Assessment of Immunoglobulins (IgG) in a Long-term Non-interventional Study

Study Overview
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Brief Summary
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This non-interventional, epidemiological study assesses long-term outcomes in subjects receiving immunoglobulins (IgG) for any treatment purpose, irrespective of the regimen prescribed by the treating physician, under routine clinical conditions in Germany. Long-term outcome data are collected on patient characteristics in the various indications, drug utilization of intravenous and subcutaneous IgG (e.g. treatment and dosing patterns), effectiveness (i.e. number of infections), tolerability, health related quality of life, and economic variables (number of hospitalizations, sick-leave days etc.) with the possibility to estimate direct costs.

Detailed Description
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In view of the broad range of indications in immunodeficiency and immunomodulation, it is of interest to document the use of IgG under the conditions of everyday practice and to analyze the endpoints (outcomes). A prospective cohort study such as this is an important evidence source for such rare diseases as those mentioned above. The aim of this outcome study is to fill the gap of the lack of long-term data in these rare diseases treated with IgG.

Official Title
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An Open, Uncontrolled, Non-interventional Observational Cohort Outcome Study of Immunoglobulins in 3 Indications: Primary and Secondary Immunodeficiencies and Neurological Auto-immune Diseases

Conditions
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Primary Immunodeficiency (PID), Secondary Immunodeficiency (SID), Neurological Autoimmune Disease

Intervention / Treatment
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* Other: Immunoglobulin G (IgG)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects of either gender with primary, severe secondary immunodeficiency and recurrent infections or neurological autoimmune diseases Naïve to IgG, or pre-treated with IgG Subject or parent/legally authorized representative has provided written informed consent. Exclusion Criteria: None
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 |
| --- | --- |
| Patient treated with any IgG<br>Any marketed SC or IV IgG can be documented | Other: Immunoglobulin G (IgG)<br>* Not applicable. All interventions are at the discretion of the investigator. All marketed IgG formulations can be documented.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Immunoglobulin IgG dosage | Dosage of immunoglobulins (IgG); frequency of IgG administrations; days of treatment with IgG; duration of infusion of IgG. | up to 54 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Infection rate | For immunodeficiencies (primary PID and secondary SID): frequency of infections; degree of severity of infections (SBIs); duration of antibiotic treatment; necessity of antibiotic treatment. | up to 54 months | 
| Neurological and muscular function (for neurological auto-immune diseases only) | Grip strength (dynamometer) Electrophysiology (EMG, ENG); Inflammatory Neuropathy Cause and Treatment (INCAT) disability score; EDSS, annual relapse rate; Myasthenia Score. | up to 54 months | 
| Duration of manifest auto-immune disease within the follow-up period(for neurological auto-immune diseases only). |  | up to 54 months | 
| Health-related quality of life |  | up to 54 months | 
| Pharmacoeconomic parameters | Number of sick-leave days Number of medical visits Days of hospitalisation due to infections or due to disability or loss of function Degree of disability | up to 54 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Non-interventional trial, immunodeficiency, outcome study, registry, long-term outcomes, drug utilization, effectiveness, treatment patterns, patient characteristics under real life conditions, factors for treatment success, long-term tolerability, quality of life