Clinical Research Directory

V-IMMUNE: A Novel Immunoglobulin Therapy for Immunodeficiency

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.

Assessing Patient Preference for Infusion Systems

This crossover study evaluates patient experiences and preferences between mechanical and electrical infusion pumps for subcutaneous immunoglobulin (SCIg) therapy. The Freedom Integrated Infusion System (FREEDOM60 and FreedomEdge) are mechanical, portable pumps that require no batteries or electricity and use Precision Flow Rate Tubing™ to control infusion speed. These devices are approved for use in the EU (CE650520). Approximately 78 adult patients with primary or secondary immunodeficiency will participate. Participants will complete questionnaires assessing ease of training, ease of use, infusion comfort, and overall satisfaction. Patients experienced with electronic pumps will complete two questionnaires: one reflecting their current pump experience and one after trying the mechanical pump. The primary goal is to determine whether mechanical pumps provide greater patient satisfaction than electrical pumps. Results will inform patient and healthcare professional decision-making regarding pump selection.