IVIG Boya: Safety, Efficacy, and Pharmacokinetics

NCT06150833 | Not Yet Recruiting Phase 3 DMC

• 1 other identifier: BoyaIVIG
• Study Type: interventional
• Target: 50
• Locations: 0 countries
• Sites: N/A

Brief Summary

The goal of this phase 3, open-label, single-group clinical trial is to assess the efficacy of Boya IVIG in maintaining the mean number of serious bacterial infections to less than one per year in participants with primary immunodeficiency (PYD) due to common variable immunodeficiency (CVID), as defined by the European Immunodeficiency Society (ESCID) / Pan American Immunodeficiency Group (PAGID), or X-linked agammaglobulinemia (XLA), as defined by molecular genetic testing (ESCID/PAGID). The safety and pharmacokinetics (PK) of the investigational product will also be evaluated. Participants must:

• Visit the research center every 21 or 28 days to receive the experimental product infusion and undergo a medical examination.
• During weekly telephone calls, report, if applicable, adverse events and hospitalizations; the number of school or workdays missed due to infections; the length of hospital stay; and the use of antibiotics for therapeutic purposes.

Conditions

Primary Immunodeficiency Disease

Keywords

IVIG; primary immunodeficiency disease; PID; common variable immunodeficiency; CVID; X-linked agammaglobulinemia; XLA

Outcome Measures

Primary Outcomes (1)

• Primary Efficacy Objective

  Average incidence of serious bacterial infections per participant between V0 and Vfinal.

  54 weeks (21-day interval schedule) or 56 weeks (28-day interval schedule)

Secondary Outcomes (4)

• Assessment of the rate of non-serious infections within one year

  Average incidence of non-serious infections per patient between Visit 0 and Final Visit (through study completion, an average of 1 year), as documented as treatment emergent adverse events (TEAEs).

• Missing time from school/work

  Average number of days off from school/work per patient/year, as collected in weekly telephone contacts.

• Length of hospitalization

  Number of days of hospitalization due to infections per participant/year between V0 and Vfinal, as documented as Adverse Events.

• Use of antibiotics

  Tabulation of all antibiotics used, separating those indicated for prophylactic and therapeutic purposes

Other Outcomes (9)

• Total IgG trough levels

  9 months

• Total IgG pharmacokinetic profile

  21 or 28 days

• Total IgG primary and secondary PK parameters

  21 or 28 days

Study Arms (1)

Boya IVIG

EXPERIMENTAL

All participants will receive Boya IVIG: Dose: 200 to 800 mg/kg Schedule of administration: 21- or 28-day interval Time frame: 54 weeks (21-day interval) or 56 weeks (28-day interval)

Biological: Boya IVIG

Interventions

Boya IVIG BIOLOGICAL

Intervention: Biological: Boya IVIG Boya IVIG is a 5% human immunoglobulin for intravenous administration

Also known as: IVIG, BOYA

Boya IVIG

Eligibility Criteria

• Age: 2 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Men or women; age between 02 and 60 years; written informed consent/assent.
• Diagnosis of primary immunodeficiency disease (PID) with decreased antibody production due to common variable immunodeficiency (CVID) or X-linked agammaglobulinemia (XLA).
• Treatment-naïve patients and those receiving intravenous immunoglobulin replacement therapy at 21- to 28-day intervals, with doses ranging from 300 to 800 mg/kg per infusion.
• Negative pregnancy test in females of childbearing potential; willingness to use effective contraceptive methods throughout the study.
• Subjects currently receiving any subcutaneous or intramuscular immunoglobulin may be enrolled by switching to IVIG therapy at the investigator's discretion, considering the potential benefit to the participant.

You may not qualify if:

• Known intolerance or hypersensitivity to immunoglobulins or components of the study drug.
• Any contraindications to the use of immunoglobulins.
• Patients with a BMI \< 18.5 or \> 40 kg/m2.
• Secondary immunodeficiency or clinical conditions that potentially cause secondary immunodeficiency, such as chronic lymphocytic leukemia, lymphoma, multiple myeloma, enteropathies, or nephropathies with protein loss and hypoalbuminemia.
• Clinically significant changes in safety assessments, defined as:
• Blood count
• Hb \< 10,5 g/dL
• Leukocytes \< 3,000 cells/mm3 or \> 11,000 cells/mm3
• Absolute neutrophil count \< 1,000 cells/mm3
• Coagulation:
• o PT and aPTT\> 2,5 x ULN.
• Biochemistry:
• glycated hemoglobin \> 6.5%
• total bilirubin and fractions, alkaline phosphatase, ALT, AST, GGT \> 2.5 x ULN
• creatinine above 3mg/dL or creatinine clearance \<30mL/min

Sponsors & Collaborators

• Azidus Brasil: lead
• Boya Bio Pharmaceutical Group Co Ltd: collaborator

Related Publications (4)

• World Health Organization (2005) Recommendations for the production, control and regulation of human plasma for fractionation, Annex 4, TRS nº 941, pp. 69.

  BACKGROUND

• EMA/CHMP/BPWP/94033/2007 rev. 4. Guideline on the clinical investigation of human normal immunoglobulin for intravenous administration (IVIg), Sep,2020.

  BACKGROUND

• Brasil, Resolução nº 251, de 07 de agosto de 1977. Disponível em; https://www.inca.gov.br/sites/ufu.sti.inca.local/files//media/document//resolucao-cns-251-97.pdf. Acesso em 25 de out de 2023

  BACKGROUND

• Food and Drug Administration (2006). Guidance for Clinical Trial Sponsors on the Establishment and Operation of Clinical Trial Data Monitoring Committees. Rockville, MD, pp. 38.

  BACKGROUND

MeSH Terms

Conditions

Primary Immunodeficiency Diseases; Common Variable Immunodeficiency; Bruton type agammaglobulinemia

Interventions

Immunoglobulins, Intravenous

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Luciana Ferrara

  Azidus Brasil

  STUDY DIRECTOR

Central Study Contacts

Luciana Ferrara

CONTACT

+55 19 981428814; luciana.ferrara@azidusbrasil.com.br

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Masking Details: The trial will be open-label and single-arm. Therefore, there will be no randomization or blinding.
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Patients with primary immunodeficiency will start therapy or switch to Boya IVIG and optimize the posology in a run-in period of 2 to 6 administrations. In the one-year test period, the patients will receive the test IVIG at 21- or 28-day intervals and be followed. IgG trough levels will be collected from all participants, at all visits. The pharmacokinetic profile will be measured after 5 infusions with stable dose in 20 adult participants, collecting additional blood samples.

Study Record Dates

• First Submitted: November 21, 2023
• First Posted: November 29, 2023
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): September 1, 2028
• Last Updated: June 3, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share