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

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ClinicalTrials.gov Identifier: NCT05986734
Recruitment Status : Recruiting
First Posted : August 14, 2023
Last Update Posted : August 14, 2023
Sponsor:
Information provided by (Responsible Party):
Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Study Description
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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

Condition or disease Intervention/treatment
Primary Immunodeficiency Diseases (PID) 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 Design
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Study Type : Observational
Estimated Enrollment : 100 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Evaluation of Subcutaneous Immunoglobulin Product Cutaquig in Terms of Safety and Efficacy in the Treatment of Patients With Primary Immunodeficiencies
Actual Study Start Date : September 1, 2022
Estimated Primary Completion Date : July 31, 2023
Estimated Study Completion Date : December 1, 2023

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Intervention Details:
  • 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.
    Other Names:
    • subcutaneous immunoglobulin
    • Octapharma

Outcome Measures
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Primary Outcome Measures :
  1. comparative characteristics of serum IgG concentrations in IVIG and SCIG therapy [ Time Frame: after 3 months from the start ]
    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

  2. Comparison of the average number of infectious complications per person per month on IVIG and SCIG therapy [ Time Frame: after 3 months from the start ]
    analysis of the results of the study after 3 months from the start of subcutaneous immunoglobulin therapy: the frequency of infectious complications, box plot


Secondary Outcome Measures :
  1. Pre-transfusion IgG level [ Time Frame: 6 months from study enrollment ]
  2. Frequency of unscheduled antibiotic use during the study drug therapy [ Time Frame: 6 months from study enrollment ]
  3. Duration of unscheduled antibiotic use during the study drug therapy [ Time Frame: 6 months from study enrollment ]
  4. The number of hospitalisations due to infectious diseases during the study drug therapy. [ Time Frame: 6 months from study enrollment ]
  5. Length of hospital stay due to infectious diseases during the study drug therapy [ Time Frame: 6 months from study enrollment ]

Eligibility Criteria
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Ages Eligible for Study:   1 Day to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
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
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05986734


Contacts
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Contact: Elena f Deripapa, PD 89067777058 deripapa@mail.ru

Locations
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Russian Federation
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology Recruiting
Moscow, Russian Federation, 117997
Contact: Anna Shcherbina, MD, PhD    +7(495)2876570 ext 6299    shcher26@hotmail.com   
Sponsors and Collaborators
Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Investigators
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Study Director: Anna Shcherbina, MD,Phd Chief HSCT department at Federal Research Center for pediatric hematology, oncology and immunology
More Information
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Responsible Party: Federal Research Institute of Pediatric Hematology, Oncology and Immunology
ClinicalTrials.gov Identifier: NCT05986734    
Other Study ID Numbers: NCPHOI-2022-09
First Posted: August 14, 2023    Key Record Dates
Last Update Posted: August 14, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Federal Research Institute of Pediatric Hematology, Oncology and Immunology:
Primary immunodeficiency diseases
Cutaquig
intravenous immunoglobulin
subcutaneous immunoglobulin
Additional relevant MeSH terms:
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Primary Immunodeficiency Diseases
Immunologic Deficiency Syndromes
Immune System Diseases
Genetic Diseases, Inborn
 Immunoglobulins
Immunologic Factors
Physiological Effects of Drugs