Facilitated Immunoglobulin Administration Registry and Outcomes Study (FIGARO)

Study Overview
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Brief Summary
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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
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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.

Official Title
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Facilitated Immunoglobulin Administration Registry and Outcomes Study

Conditions
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Primary Immunodeficiency, Secondary Immune Deficiency

Intervention / Treatment
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* Biological: HyQvia


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: No explicit exclusion criteria apply.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| HyQvia<br>Recombinant human hyaluronidase and normal immunoglobulin 10% | Biological: HyQvia<br> <br> * Other names: Recombinant human hyaluronidase and normal immunoglobulin 10%;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Utilisation in terms of dose and dosing interval | mean monthly dose | up to 3 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse events | Incidence and type | up to 3 years | 
| Concomitant diseases |  | up to 3 years | 
| mean immunoglobulin trough level | after HyQvia infusion | up to 3 years | 
| Number of participants with treatment-related adverse events | Acute bacterial infections; overall infections | up to 3 years | 
| Number of training session | about appropriate self-infusion | up to 3 years | 
| Number of days in hospital |  | up to 3 years | 
| Number of days in rehabilitation clinic |  | up to 3 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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chronic, Observational, Infection, Utilisation, Safety, Tolerability, Infusion