V-IMMUNE: A Novel Immunoglobulin Therapy for Immunodeficiency

Summary

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.

Conditions

• Immunodeficiencies
• Primary Immunodeficiencies (PID)
• Agammaglobulinemia

Interventions

BIOLOGICAL

Intravenous immunoglobulin (IVIG)

The investigational product is V-IMMUNE®, a 5% human normal immunoglobulin I.P. (5 g/100 mL) manufactured from qualified human plasma for intravenous use. Each vial contains human immunoglobulin at 50 g/L, maltose at 100 g/L, and water for injection. The 5% Human Immunoglobulin Solution for Intravenous Administration (I.P.) is a sterile and pyrogen-free preparation of human normal immunoglobulin in a single-dose form for intravenous administration. Each 10 mL, 50 mL, or 100 mL vial contains 0.5 g, 2.5 g, or 5 g of human normal immunoglobulin, respectively, and is produced from qualified human plasma using membrane filtration and a combination of chromatographic steps and viral inactivation procedures. The IgA content does not exceed 2 mg/mL. This manufacturing process uses plasma collected from donors who undergo screening according to guidelines set by regulatory authorities. In case of thromboembolic risk: use the lowest feasible dose

Sponsors & Collaborators

• Hospital do Coracao

  collaborator OTHER

• On Pharma Importadora, Exportadora e Distribuidora de Medicamentos LTDA.

  lead OTHER

Principal Investigators

• Israel Silva Maia, PhD · Hospital do Coracao

• Dewton Moraes Vasconcelos, PhD · Hospital do Coracao

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

2 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2025-08-15

Primary Completion

2026-09-27

Completion

2027-02-27

Countries

• Brazil

Study Locations