicenter, prospective clinical trial evaluating the efficacy and safety of V-IMMUNE®, a 5% human normal immunoglobulin formulation administered intravenously, for the treatment of immune thrombocytopenia (ITP) in patients aged ≥1 year. The primary objective is to assess the proportion of patients achieving a platelet count ≥50,000/mm³ on or before Day 9 following the first infusion. The trial employs a single-group design, comparing outcomes to historical controls derived from the literature. Eligible patients must have a confirmed diagnosis of ITP with a platelet count ≤20,000/mm³ and no concurrent conditions likely to cause thrombocytopenia. Key exclusions include non-immune thrombocytopenia, active sepsis, pregnancy or lactation, hypersensitivity to blood products or IgG preparations, and various significant comorbidities (e.g., uncontrolled hypertension, severe hepatic or renal impairment, recent rituximab use). The intervention consists of V-IMMUNE® at a dose of 1 g/kg, administered once daily for two consecutive days, with infusion rates titrated from 0.01 mL/kg/min to 0.06 mL/kg/min. Standard pre-medication protocols (IV normal saline and diphenhydramine) are administered to mitigate infusion-related reactions and reduce the risk of thromboembolic events. Patients will be monitored at multiple time points from baseline through Day 90, with primary efficacy evaluation at Day 9. Secondary endpoints include duration of platelet response, overall treatment response rate, bleeding events, and incidence of infusion-related adverse events.

This is a phase III, non-randomized clinical trial (VIP Study) designed to assess the safety and efficacy of V-IMMUNE®, a 5% human normal immunoglobulin preparation, in approximately 50 patients with primary immunodeficiency (PID). Participants, all aged ≥2 years and already receiving IVIG therapy, will be switched to V-IMMUNE® at a dose of 600 mg/kg every three weeks via intravenous infusion. The study will use historical data as a control and extend over 12 months, with scheduled visits at each infusion (an estimated 17 infusions per participant). Objectives and Outcomes Primary Efficacy Endpoint: Rate of serious bacterial infections over 12 months. Primary Safety Endpoint: Proportion of infusions with one or more temporally associated adverse events (AEs). Secondary Endpoints: Additional safety outcomes (e.g., average number of AEs within 72 hours per infusion), efficacy measures (non-serious bacterial infections, time to resolution, antibiotic use, hospitalizations), and quality of life (SF-36) at 6 and 12 months. A pharmacokinetic (PK) sub-study will be conducted in 20 participants aged ≥16 years to evaluate total IgG levels, half-life, AUC, Cmax, and other PK parameters. Study Design and Intervention V-IMMUNE® is given at an initial infusion rate of 0.01 mL/kg/min for 30 minutes, increasing stepwise up to 0.06 mL/kg/min if well tolerated. Pre-medication, including rapid IV saline, diphenhydramine, and hydrocortisone, will be administered for the first three months to reduce the risk of infusion-related AEs. Patients at elevated thromboembolic risk will receive the lowest feasible infusion rate. Sample Size and Analysis Fifty patients total will be enrolled to ensure adequate power to demonstrate a severe infection rate below one event per person-year (with a one-sided 1% significance level). Safety endpoints will be met if the upper bound of the 95% confidence interval for the proportion of temporally associated infusion-related AEs remains below 40%, assuming a true rate under 20%. An interim analysis is planned at six months or upon reaching 50% enrollment. 20 patients at total including adults and \<16 years old, 6 children from 2 to 12 years old and 6 children from 12 to 16 years old.