Efficacy, Safety, and Pharmacokinetics of Shu Yang IVIG in Patients With Primary Immunodeficiency

IMUNOFORTE

To evaluate the safety, efficacy, and pharmacokinetic properties of Shu Yang intravenous immune globulin in patients with primary immune deficiency aged less than 60 years. The main benefit of IVIG is to help the body fight against a large variety of infections generally associated with morbidity and mortality in patients with primary immunodeficiency diseases, particularly in CVID and XLA. In addition, a decrease in the number of infections, a reduction in medications and hospitalizations, and a better quality of life are expected. Throughout treatment, approximately one-fourth of persons may experience a side effect. These are usually mild or bothersome but not dangerous. Very rarely, more serious side effects like allergic reactions or low blood counts (anemia) can occur. One of the most common side effects is headache. Other side effects include chills, fever, flushing, flu-like muscle pains or joint pains, feeling tired, nausea, vomiting, and rash. For the most part, these reactions typically happen with the first dose of IVIG or because change to a different brand of IVIG. All IVIG products have similar warnings and contraindications, such as the potential for renal failure, thrombotic events, aseptic meningitis, hemolysis, and anaphylactic reactions.

2024-04-01

2025-01-08

2025-06-08

Not yet recruiting

Early phase I

50

Inclusion Criteria: 1. Written informed consent/assent. 2. Male or female. 3. Ages ≤ 60 years old and ≥ 06 years old. 4. Diagnosis of Primary Immunodeficiency Disease (PID) with a reduction in antibody production due to: a. Common Variable Immunodeficiency (CVID) as per European Immunodeficiency Society (ESID)/Pan American Immunodeficiency Group (PAGID), as defined in section 5.1, OR b. X-linked agammaglobulinemia (XLA) as per ESID/PAGID, as defined in section 5.1. 5. Receiving replacement therapy with intravenous immunoglobulin at 21- to 28-day intervals at 300-600 mg/kg/month for a minimum of 2 months before the start of the study; 6. Absence of episodes of serious bacterial infections with previous use of an IV immunoglobulin for at least 3 months before screening; 7. Negative pregnancy test (in female patients with childbearing potential); readiness to use reliable methods of contraception throughout the study period; 8. Patients who participated in a clinical trial with another experimental IVIG may be enrolled if they have a potential benefit according to Res. CNS 251/1997; 9. Patients currently on treatment with any subcutaneous or intramuscular immunoglobulin may be enrolled switching to IVIG therapy at the investigator's discretion, considering the potential benefit to the patient. 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 o Hb \< 10.5 g/dL o Leukocytes \< 3,000 /uL or \>10,000 cells / uL o Absolute neutrophil count \< 1,000 cells/mm3; * Coagulation o TP and aPTT \> 2.5 x ULN * Biochemistry o 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 Shu Yang 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 at all visits. At Visit 4, at least 20 patients will perform the PK assessment, 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'}}

2024-03-12