Retrospective, Observational Chart Review Study Conducted in Poland to Document the Management and Clinical Outcome of CUVITRU and HYQVIA in Pediatric Participants (< 18 Years) With Primary Immunodeficiency (PID) (IG-TATRY)

Subcutaneous Immune Globulin (SCIG 20%) and Facilitated Subcutaneous Immunoglobulin (fSCIG) Treatment in Polish Paediatric Patients With Primary Immunodeficiencies (PID) - Retrospective Medical Chart Review Study

The purpose of this observational, multi-center, retrospective cohort study is to assess treatment patterns of Cuvitru (SCIG) 20 percent (%) and HyQvia (fSCIG) in polish pediatric participants with PID. The study will collect pediatric patient data. These data are gathered and collected during routine clinical care. As this is a non-interventional/observational study, no treatment/pharmacotherapy is provided as part of the study.

Study Overview

• Status: Completed
• Conditions: Primary Immunodeficiencies (PID)

Detailed Description

Subcutaneous immune globulin (SCIG 20%) and facilitated subcutaneous immunoglobulin (fSCIG) treatment in Polish paediatric patients with primary immunodeficiencies (PID) - retrospective medical chart review study

• Study Type: Observational
• Enrollment (Actual): 96

Contacts and Locations

Study Locations

• Poland
  • Gdańsk, Poland, 80-952
    • Uniwersyteckie Centrum Kliniczne
  • Kraków, Poland, 30-663
    • Uniwersytecki Szpital Dzieciecy w Krakowie
  • Warszawa, Poland, 04-730
    • Instytut "Pomnik - Centrum Zdrowia Dziecka"
  • Wrocław, Poland, 51-149
    • Wojewódzki Szpital Specjalistyczny im. J. Gromkowskiego
  • Zabrze, Poland, 41-800
    • Samodzielny Publiczny Szpital Kliniczny Nr 1 im. Prof. Stanisława Szyszko Śląskiego Uniwersytetu Medycznego w Katowicach

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Participants with PID on treatment with SCIG 20% or fSCIG will be included in this study.

Description

Inclusion Criteria:

Participant eligibility is determined according to the following criteria prior to entry into the study:

• The participant or, when applicable, the participant's legally acceptable representative signs and dates a written, informed consent form and any required privacy authorization prior to the initiation of any study procedures.
• Male or female participants with PID, aged less than (<)18 years treated with SCIG 20% or fSCIG..
• Diagnosis of PID according to the criteria developed by the European Society for Immunodeficiencies (ESID) https://esid.org/About-ESID
• Treatment period for 20% SCIG ranges from November 1, 2017 until June 30, 2020, and for fSCIG from July 1, 2018 until June 30, 2020.

Exclusion Criteria:

• None

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Retrospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Cohort; Participants who had treated with fSCIG (HyQvia) for not more than 27 months and SCIG 20% (Cuvitru) for not more than 35 months.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Doses per Infusion		From start of the study upto end of the study (6 months)
Number of Infusion Sites		From start of the study upto end of the study (6 months)
Infusion Volume		From start of the study upto end of the study (6 months)
Method of Administration	Method of administration in participants will be through pump or rapid push.	From start of the study upto end of the study (6 months)
Length of Needle	Length of needle used for infusions will be assessed.	From start of the study upto end of the study (6 months)
Assessment of Person who Perform the Infusion	Assessment of person (parent/guardian or self) who performs the infusion will be assessed.	From start of the study upto end of the study (6 months)
Type of Pump		From start of the study upto end of the study (6 months)
Total Dose per 4 weeks	Total dose administered per 4 weeks data will be assessed.	From start of the study upto end of the study (6 months)
Total Infusion Volume per 4 Weeks		From start of the study upto end of the study (6 months)
Treatment Interval per 4 Weeks		From start of the study upto end of the study (6 months)

Collaborators and Investigators

• Sponsor: Shire

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link

Study record dates

Study Major Dates

• Study Start (Actual): February 8, 2021
• Primary Completion (Actual): September 14, 2021
• Study Completion (Actual): September 14, 2021

Study Registration Dates

• First Submitted: November 18, 2020
• First Submitted That Met QC Criteria: November 18, 2020
• First Posted (Actual): November 19, 2020

Study Record Updates

• Last Update Posted (Actual): November 17, 2021
• Last Update Submitted That Met QC Criteria: November 15, 2021
• Last Verified: November 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Genetic Diseases, Inborn; Immunologic Deficiency Syndromes; Primary Immunodeficiency Diseases
• Other Study ID Numbers: PID-4001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: Study Protocol; Statistical Analysis Plan (SAP); Informed Consent Form (ICF); Clinical Study Report (CSR)