Primary ImmunodeficiencyFIGARONCT03054181
Facilitated Immunoglobulin Administration Registry and Outcomes Study (FIGARO)
An observational study of Primary Immunodeficiency and Secondary Immune Deficiency, run by GWT-TUD GmbH at 4 sites in 3 countries.
Trial summary
Long-term observational study on the utilisation and outcomes of HyQvia (a product consisting of recombinant human hyaluronidase and a human normal immunoglobulin 10% solution) under everyday clinical practice conditions. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Primary Immunodeficiency, Secondary Immune Deficiency
- What is being tested?
- HyQvia
- How many people
- 156 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 22 Dec 2016to 30 Nov 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with primary (PID) or secondary immunodeficiencies (SID)
Who may be able to join
- Patient has received/will receive at least 1 HyQvia infusion for PID or SID
- Patient has an indication for chronic immunoglobulin treatment
- Patient is likely available for long-term documentation
- Patient provides informed consent for documentation
Who cannot join
- No explicit exclusion criteria apply.
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
Germany2 sites
Charité
Berlin
Hospital for Children and Adolescents, St. Georg Hospital, Academic Teaching Hospital
Leipzig
About this study
Official title: Facilitated Immunoglobulin Administration Registry and Outcomes Study
Long-term observational study on the utilisation and outcomes of HyQvia (a product consisting of recombinant human hyaluronidase and a human normal immunoglobulin 10% solution) under everyday clinical practice conditions.
Read the detailed description
Recombinant hyaluronidase is an established therapeutic principle to facilitate the infusion of large volumes of fluids subcutaneously (e.g. Ringer solution and antibodies such as IG, rituximab, trastuzumab).
HyQvia is a product consisting of recombinant human hyaluronidase (rHuPH20, Hylenex®), and a human normal immunoglobulin (IG). The subcutaneous (SC) IG is a 10% solution prepared from human plasma consisting of at least 98% immunoglobulin G, which contains a broad spectrum of antibodies.
The two components are packaged together as a dual vial unit: IG provides the therapeutic effect and the recombinant human hyaluronidase facilitates the dispersion, which alters the kinetics of absorption and increases the bioavailability of the IG.
HyQvia is marketed in the European Union (EU) since July 2013 and in the USA since September 2014. In the EU, HyQvia is indicated as replacement therapy in patients of all age groups in primary immunodeficiency syndromes (PID), and in myeloma or chronic lymphocytic leukaemia with severe secondary hypogammaglobulinaemia and recurrent infections (secondary immunodeficiency syndromes, SID), as well as hypogammaglobulinaemia and pre- and post-allogeneic hematopoietic stem cell transplantation.
HyQvia was investigated in one pivotal study lasting over a year, involving 89 patients with PID who had already had treatment with human normal IG for at least three months. The number of acute serious bacterial infections (SBI) as main efficacy outcomes was 0.025 per year. This was below the FDA predefined number needed to show efficacy (SBI rate <1.0 per subject per year at the 0.01 level of significance), and was similar to that seen with other licensed human normal IG products. In the pivotal study and its extension, 188 patient years under HyQvia treatment have been documented. HyQvia was efficacious, safe, and bioequivalent to intravenous IG at the same administration intervals, but it caused fewer systemic reactions. Tolerability was good despite high infusion volumes and rates. There are no preclinical data that suggest an increased risk of mutagenicity, teratogenicity, fertility or neuronal development.
The most current and comprehensive data on real-life utilisation and outcomes of various IgG preparations is available from the German SIGNS registry. This registry confirmed the effectiveness of IgG substitution or treatment in terms of reduction of infections (PID and SID), as well as stabilization or improvement of the clinical condition in neurological and autoimmune diseases. In a cohort of 24 PID and SID patients on HyQvia in the German SIGNS study, the preparation was well tolerated and treatment satisfaction was high. In other countries, data on the utilisation and outcomes of HyQvia under clinical practice conditions are sparse or completely missing.
The present study aims to fill these gaps and to provide a detailed and complete description of the utilisation of under everyday clinical practice conditions.
- Being tested
- HyQvia
- Keywords
- Observational, Infection, Utilisation, Safety, Tolerability, Infusion, chronic
- Medical subject headings
- Primary Immunodeficiency Diseases, Infections, Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HyQviaBiological
Also called Recombinant human hyaluronidase and normal immunoglobulin 10%
| Study group | Role | What they receive |
|---|---|---|
| HyQvia | Recombinant human hyaluronidase and normal immunoglobulin 10% Receives: Biological: HyQvia |
What is being measured
The primary outcome is the main question the study is designed to answer.
Utilisation in terms of dose and dosing interval
mean monthly dose
Measured: up to 3 years
Secondary outcomes (7)
Adverse events
Incidence and type
Measured: up to 3 years
Concomitant diseases
Measured: up to 3 years
mean immunoglobulin trough level
after HyQvia infusion
Measured: up to 3 years
Number of participants with treatment-related adverse events
Acute bacterial infections; overall infections
Measured: up to 3 years
Number of training session
about appropriate self-infusion
Measured: up to 3 years
Number of days in hospital
Measured: up to 3 years
Number of days in rehabilitation clinic
Measured: up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Feb 2017
- Study start22 Dec 2016
- Primary completion30 Nov 2021
- Study completion30 Nov 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary ImmunodeficiencyStudies 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 156 participants (actual)
Sponsor and investigators
- Lead sponsor
- GWT-TUD GmbH
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Michael Borte, MD, PhD, Principal investigator, Fachbereich Pädiatrische Rheumatologie, Immunologie und Infektiologie am Klinikum St. Georg LeipzigDavid Pittrow, MD, Study director, GWT-TUD GmbH, Dresden, GermanyIsabelle Quinti, MD, Study chair, Sapienza University Rome, ItalyLeif Hanitsch, MD, Study chair, Charité Berlin, GermanyNizar Mahlaoui, MD, Study chair, University Hospital, Paris, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03054181
- Sponsor's protocol ID
- FIGARO
- Acronym
- FIGARO
- Status last verified
- Mar 2022
- First submitted
- 13 Feb 2017
- Last updated
- 14 Sep 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Borte M, Hanitsch LG, Mahlaoui N, Fasshauer M, Huscher D, Speletas M, Dimou M, Kamieniak M, Hermann C, Pittrow D, Milito C. Facilitated Subcutaneous Immunoglobulin Treatment in Patients with Immunodeficiencies: the FIGARO Study. J Clin Immunol. 2023 Aug;43(6):1259-1271. doi: 10.1007/s10875-023-01470-2. Epub 2023 Apr 10.
PubMed 37036560
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Facilitated Immunoglobulin Administration Registry and Outcomes Study
- ClinicalTrials.gov ID
- NCT03054181
- Acronym / sponsor ID
- FIGARO / FIGARO
- Keywords
- Observational, Infection, Utilisation, Safety, Tolerability, Infusion, chronic
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 156 participants (actual)
- Lead sponsor
- GWT-TUD GmbHUniversity, hospital or other
- Study start
- 22 Dec 2016 (actual)
- Primary completion
- 30 Nov 2021 (actual)
- Study completion
- 30 Nov 2021 (actual)
- First posted
- 15 Feb 2017 (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.
Long-term observational study on the utilisation and outcomes of HyQvia (a product consisting of recombinant human hyaluronidase and a human normal immunoglobulin 10% solution) under everyday clinical practice conditions.
Detailed description
Recombinant hyaluronidase is an established therapeutic principle to facilitate the infusion of large volumes of fluids subcutaneously (e.g. Ringer solution and antibodies such as IG, rituximab, trastuzumab).
HyQvia is a product consisting of recombinant human hyaluronidase (rHuPH20, Hylenex®), and a human normal immunoglobulin (IG). The subcutaneous (SC) IG is a 10% solution prepared from human plasma consisting of at least 98% immunoglobulin G, which contains a broad spectrum of antibodies.
The two components are packaged together as a dual vial unit: IG provides the therapeutic effect and the recombinant human hyaluronidase facilitates the dispersion, which alters the kinetics of absorption and increases the bioavailability of the IG.
HyQvia is marketed in the European Union (EU) since July 2013 and in the USA since September 2014. In the EU, HyQvia is indicated as replacement therapy in patients of all age groups in primary immunodeficiency syndromes (PID), and in myeloma or chronic lymphocytic leukaemia with severe secondary hypogammaglobulinaemia and recurrent infections (secondary immunodeficiency syndromes, SID), as well as hypogammaglobulinaemia and pre- and post-allogeneic hematopoietic stem cell transplantation.
HyQvia was investigated in one pivotal study lasting over a year, involving 89 patients with PID who had already had treatment with human normal IG for at least three months. The number of acute serious bacterial infections (SBI) as main efficacy outcomes was 0.025 per year. This was below the FDA predefined number needed to show efficacy (SBI rate <1.0 per subject per year at the 0.01 level of significance), and was similar to that seen with other licensed human normal IG products. In the pivotal study and its extension, 188 patient years under HyQvia treatment have been documented. HyQvia was efficacious, safe, and bioequivalent to intravenous IG at the same administration intervals, but it caused fewer systemic reactions. Tolerability was good despite high infusion volumes and rates. There are no preclinical data that suggest an increased risk of mutagenicity, teratogenicity, fertility or neuronal development.
The most current and comprehensive data on real-life utilisation and outcomes of various IgG preparations is available from the German SIGNS registry. This registry confirmed the effectiveness of IgG substitution or treatment in terms of reduction of infections (PID and SID), as well as stabilization or improvement of the clinical condition in neurological and autoimmune diseases. In a cohort of 24 PID and SID patients on HyQvia in the German SIGNS study, the preparation was well tolerated and treatment satisfaction was high. In other countries, data on the utilisation and outcomes of HyQvia under clinical practice conditions are sparse or completely missing.
The present study aims to fill these gaps and to provide a detailed and complete description of the utilisation of under everyday clinical practice conditions.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HyQvia | – | Biological: HyQviaRecombinant human hyaluronidase and normal immunoglobulin 10% |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with primary (PID) or secondary immunodeficiencies (SID)
Key inclusion criteria (4 listed)
- Patient has received/will receive at least 1 HyQvia infusion for PID or SID
- Patient has an indication for chronic immunoglobulin treatment
- Patient is likely available for long-term documentation
- Patient provides informed consent for documentation
Key exclusion criteria (1 listed)
- No explicit exclusion criteria apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HyQviaAlso: Recombinant human hyaluronidase and normal immunoglobulin 10% | Biological | HyQvia |
Primary outcome
Utilisation in terms of dose and dosing interval
Time frame: up to 3 years
mean monthly dose
7 secondary outcomes
Adverse events
Time frame: up to 3 years
Concomitant diseases
Time frame: up to 3 years
mean immunoglobulin trough level
Time frame: up to 3 years
Number of participants with treatment-related adverse events
Time frame: up to 3 years
Number of training session
Time frame: up to 3 years
Number of days in hospital
Time frame: up to 3 years
Number of days in rehabilitation clinic
Time frame: up to 3 years
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 14 Sep 2023.
- Results publication: PubMed 37036560Borte M, Hanitsch LG, Mahlaoui N, Fasshauer M, Huscher D, Speletas M, Dimou M, Kamieniak M, Hermann C, Pittrow D, Milito C. Facilitated Subcutaneous Immunoglobulin Treatment in Patients with Immunodeficiencies: the FIGARO Study. J Clin Immunol. 2023 Aug;43(6):1259-1271. doi: 10.1007/s10875-023-01470-2. Epub 2023 Apr 10.
- Individual participant dataSharing planned: No
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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.
Michael Borte, MD, PhD principal investigator
Fachbereich Pädiatrische Rheumatologie, Immunologie und Infektiologie am Klinikum St. Georg Leipzig
David Pittrow, MD study director
GWT-TUD GmbH, Dresden, Germany
Isabelle Quinti, MD study chair
Sapienza University Rome, Italy
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Immunodeficiency and Secondary Immune Deficiency, run by GWT-TUD GmbH at 4 sites in 3 countries.
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 4 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 14 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Germany, France, Italy. 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?
Utilisation in terms of dose and dosing interval (time frame: up to 3 years). 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 NCT03054181, which also lists 1 linked publication.
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 14 Sep 2023.