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

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

Study Overview

• Status: Recruiting
• Conditions: Primary Immunodeficiency Diseases (PID)
• Intervention / Treatment: Drug: Cutaquig

Detailed 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.

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

• Study Contact: Name: Elena f Deripapa, PD; Phone Number: 89067777058; Email: deripapa@mail.ru

Study Locations

• Russian Federation
  • Moscow, Russian Federation, 117997
    • Recruiting
    • Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
    • Contact: Anna Shcherbina, MD, PhD; Phone Number: 6299 +7(495)2876570; Email: shcher26@hotmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: N/A

• Sampling Method: Probability Sample

Study Population

patients diagnosed with PID. Patients of both sexes over the age of 0 years with a verified diagnosis of primary immunodeficiency, who require regular immunoglobulin replacement therapy

Description

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 Plan

How is the study designed?

What is the study measuring?

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	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

Collaborators and Investigators

• Sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology
• Investigators: Study Director: Anna Shcherbina, MD,Phd, Chief HSCT department at Federal Research Center for pediatric hematology, oncology and immunology

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2022
• Primary Completion (Estimated): July 31, 2023
• Study Completion (Estimated): December 1, 2023

Study Registration Dates

• First Submitted: March 6, 2023
• First Submitted That Met QC Criteria: August 8, 2023
• First Posted (Actual): August 14, 2023

Study Record Updates

• Last Update Posted (Actual): August 14, 2023
• Last Update Submitted That Met QC Criteria: August 8, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Keywords: intravenous immunoglobulin; subcutaneous immunoglobulin; Primary immunodeficiency diseases; Cutaquig
• Additional Relevant MeSH Terms: Immune System Diseases; Genetic Diseases, Inborn; Immunologic Deficiency Syndromes; Primary Immunodeficiency Diseases; Physiological Effects of Drugs; Immunologic Factors; Immunoglobulins
• Other Study ID Numbers: NCPHOI-2022-09

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No