NCT06150833

Efficacy and Safety and Pharmacokinetics of Boya IVIG

Efficacy, Safety and Pharmacokinetics of Boya Intravenous Immunoglobulin (IVIG) in Participants With Primary Immunodeficiency

• Status: Unknown
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Azidus Brasil · Industry
• Sex: All
• Age: 6 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the safety, efficacy and pharmacokinetic properties of Boya's IVIG preparation in participants with PID aged less than 60 years and more than 6 years.

Detailed description

This is a phase 3, open-label, prospective, single-group, multicenter study to evaluate the efficacy of IVIG in keeping the average number of serious bacterial infections to less than one per year. The safety and pharmacokinetics (PK) of the investigational product will also be assessed. Fifty male or female participants aged up to 60 years will be selected, with at least 20 participants aged between 06 and 17 years. During the study, at least 20 adult participants will be invited to make up the subgroups evaluating the pharmacokinetic parameters. The main benefit of IVIG is to help the immune system respond to a wide range of infections, which are often correlated with high morbidity and mortality rates in individuals with PID, particularly in cases of CVID and XLA. In addition, a reduction in the use of medication and hospitalizations is expected, promoting an improvement in the quality of life of these patients. IVIG therapy is generally safe, although unwanted effects are reported in a proportion ranging from 1% to 81% of patients or infusions, with an average incidence of 30% to 40% among patients and 5% to 15% among infusions. These effects can manifest themselves in varying degrees of intensity, ranging from mild to severe. They can occur immediately, during or shortly after the infusion, as well as late, appearing hours or even days after the procedure. Most adverse events are mild and immediate, occurring in the first few infusions, related to the infusion rate and quickly reversible. Headache, fever, general malaise, flu-like symptoms, nausea, chills, fatigue, myalgia, low back pain, tachycardia, changes in blood pressure and erythroderma are the most common events. Serious reactions occur in less than 1% of applications and usually with the use of higher doses, indicated in autoimmune and inflammatory diseases. Special care is needed in patients with comorbidities such as heart disease, nephropathy, liver disease, coagulation disorders (thrombophilia) and diabetes mellitus. In these patients, certain characteristics of IVIG should be assessed, such as the presence of sugars, osmolality, sodium, among others.

Conditions

• Primary Immunodeficiency Disease

Interventions

Type	Name	Description
BIOLOGICAL	Boya IVIG	The initial dose and other dose changes will be determined by the investigator on a case-by-case basis aiming to prevent infection and minimum serum IgG levels of 5 g/L. The total number of doses administered will depend on the treatment regimen and run-in period: * Between 16 and 20 intravenous injections for participants receiving infusions every 28 days, or; * Between 21 and 25 intravenous injections for participants receiving infusions every 21 days

Timeline

• Start date: 2024-04-01
• Primary completion: 2025-04-30
• Completion: 2025-09-30
• First posted: 2023-11-29
• Last updated: 2023-11-29