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

NCT05986734 | Unknown

• 1 other identifier: NCPHOI-2022-09
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 1

Brief 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

Conditions

Primary Immunodeficiency Diseases (PID)

Keywords

Primary immunodeficiency diseases; Cutaquig; intravenous immunoglobulin; subcutaneous immunoglobulin

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (5)

• 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

Interventions

Cutaquig DRUG

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.

Also known as: subcutaneous immunoglobulin, Octapharma

Eligibility Criteria

• Age: 1 Day - 18 Years
• Sex: all
• Age Groups: Child (0-17), Adult (18-64)
• 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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Federal Research Institute of Pediatric Hematology, Oncology and Immunology: lead

Study Sites (1)

Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology

Moscow, 117997, Russia

RECRUITING

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases

Study Officials

• Anna Shcherbina, MD,Phd

  Chief HSCT department at Federal Research Center for pediatric hematology, oncology and immunology

  STUDY DIRECTOR

Central Study Contacts

Elena f Deripapa, PD

CONTACT

89067777058; deripapa@mail.ru

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 6, 2023
• First Posted: August 14, 2023
• Study Start: September 1, 2022
• Primary Completion: July 31, 2023
• Study Completion: December 1, 2023
• Last Updated: August 14, 2023

Record last verified: 2023-03

Data Sharing

• IPD Sharing: Will not share

Locations

RU (1)