Severe Immune Cytopenia Registry Www.Sic-reg.Org

Immune Thrombocytopenia Clinical Trial

— sic-reg  

Official title:

Register für Schwere Immunzytopenien - Severe Immune Cytopenia Registry (SIC-Reg.Org)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03576742
• Other study ID #: 30-155 ex 17/18
• Status: Active, not recruiting
• Start date: March 9, 2018
• Est. completion date: December 31, 2024

Study information

• Verified date: January 2024
• Source: Medical University of Graz
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

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.

Description:

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.

Primary Goal:

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

Secondary Goals:

• 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).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 80
• Est. completion date: December 31, 2024
• Est. primary completion date: December 31, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 25 Years

Eligibility

Inclusion Criteria:

• Autoimmune hemolytic anemia (AIHA)

• Evans syndrome (ES)

• Persistent or chronic immune thrombocytopenia (ITP; >6 months after first manifestation)

Exclusion Criteria:

• (history of) malignancies

• (history of) hematopoietic stem cell transplantation

Related Conditions & MeSH terms

• Anemia, Hemolytic
• Anemia, Hemolytic, Autoimmune
• Autoimmune Hemolytic Anemia
• Evans Syndrome
• Immune Thrombocytopenia
• Immunologic Deficiency Syndromes
• Primary Immunodeficiency
• Primary Immunodeficiency Diseases
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Diagnostic Test:
• potential biomarkers
facs analysis, microbiome analysis

Locations

Country	Name	City	State
Austria	Pediatric Hematology-Oncology Outpatient Clinic	Graz	Styria

Sponsors (1)

Lead Sponsor	Collaborator
Medical University of Graz

Country where clinical trial is conducted

Austria, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	underlying disease that causes or is associated with severe immune cytopenia	identify the underlying condition or other disease, e.g., primary immunodeficiency or bone marrow failure syndrome by diagnostic procedures according to a standardized algorithm	0-4 years
Secondary	Clinical course	Documentation of physician-reported clinical symptoms including outcome measures/performance scores	0-4 years
Secondary	Biomarkers - Blood	Identification of novel biomarkers by flow cytometry of leukocytes	0-4 years
Secondary	Biomarkers - Stool	Identification of potential modifiers of immune tolerance by studying the intestinal microbiome	0-4 years
Secondary	Routine laboratory parameters	documentation of laboratory parameters that are routinely assessed for immune cytopenia	0-4 years
Secondary	Number of participants with the diagnosis of severe immune cytopenia per participating centre and per year	epidemiological data acquisition, participants included according to the inclusion criteria	0-4 years

External Links

•   Study website with information and contact details for participating or interested patients, parents, institutions, physicians