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

Primary Immunodeficiency (PID) Clinical Trial

— SIGNS  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01287689
• Other study ID #: SIGNS
• Status: Completed
• Phase: N/A
• First received: January 24, 2011
• Last updated: July 31, 2017
• Start date: July 2010
• Est. completion date: December 31, 2016

Study information

• Verified date: July 2017
• Source: Technische Universität Dresden
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 685
• Est. completion date: December 31, 2016
• Est. primary completion date: December 31, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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:

Related Conditions & MeSH terms

• Autoimmune Diseases
• Immunologic Deficiency Syndromes
• Neoplasm Metastasis
• Neurological Autoimmune Disease
• Primary Immunodeficiency (PID)
• Secondary Immunodeficiency (SID)

Intervention

Other:
• Immunoglobulin G (IgG)
Not applicable. All interventions are at the discretion of the investigator. All marketed IgG formulations can be documented.

Locations

Country	Name	City	State
Germany	Klinik für Neurologie, St. Josefs-Hospital der Ruhr-Univ.	Bochum
Germany	Institute for Clinical Pharmacology	Dresden
Germany	Klinik für Neurologie, Medizinische Hochschule	Hannover
Germany	Klinik für Pädiatrische Pneumologie, Allergologie und Neonatologie, Medizinische Hochschule (MHH).	Hannover
Germany	Praxis für Hämatologie und Internistische Onkologie	Köln
Germany	Fachbereich Pädiatrische Rheumatologie, Immunologie und Infektiologie am Klinikum St. Georg gGmbH Leipzig, Akademisches Lehrkrankenhaus der Universität	Leipzig
Germany	Mannheimer Onkologie-Praxis	Mannheim

Sponsors (2)

Lead Sponsor	Collaborator
Technische Universität Dresden	GWT-TUD GmbH

Country where clinical trial is conducted

Germany, 

References & Publications (5)

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 neurologica — View 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. — View 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 SIGN — View 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 Supp — View 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; — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	Duration of manifest auto-immune disease within the follow-up period(for neurological auto-immune diseases only).		up to 54 months
Secondary	Health-related quality of life		up to 54 months
Secondary	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