Evaluation of Subcutaneous Immunoglobulin Product Cutaquig in Terms of Safety and Efficacy in the Treatment of Patients With Primary Immunodeficiencies

Study Overview
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Brief Summary
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In the prospective part of the study, patients diagnosed with PID will receive replacement therapy with Cutaquig for at least 6 months and will be randomised with the help of physicians of the Russian PID Registry. At the beginning of the study, data on the prior intravenous immunoglobulin (IVIG) therapy efficacy will be extracted from patients' medical records (retrospective part of study). Also, at the beginning of the study, patients or their parents will be asked to complete Quality of Life Questionnaire for children, version 4.0; short form in Russian - Pediatric Quality of Life Inventory Russian, Version 4.0 (Russia), PedSQL (Appendix 14.1). During the 6 months of treatment with Cutaquig, investigators will enter information on infectious episodes, IgG levels (at least 3 times during the study period), and definition of an adverse drug reaction / serious adverse drug reaction, in the database. After 6 months, patients/their parents will be asked to complete the PedSQL, Quality of Life Questionnaire again. If the patient continues to receive the drug, and patient and his/her legally acceptable representatives (for patients under 14 years of age) agree to continue participating in the study, similar information will be collected for another 6 months. The first 6 months are planned for the main study period, 6-12 months for an extended study period. Data obtained in the prospective phase of the study will be compared with similar data obtained in the retrospective phase in the same patients

Detailed Description
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In the prospective part of the study, patients diagnosed with PID will receive replacement therapy with Cutaquig for at least 6 months and will be randomised with the help of physicians of the Russian PID Registry. At the beginning of the study, data on the prior intravenous immunoglobulin (IVIG) therapy efficacy will be extracted from patients' medical records (retrospective part of study). Also, at the beginning of the study, patients or their parents will be asked to complete Quality of Life Questionnaire for children, version 4.0; short form in Russian - Pediatric Quality of Life Inventory Russian, Version 4.0 (Russia), PedSQL (Appendix 14.1). During the 6 months of treatment with Cutaquig, investigators will enter information on infectious episodes, IgG levels (at least 3 times during the study period), and definition of an adverse drug reaction / serious adverse drug reaction, in the database. After 6 months, patients/their parents will be asked to complete the PedSQL, Quality of Life Questionnaire again. If the patient continues to receive the drug, and patient and his/her legally acceptable representatives (for patients under 14 years of age) agree to continue participating in the study, similar information will be collected for another 6 months. The first 6 months are planned for the main study period, 6-12 months for an extended study period. Data obtained in the prospective phase of the study will be compared with similar data obtained in the retrospective phase in the same patients. Considering the lack of the study drug marketing authorisation in the Russian Federation, study drug doses and treatment regimens were chosen as per current clinical practice and instructions for human use approved in other countries. Conventionally, on switching from IVIG therapy, the subcutaneous immunoglobulin products are administered at the same monthly dose (0.4-0.8 g/kg), evenly divided into 3-5 administrations per month, subject to monitoring of the pre transfusion IgG level with target IgG values of at least 7 g/L. All this information will be reflected in the Case record forms (CRF). Besides, information about therapeutic strategies, such as dose change or therapy change, will also be entered in the CRF.

Official Title
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Evaluation of Subcutaneous Immunoglobulin Product Cutaquig in Terms of Safety and Efficacy in the Treatment of Patients With Primary Immunodeficiencies

Conditions
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Primary Immunodeficiency Diseases (PID)

Intervention / Treatment
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* Drug: Cutaquig


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients of both sexes over the age of 0 years with a verified diagnosis of primary immunodeficiency, who require regular immunoglobulin replacement therapy Patients' and/or their legal representatives' (for patients under 14 years of age) consent to participate in the study Exclusion Criteria: Hypersensitivity to the study drug or any component of the study drug Active oncological disease Condition after haematopoietic stem cell transplantation Use of other immunoglobulin products during the study drug therapy. Discontinuation or irregular use of the study drug

Ages Eligible for Study
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Minimum Age: 1 Day
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Cutaquig|Subcutaneous immunoglobulin products are administered at the same monthly dose (0.4-0.8 g/kg), evenly divided into 3-5 administrations per month, subject to monitoring of the pre transfusion IgG level with target IgG values of at least 7 g/L.|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| comparative characteristics of serum IgG concentrations in IVIG and SCIG therapy | analysis of the results of the study after 3 months from the start of subcutaneous immunoglobulin therapy: assessment of IgG concentration over time, box plot | after 3 months from the start | 
| Comparison of the average number of infectious complications per person per month on IVIG and SCIG therapy | analysis of the results of the study after 3 months from the start of subcutaneous immunoglobulin therapy: the frequency of infectious complications, box plot | after 3 months from the start | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pre-transfusion IgG level |  | 6 months from study enrollment | 
| Frequency of unscheduled antibiotic use during the study drug therapy |  | 6 months from study enrollment | 
| Duration of unscheduled antibiotic use during the study drug therapy |  | 6 months from study enrollment | 
| The number of hospitalisations due to infectious diseases during the study drug therapy. |  | 6 months from study enrollment | 
| Length of hospital stay due to infectious diseases during the study drug therapy |  | 6 months from study enrollment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Primary immunodeficiency diseases, Cutaquig, intravenous immunoglobulin, subcutaneous immunoglobulin