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)

NCT04636502 | Completed

• 1 other identifier: PID-4001
• Study Type: observational
• Target: 96
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

Primary Immunodeficiencies (PID)

Outcome Measures

Primary Outcomes (10)

• 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)

Study Arms (1)

Cohort

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

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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.

You may not qualify if:

• None

Sponsors & Collaborators

• Shire: lead

Study Sites (5)

Uniwersyteckie Centrum Kliniczne

Gdansk, 80-952, Poland

Location

Uniwersytecki Szpital Dziecięcy w Krakowie

Krakow, 30-663, Poland

Location

Instytut "Pomnik - Centrum Zdrowia Dziecka"

Warsaw, 04-730, Poland

Location

Wojewódzki Szpital Specjalistyczny im. J. Gromkowskiego

Wroclaw, 51-149, Poland

Location

Samodzielny Publiczny Szpital Kliniczny Nr 1 im. prof. Stanisława Szyszko Śląskiego Uniwersytetu Medycznego w Katowicach

Zabrze, 41-800, Poland

Location

Related Publications (2)

• Mach-Tomalska M, Pituch-Noworolska A, Bien E, Malanowska M, Machura E, Pukas-Bochenek A, Chrobak E, Pac M, Pietrucha B, Drygala S, Kamieniak M, Kasprzak J, Heropolitanska-Pliszka E. Facilitated subcutaneous immunoglobulin treatment patterns in pediatric patients with primary immunodeficiency diseases. Immunotherapy. 2024 Apr;16(6):391-403. doi: 10.2217/imt-2023-0305. Epub 2024 Feb 16.

  PMID: 38362629 DERIVED

• Heropolitanska-Pliszka E, Pac M, Pietrucha B, Machura E, Pukas-Bochenek A, Chrobak E, Bien E, Malanowska M, Pituch-Noworolska A, Drygala S, Kamieniak M, Kasprzak J, Mach-Tomalska M. Subcutaneous immunoglobulin 20% (Ig20Gly) treatment regimens in pediatric patients with primary immunodeficiencies - real-world data from the IG TATRY study. Expert Rev Clin Immunol. 2023 Jul-Dec;19(10):1281-1291. doi: 10.1080/1744666X.2023.2240514. Epub 2023 Jul 25.

  PMID: 37489744 DERIVED

Related Links

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

Study Officials

• Study Director

  Shire

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 18, 2020
• First Posted: November 19, 2020
• Study Start: February 8, 2021
• Primary Completion: September 14, 2021
• Study Completion: September 14, 2021
• Last Updated: November 17, 2021

Record last verified: 2021-11

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• 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.

Locations

PL (5)