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Severe Immune Cytopenia Registry Www.Sic-reg.Org (sic-reg)

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ClinicalTrials.gov Identifier: NCT03576742
Recruitment Status : Recruiting
First Posted : July 3, 2018
Last Update Posted : July 3, 2018
Sponsor:
Information provided by (Responsible Party):
Medical University of Graz

Brief 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.


Condition or disease Intervention/treatment
Immune Thrombocytopenia Autoimmune Hemolytic Anemia Evan Syndrome Primary Immunodeficiency Diagnostic Test: potential biomarkers

Detailed 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).


Study Type : Observational [Patient Registry]
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Prospective
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


Group/Cohort Intervention/treatment
patients
all who fulfil inclusion criteria and consent to participation; potential biomarkers will be documented
Diagnostic Test: potential biomarkers
facs analysis, microbiome analysis




Primary Outcome Measures :
  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 Outcome Measures :
  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


Biospecimen Retention:   Samples Without DNA
stool, peripheral blood mononuclear cells


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
consecutive registration of pediatric, adolescent, and young adult patients with severe immune cytopenia who consent
Criteria

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

Information from the National Library of Medicine

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


Contacts
Contact: Seidel +43316385 ext 12621 markus.seidel@medunigraz.at
Contact: Karastaneva +43316385 ext 12621

Locations
Austria
Pediatric Hematology-Oncology Outpatient Clinic Recruiting
Graz, Styria, Austria, 8036
Contact: Seidel    +43316385 ext 12621      
Sponsors and Collaborators
Medical University of Graz
Investigators
Principal Investigator: Seidel Medical University of Graz

Additional Information:
Responsible Party: Medical University of Graz
ClinicalTrials.gov Identifier: NCT03576742     History of Changes
Other Study ID Numbers: 30-155 ex 17/18
First Posted: July 3, 2018    Key Record Dates
Last Update Posted: July 3, 2018
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Medical University of Graz:
pancytopenia
aplastic anemia
myelodysplastic syndrome
primary immunodeficiency
immune dysregulation
autoimmune cytopenia

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Immune System Diseases
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Anemia