Primary Immunodeficiencies (PID)IG-TATRYNCT04636502
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)
An observational study of Primary Immunodeficiencies (PID), run by Shire at 5 sites in Poland.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Primary Immunodeficiencies (PID)
- How many people
- 96 participants
- Where
- 5 sitesPoland
- Study dates
- 8 Feb 2021to 14 Sep 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Participants with PID on treatment with SCIG 20% or fSCIG will be included in this study.
Who may be able to join
- 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.
Who cannot join
- None
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
Poland5 sites
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-952
Uniwersytecki Szpital Dziecięcy w Krakowie
Krakow, 30-663
Instytut "Pomnik - Centrum Zdrowia Dziecka"
Warsaw, 04-730
Wojewódzki Szpital Specjalistyczny im. J. Gromkowskiego
Wroclaw, 51-149
Samodzielny Publiczny Szpital Kliniczny Nr 1 im. prof. Stanisława Szyszko Śląskiego Uniwersytetu Medycznego w Katowicach
Zabrze, 41-800
About this study
Official title: 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.
Read the 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
- Conditions
- Primary Immunodeficiencies (PID)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
Number of Doses per Infusion
Measured: From start of the study upto end of the study (6 months)
Number of Infusion Sites
Measured: From start of the study upto end of the study (6 months)
Infusion Volume
Measured: 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.
Measured: From start of the study upto end of the study (6 months)
Length of Needle
Length of needle used for infusions will be assessed.
Measured: 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.
Measured: From start of the study upto end of the study (6 months)
Type of Pump
Measured: 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.
Measured: From start of the study upto end of the study (6 months)
Total Infusion Volume per 4 Weeks
Measured: From start of the study upto end of the study (6 months)
Treatment Interval per 4 Weeks
Measured: From start of the study upto end of the study (6 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2020
- Study start8 Feb 2021
- Primary completion14 Sep 2021
- Study completion14 Sep 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Primary Immunodeficiencies (PID)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Retrospective
- Enrolment
- 96 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Shire
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04636502
- Sponsor's protocol ID
- PID-4001
- Acronym
- IG-TATRY
- Status last verified
- Nov 2021
- First submitted
- 18 Nov 2020
- Last updated
- 17 Nov 2021
- Results posted
- No
- Individual data sharing
- Yes: 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.
Publications
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.
PubMed 38362629Heropolitanska-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.
PubMed 37489744
Study at a glance
From the registry record, last updated by the sponsor on 17 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Subcutaneous Immune Globulin (SCIG 20%) and Facilitated Subcutaneous Immunoglobulin (fSCIG) Treatment in Polish Paediatric Patients With Primary Immunodeficiencies (PID) - Retrospective Medical Chart Review Study
- ClinicalTrials.gov ID
- NCT04636502
- Acronym / sponsor ID
- IG-TATRY / PID-4001
- Conditions
- Primary Immunodeficiencies (PID)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 96 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 8 Feb 2021 (actual)
- Primary completion
- 14 Sep 2021 (actual)
- Study completion
- 14 Sep 2021 (actual)
- First posted
- 19 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Other
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cohort | – | –Participants who had treated with fSCIG (HyQvia) for not more than 27 months and SCIG 20% (Cuvitru) for not more than 35 months. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Participants with PID on treatment with SCIG 20% or fSCIG will be included in this study.
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Number of Doses per Infusion
Time frame: From start of the study upto end of the study (6 months)
Number of Infusion Sites
Time frame: From start of the study upto end of the study (6 months)
Infusion Volume
Time frame: From start of the study upto end of the study (6 months)
Method of Administration
Time frame: From start of the study upto end of the study (6 months)
Method of administration in participants will be through pump or rapid push.
Length of Needle
Time frame: From start of the study upto end of the study (6 months)
Length of needle used for infusions will be assessed.
Assessment of Person who Perform the Infusion
Time frame: From start of the study upto end of the study (6 months)
Assessment of person (parent/guardian or self) who performs the infusion will be assessed.
Type of Pump
Time frame: From start of the study upto end of the study (6 months)
Total Dose per 4 weeks
Time frame: From start of the study upto end of the study (6 months)
Total dose administered per 4 weeks data will be assessed.
Total Infusion Volume per 4 Weeks
Time frame: From start of the study upto end of the study (6 months)
Treatment Interval per 4 Weeks
Time frame: From start of the study upto end of the study (6 months)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Nov 2021.
- Derived from this study: PubMed 38362629Mach-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.
- Derived from this study: PubMed 37489744Heropolitanska-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.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Shire
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Immunodeficiencies (PID), run by Shire at 5 sites in Poland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Doses per Infusion (time frame: From start of the study upto end of the study (6 months)). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04636502, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Nov 2021.