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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05986734
Other study ID # NCPHOI-2022-09
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date September 1, 2022
Est. completion date December 1, 2023

Study information

Verified date March 2023
Source Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Contact Elena f Deripapa, PD
Phone 89067777058
Email deripapa@mail.ru
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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


Description:

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.


Recruitment information / eligibility

Status Recruiting
Enrollment 100
Est. completion date December 1, 2023
Est. primary completion date July 31, 2023
Accepts healthy volunteers
Gender All
Age group 1 Day to 18 Years
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

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.

Locations

Country Name City State
Russian Federation Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology Moscow

Sponsors (1)

Lead Sponsor Collaborator
Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Country where clinical trial is conducted

Russian Federation, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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
Primary 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 Pre-transfusion IgG level 6 months from study enrollment
Secondary Frequency of unscheduled antibiotic use during the study drug therapy 6 months from study enrollment
Secondary Duration of unscheduled antibiotic use during the study drug therapy 6 months from study enrollment
Secondary The number of hospitalisations due to infectious diseases during the study drug therapy. 6 months from study enrollment
Secondary Length of hospital stay due to infectious diseases during the study drug therapy 6 months from study enrollment
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