Primary Immunodeficiency (PID)SIGNSNCT01287689
Assessment of Immunoglobulins (IgG) in a Long-term Non-interventional Study
An observational study of Primary Immunodeficiency (PID) and Secondary Immunodeficiency (SID), run by Technische Universität Dresden at 7 sites in Germany.
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Primary Immunodeficiency (PID), Secondary Immunodeficiency (SID), Neurological Autoimmune Disease
- What is being tested?
- Immunoglobulin G (IgG)
- Type of treatment
- Injection or infusion
- How many people
- 685 participants
- Where
- 7 sitesGermany
- Study dates
- Jul 2010to 31 Dec 2016
- How long will I take part?
- 5 Years
- 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: Ambulatory or hospital-based patients (no age restriction)
Who may be able to join
- 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.
Who cannot join
- None
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
Germany7 sites
Klinik für Neurologie, St. Josefs-Hospital der Ruhr-Univ.
Bochum
Praxis für Hämatologie und Internistische Onkologie
Cologne
Institute for Clinical Pharmacology
Dresden, D-01307
Klinik für Neurologie, Medizinische Hochschule
Hanover
Klinik für Pädiatrische Pneumologie, Allergologie und Neonatologie, Medizinische Hochschule (MHH).
Hanover
Fachbereich Pädiatrische Rheumatologie, Immunologie und Infektiologie am Klinikum St. Georg gGmbH Leipzig, Akademisches Lehrkrankenhaus der Universität
Leipzig
Mannheimer Onkologie-Praxis
Mannheim
About this 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
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.
Read the 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.
- Conditions
- Primary Immunodeficiency (PID), Secondary Immunodeficiency (SID), Neurological Autoimmune Disease
- Being tested
- Immunoglobulin G (IgG)
- 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
- Medical subject headings
- Primary Immunodeficiency Diseases, Immunologic Deficiency Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Immunoglobulin G (IgG)Other
Not applicable. All interventions are at the discretion of the investigator. All marketed IgG formulations can be documented.
| Study group | Role | What they receive |
|---|---|---|
| Patient treated with any IgG | Any marketed SC or IV IgG can be documented Receives: Other: Immunoglobulin G (IgG) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Immunoglobulin IgG dosage
Dosage of immunoglobulins (IgG); frequency of IgG administrations; days of treatment with IgG; duration of infusion of IgG.
Measured: up to 54 months
Secondary outcomes (5)
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.
Measured: 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.
Measured: up to 54 months
Duration of manifest auto-immune disease within the follow-up period(for neurological auto-immune diseases only).
Measured: up to 54 months
Health-related quality of life
Measured: 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
Measured: up to 54 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Feb 2011 (estimated)
- Study startJul 2010
- Primary completion31 Dec 2016
- Study completion31 Dec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Primary Immunodeficiency (PID)Studies of the same condition, open and under way.
- More trials run by Technische Universität DresdenThe sponsor's other registered studies.
- 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
- 685 participants (actual)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Technische Universität Dresden
- Sponsor type
- University, hospital or other
- Collaborators
- GWT-TUD GmbH
- Responsible party
- Sponsor
- Investigators
- Wilhelm Kirch, MD, PhD, Principal investigator, Institute for Clinical Pharmacology, Medical Faculty, Technical University Dresden, GermanyDavid Pittrow, MD, PhD, Study chair, Institute for Clinical Pharmacology, Medical Faculty, Technical University, Dresden, GermanyMichael Borte, MD, PhD, Study director, Fachbereich Pädiatrische Rheumatologie, Immunologie und Infektiologie am Klinikum St. Georg gGmbH Leipzig, Akademisches Lehrkrankenhaus der Universität Leipzig, GermanyUlrich Baumann, MD, PhD, Study director, Klinik für Pädiatrische Pneumologie, Allergologie und Neonatologie, Medizinische Hochschule Hannover (MHH), GermanyManfred Hensel, MD, PhD, Study director, Mannheimer Onkologie Praxis, Mannheim, GermanyDörte Huscher, Study director, Epidemiologie, Rheumaforschungszentrum Berlin, GermanyMarcel Reiser, MD, PhD, Study director, PIOH - Praxis Internistische Onkologie, Hämatologie, Köln, GermanyMartin Stangel, MD, PhD, Study director, Klinik für Neurologie, Medizinische Hochschule Hannover (MHH), Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01287689
- Sponsor's protocol ID
- SIGNS
- Acronym
- SIGNS
- Status last verified
- Jul 2017
- First submitted
- 24 Jan 2011
- Last updated
- 1 Aug 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 20878302Borte 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.
PubMed 22434180Stangel 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.
PubMed 22976553Stangel 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.
PubMed 27134672Reiser 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.
PubMed 28467615
Study at a glance
From the registry record, last updated by the sponsor on 1 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01287689
- Acronym / sponsor ID
- SIGNS / SIGNS
- 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
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 685 participants (actual)
- Lead sponsor
- Technische Universität DresdenUniversity, hospital or other
- Collaborators
- GWT-TUD GmbH
- Study start
- Jul 2010 (actual)
- Primary completion
- 31 Dec 2016 (actual)
- Study completion
- 31 Dec 2016 (actual)
- First posted
- 1 Feb 2011 (estimated)
- 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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patient treated with any IgG | – | Other: Immunoglobulin G (IgG)Any marketed SC or IV IgG can be documented |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Ambulatory or hospital-based patients (no age restriction)
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (1 listed)
- None
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 |
|---|---|---|
| Immunoglobulin G (IgG)Not applicable. All interventions are at the discretion of the investigator. All marketed IgG formulations can be documented. | Other | Patient treated with any IgG |
Primary outcome
Immunoglobulin IgG dosage
Time frame: up to 54 months
Dosage of immunoglobulins (IgG); frequency of IgG administrations; days of treatment with IgG; duration of infusion of IgG.
5 secondary outcomes
Infection rate
Time frame: up to 54 months
Neurological and muscular function (for neurological auto-immune diseases only)
Time frame: up to 54 months
Duration of manifest auto-immune disease within the follow-up period(for neurological auto-immune diseases only).
Time frame: up to 54 months
Health-related quality of life
Time frame: up to 54 months
Pharmacoeconomic parameters
Time frame: up to 54 months
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 1 Aug 2017.
- Background: PubMed 20878302Kirch 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.
- Results publication: PubMed 22434180Borte 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.
- Results publication: PubMed 22976553Stangel 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.
- Results publication: PubMed 27134672Stangel 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.
- Results publication: PubMed 28467615Reiser 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.
Related trials for Primary Immunodeficiency (PID)
RecruitingObservational study
Studies of Disorders With Increased Susceptibility to Fungal Infections
Fungal Infections, Primary Immune Deficiencies
RecruitingObservational study
COVID-19 in PID Survey
Primary Immune Deficiency, COVID
Not yet recruitingPhase 4
A Study of TAK-664 in People With Primary Immunodeficiency Diseases Who Have Not Yet Been Treated With Immunoglobulins
Primary Immunodeficiency Diseases (PID)
RecruitingObservational study
A Database Survey to Evaluate the Safety of Immune Globulin Subcutaneous (Human), 20% Solution in Participants With Primary Immunodeficiency
Primary Immunodeficiency Diseases (PID)
Active, not recruitingPhase 2
A Study to Evaluate Tabelecleucel in Participants With Epstein Barr Virus (EBV) Associated Diseases
Epstein-Barr Virus (EBV)-Associated Diseases, EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (EBV+ PID LPD)
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.
Wilhelm Kirch, MD, PhD principal investigator
Institute for Clinical Pharmacology, Medical Faculty, Technical University Dresden, Germany
David Pittrow, MD, PhD study chair
Institute for Clinical Pharmacology, Medical Faculty, Technical University, Dresden, Germany
Michael Borte, MD, PhD study director
Fachbereich Pädiatrische Rheumatologie, Immunologie und Infektiologie am Klinikum St. Georg gGmbH Leipzig, Akademisches Lehrkrankenhaus der Universität Leipzig, Germany
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Immunodeficiency (PID) and Secondary Immunodeficiency (SID), run by Technische Universität Dresden at 7 sites in Germany.
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 3 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 1 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Germany. 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?
Immunoglobulin IgG dosage (time frame: up to 54 months). 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 NCT01287689, which also lists 5 linked publications.
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 1 Aug 2017.