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NCT Number: NCT03576742

Severe Immune Cytopenia Registry Www.Sic-reg.Org

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.

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Key information

Conditions

Immune Thrombocytopenia Anemia Anemia, Aplastic Anemia, Hemolytic Anemia, Hemolytic, Autoimmune Autoimmune Diseases Autoimmune Hemolytic Anemia Blood Coagulation Disorders Blood Platelet Disorders Bone Marrow Diseases Bone Marrow Failure Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cytopenia Evans Syndrome Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Immune System Diseases Immunologic Deficiency Syndromes Myelodysplastic Syndromes Pancytopenia Pathologic Processes Pathological Conditions, Signs and Symptoms Primary Immunodeficiency Primary Immunodeficiency Diseases Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombotic Microangiopathies

Age range

6 month–25 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Pediatric Hematology-Oncology Outpatient Clinic

Graz, Styria, 8036, Austria

About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

potential biomarkers

Diagnostic Test

facs analysis, microbiome analysis

Primary outcomes

  1. 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

Secondary outcomes

  1. Clinical course

    Time frame: 0-4 years

    Documentation of physician-reported clinical symptoms including outcome measures/performance scores

  2. Biomarkers - Blood

    Time frame: 0-4 years

    Identification of novel biomarkers by flow cytometry of leukocytes

  3. Biomarkers - Stool

    Time frame: 0-4 years

    Identification of potential modifiers of immune tolerance by studying the intestinal microbiome

  4. Routine laboratory parameters

    Time frame: 0-4 years

    documentation of laboratory parameters that are routinely assessed for immune cytopenia

  5. 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

Sponsors and collaborators

Lead sponsor

Medical University of Graz

Other

Registry information

Official study title

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

Acronym: sic-reg

Important dates

Study start
2018
Primary completion
2023
Study completion
2024
First posted
Jul 3, 2018
Registry last updated
Nov 21, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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