Severe Immune Cytopenia Registry Www.Sic-reg.Org (sic-reg)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03576742|
Recruitment Status : Recruiting
First Posted : July 3, 2018
Last Update Posted : September 6, 2019
Prospective registry study for children and young adults with severe immune cytopenias (persisting/chronic immune thrombocytopenia, autoimmune hemolytic anemia, and Evans syndrome) to improve the management, facilitate the differential diagnostic work-up, and document the clinical course under various treatments.
Time points: at inclusion, after 6 months, after 12 months, then yearly up to 4 years after inclusion.
No intervention, mere observation and documentation. Guided pre-inclusion (differential) diagnostic work-up.
|Condition or disease||Intervention/treatment|
|Immune Thrombocytopenia Autoimmune Hemolytic Anemia Evan Syndrome Primary Immunodeficiency||Diagnostic Test: potential biomarkers|
The study aims to improve the management and care of patients with severe immune cytopenias, to identify underlying causes of severe immune cytopenias and to develop a strategy for early treatment stratification based on a standardized diagnostic algorithm, potentially supported by biomarker analyses and (off study) genetic analyses, where clinically indicated.
Rapid detection of underlying causes of severe immune cytopenias with the aid of a structured diagnostic approach and access to a clinical care network of the participating centers, allowing early treatment stratification
- Collection of data about epidemiology of rare diseases
- Systemic documentation of response rates to various treatments
- Identification of biomarkers and modifiers of immune tolerance
- Collection of data about the usage of novel/experimental therapeutic agents
- Documentation of physician-reported outcome measures/performance scores
- Consultation of the caring physicians through a regular SIC-Reg board
There will be no additional venous punctures or investigational time points. At clinical visits around planned study time points, additional blood parameters and stool specimen will be obtained. The current clinical management follows international guidelines, which are summarized in the study documents but do not represent part of the study (no diagnostic or therapeutic investigational arm).
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||50 participants|
|Target Follow-Up Duration:||4 Years|
|Official Title:||Register für Schwere Immunzytopenien - Severe Immune Cytopenia Registry (SIC-Reg.Org)|
|Actual Study Start Date :||March 9, 2018|
|Estimated Primary Completion Date :||March 8, 2022|
|Estimated Study Completion Date :||March 8, 2022|
all who fulfil inclusion criteria and consent to participation; potential biomarkers will be documented
Diagnostic Test: potential biomarkers
facs analysis, microbiome analysis
- underlying disease that causes or is associated with severe immune cytopenia [ Time Frame: 0-4 years ]identify the underlying condition or other disease, e.g., primary immunodeficiency or bone marrow failure syndrome by diagnostic procedures according to a standardized algorithm
- Clinical course [ Time Frame: 0-4 years ]Documentation of physician-reported clinical symptoms including outcome measures/performance scores
- Biomarkers - Blood [ Time Frame: 0-4 years ]Identification of novel biomarkers by flow cytometry of leukocytes
- Biomarkers - Stool [ Time Frame: 0-4 years ]Identification of potential modifiers of immune tolerance by studying the intestinal microbiome
- Routine laboratory parameters [ Time Frame: 0-4 years ]documentation of laboratory parameters that are routinely assessed for immune cytopenia
- Number of participants with the diagnosis of severe immune cytopenia per participating centre and per year [ Time Frame: 0-4 years ]epidemiological data acquisition, participants included according to the inclusion criteria
Biospecimen Retention: Samples Without DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03576742
|Contact: Seidel||+43316385 ext firstname.lastname@example.org|
|Contact: Karastaneva||+43316385 ext 12621|
|Pediatric Hematology-Oncology Outpatient Clinic||Recruiting|
|Graz, Styria, Austria, 8036|
|Contact: Seidel +43316385 ext 12621|
|Principal Investigator:||Seidel||Medical University of Graz|