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

BoyaIVIG

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.

2024-04-01

2025-04-30

2025-09-30

Not yet recruiting

Early phase I

50

Inclusion Criteria: 1. Signature of written informed consent. 2. Men or women. 3. Age ≤ 60 years. 4. Diagnosis of PID disease (PID) with a reduction in antibody production due to: 1. Common variable immunodeficiency (CVID) as defined ESID/PAGID, OR 2. X-linked agammaglobulinemia (XLA) as defined by ESID/PAGID. 5. Receiving intravenous immunoglobulin replacement therapy at 21- or 28-day intervals at 300 to 600 mg/kg/month for a minimum of 2 months prior to study entry. 6. Absence of episodes of serious bacterial infections with prior use of IV immunoglobulin for at least 3 months prior to screening. 7. Negative pregnancy test (in female participants of childbearing potential); readiness to use reliable contraceptive methods throughout the study period. 8. Patients who have participated in a clinical study with another investigational IVIG may be included if they have a potential benefit in accordance with CNS Res. 251/1997. 9. Participants undergoing treatment with any subcutaneous or intramuscular immunoglobulin may be included by switching to IVIG therapy at the discretion of the investigator, considering the possible benefit to the participant. Exclusion Criteria: 1. Known intolerance or hypersensitivity to immunoglobulins or components of the test article; 2. Any contraindications to the use of immunoglobulins; 3. Secondary immunodeficiency or conditions potentially causing secondary immunodeficiency such as chronic lymphoid leukemia, lymphoma, multiple myeloma, protein-losing enteropathies or nephropathies, and hypoalbuminemia; 4. Clinically relevant changes in the safety exams are defined as: * Blood count * Hb \< 10.5 g/dL * Leukocytes \< 3,000 / mm3 or \>10,000 cells / mm3 * Absolute neutrophil count \< 1,000 cells/mm3; * Coagulation * TP 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 * Urine I. * Leukocyturia \> 10,000 cells/mL 5. Any cancer either active or resolved within the last 12 months before screening; 6. Receiving any blood products (except intravenous immunoglobulins) during the last 3 months before screening; 7. Any febrile illness within 14 days before enrollment; Note: The patient may be rescreened after recovery. 8. History of thrombotic events (including myocardial infarction, stroke, pulmonary embolism, and deep vein thrombosis) within 6 months before enrollment; 9. Previous use of live attenuated virus vaccines; 10. Selective deficiency of immunoglobulin A (IgA) or known antibodies to IgA; 11. Known drug or alcohol abuse; 12. The need to use other investigational drugs, systemic immunosuppressants, and any other immunoglobulins; 13. Pregnancy or lactation; 14. Inability to comply with the protocol activities; 15. PIDs other than CVID or X-linked agammaglobulinemia 16. Patients infected with HIV, HBV or HCV 17. Patients with AIDS, cystic fibrosis, or active hepatitis B or C. 18. Any other condition that, in the Investigator's opinion may increase the risk of participation in this study.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Patients with primary immunodeficiency will 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 remaining pharmacokinetic parameters will be measured between visits 4 and 5 in 20 adult participants, collecting additional blood samples.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE', 'maskingDescription': 'The trial will be open-label and single-arm. Therefore, there will be no randomization or blinding.'}}, 'enrollmentInfo': {'count': 50, 'type': 'ESTIMATED'}}

2023-11-29