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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT03576742
Other study ID # 30-155 ex 17/18
Secondary ID
Status Active, not recruiting
Phase
First received
Last updated
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
Health authority
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

Study Design


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
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