Clinical trial NCT01287689: Assessment of Immunoglobulins (IgG) in a Long-term Non-interventional Study
Official title: An Open, Uncontrolled, Non-interventional Observational Cohort Outcome Study of Immunoglobulins in 3 Indications: Primary and Secondary Immunodeficiencies and Neurological Auto-immune Diseases
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Primary Immunodeficiency (PID)
Secondary Immunodeficiency (SID)
Neurological Autoimmune Disease
Keywords: 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
Lead sponsor: Technische Universität Dresden
Sponsor class: OTHER
Collaborators: name: GWT-TUD GmbH
First posted: 2011-02-01
Enrollment: 685
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Ambulatory or hospital-based patients (no age restriction)

Brief summary: 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: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Immunoglobulin IgG dosage
timeFrame: up to 54 months
Secondary outcomes:
measure: Infection rate
timeFrame: up to 54 months
measure: Neurological and muscular function (for neurological auto-immune diseases only)
timeFrame: up to 54 months
measure: Duration of manifest auto-immune disease within the follow-up period(for neurological auto-immune diseases only).
timeFrame: up to 54 months
measure: Health-related quality of life
timeFrame: up to 54 months
measure: Pharmacoeconomic parameters
timeFrame: up to 54 months
Other outcomes:
Locations:
city: Bochum
country: Germany
facility: Klinik für Neurologie, St. Josefs-Hospital der Ruhr-Univ.
city: Dresden
country: Germany
facility: Institute for Clinical Pharmacology
city: Hannover
country: Germany
facility: Klinik für Neurologie, Medizinische Hochschule
city: Hannover
country: Germany
facility: Klinik für Pädiatrische Pneumologie, Allergologie und Neonatologie, Medizinische Hochschule (MHH).
city: Köln
country: Germany
facility: Praxis für Hämatologie und Internistische Onkologie
city: Leipzig
country: Germany
facility: Fachbereich Pädiatrische Rheumatologie, Immunologie und Infektiologie am Klinikum St. Georg gGmbH Leipzig, Akademisches Lehrkrankenhaus der Universität
city: Mannheim
country: Germany
facility: Mannheimer Onkologie-Praxis
Study officials:
affiliation: Institute for Clinical Pharmacology, Medical Faculty, Technical University Dresden, Germany
name: Wilhelm Kirch, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Institute for Clinical Pharmacology, Medical Faculty, Technical University, Dresden, Germany
name: David Pittrow, MD, PhD
role: STUDY_CHAIR
affiliation: Fachbereich Pädiatrische Rheumatologie, Immunologie und Infektiologie am Klinikum St. Georg gGmbH Leipzig, Akademisches Lehrkrankenhaus der Universität Leipzig, Germany
name: Michael Borte, MD, PhD
role: STUDY_DIRECTOR
affiliation: Klinik für Pädiatrische Pneumologie, Allergologie und Neonatologie, Medizinische Hochschule Hannover (MHH), Germany
name: Ulrich Baumann, MD, PhD
role: STUDY_DIRECTOR
affiliation: Mannheimer Onkologie Praxis, Mannheim, Germany
name: Manfred Hensel, MD, PhD
role: STUDY_DIRECTOR
affiliation: Epidemiologie, Rheumaforschungszentrum Berlin, Germany
name: Dörte Huscher
role: STUDY_DIRECTOR
affiliation: PIOH - Praxis Internistische Onkologie, Hämatologie, Köln, Germany
name: Marcel Reiser, MD, PhD
role: STUDY_DIRECTOR
affiliation: Klinik für Neurologie, Medizinische Hochschule Hannover (MHH), Germany
name: Martin Stangel, MD, PhD
role: STUDY_DIRECTOR
affiliation: Klinik für Neurologie, St. Josef-Hospital, Klinikum der Ruhr-Universität Bochum, Germany
name: Ralph Gold, MD, PhD
role: STUDY_DIRECTOR
affiliation: Neurologische Klinik und Poliklinik, Universitätsklinik Würzburg, Germany
name: Claudia Sommer, MD, PhD
role: STUDY_DIRECTOR
References:
citation: Kirch W, Gold R, Hensel M, Fasshauer M, Pittrow D, Huscher D, Reiser M, Stangel M, Baumann U, Borte M. [Assessment of immunoglobulins in a long-term non-interventional study (SIGNS Study). Rationale, design, and methods]. Med Klin (Munich). 2010 Sep;105(9):647-51. doi: 10.1007/s00063-010-1105-8. Epub 2010 Sep 28. German.
citation: Borte M, Baumann U, Pittrow D, Hensel M, Fasshauer M, Huscher D, Reiser M, Stangel M, Gold R, Kirch W; Liste der aktuell beitragenden Zentren, sortiert nach Postleitzahlen (mindestens ein Patient zum 1.3.2012). [Immunoglobulins in PID, SID and neurological autoimmune disease]. Dtsch Med Wochenschr. 2012 Mar;137(13):675-80. doi: 10.1055/s-0032-1304844. Epub 2012 Mar 20. German.
citation: Stangel M, Baumann U, Borte M, Fasshauer M, Hensel M, Huscher D, Kirch W, Pittrow D, Reiser M, Gold R. Treatment of neurological autoimmune diseases with immunoglobulins: first insights from the prospective SIGNS registry. J Clin Immunol. 2013 Jan;33 Suppl 1:S67-71. doi: 10.1007/s10875-012-9789-6. Epub 2012 Sep 14.
citation: Stangel M, Gold R, Pittrow D, Baumann U, Borte M, Fasshauer M, Hensel M, Huscher D, Reiser M, Sommer C. Treatment of patients with multifocal motor neuropathy with immunoglobulins in clinical practice: the SIGNS registry. Ther Adv Neurol Disord. 2016 May;9(3):165-79. doi: 10.1177/1756285616629869. Epub 2016 Feb 16.
citation: Reiser M, Borte M, Huscher D, Baumann U, Pittrow D, Sommer C, Stangel M, Fasshauer M, Gold R, Hensel M. Management of patients with malignancies and secondary immunodeficiencies treated with immunoglobulins in clinical practice: Long-term data of the SIGNS study. Eur J Haematol. 2017 Aug;99(2):169-177. doi: 10.1111/ejh.12900. Epub 2017 Jun 21.