Immune Thrombocytopeniasic-regNCT03576742
Severe Immune Cytopenia Registry Www.Sic-reg.Org
An observational study of Immune Thrombocytopenia and Autoimmune Hemolytic Anemia, run by Medical University of Graz at 1 site in Austria.
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. Read more about this study
- Who is it for?
- Ages 6 months to 25 years
- Conditions the study is for
- Immune Thrombocytopenia, Autoimmune Hemolytic Anemia, Evans Syndrome
- What is being tested?
- potential biomarkers
- How many people
- 80 participants
- Where
- 1 siteAustria
- Study dates
- 9 Mar 2018to 31 Dec 2024
- How long will I take part?
- 4 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: consecutive registration of pediatric, adolescent, and young adult patients with severe immune cytopenia who consent
Who may be able to join
- Autoimmune hemolytic anemia (AIHA)
- Evans syndrome (ES)
- Persistent or chronic immune thrombocytopenia (ITP; >6 months after first manifestation)
Who cannot join
- (history of) malignancies
- (history of) hematopoietic stem cell transplantation
Trial locations and contacts
1 site in 1 country.
Austria1 site
Pediatric Hematology-Oncology Outpatient Clinic
Graz, Styria, 8036
About this study
Official title: Register für Schwere Immunzytopenien - Severe Immune Cytopenia Registry (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.
Read the 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).
- Conditions
- Immune Thrombocytopenia, Autoimmune Hemolytic Anemia, Evans Syndrome, Primary Immunodeficiency
- Being tested
- potential biomarkers
- Keywords
- pancytopenia, aplastic anemia, myelodysplastic syndrome, primary immunodeficiency, immune dysregulation, autoimmune cytopenia
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Anemia, Hemolytic, Autoimmune, Evans Syndrome, Primary Immunodeficiency Diseases, Pancytopenia, Anemia, Aplastic, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
potential biomarkersDiagnostic test
facs analysis, microbiome analysis
| Study group | Role | What they receive |
|---|---|---|
| patients | all who fulfil inclusion criteria and consent to participation; potential biomarkers will be documented Receives: Diagnostic Test: potential biomarkers |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 0-4 years
Secondary outcomes (5)
Clinical course
Documentation of physician-reported clinical symptoms including outcome measures/performance scores
Measured: 0-4 years
Biomarkers - Blood
Identification of novel biomarkers by flow cytometry of leukocytes
Measured: 0-4 years
Biomarkers - Stool
Identification of potential modifiers of immune tolerance by studying the intestinal microbiome
Measured: 0-4 years
Routine laboratory parameters
documentation of laboratory parameters that are routinely assessed for immune cytopenia
Measured: 0-4 years
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
Measured: 0-4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jul 2018
- Study start9 Mar 2018
- Primary completion31 Dec 2023
- Study completion31 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Medical University of GrazThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 80 participants (actual)
- Target duration
- 4 Years
Sponsor and investigators
- Lead sponsor
- Medical University of Graz
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Seidel, Principal investigator, Medical University of Graz
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03576742
- Sponsor's protocol ID
- 30-155 ex 17/18
- Acronym
- sic-reg
- Status last verified
- Oct 2025
- First submitted
- 8 Jun 2018
- Last updated
- 21 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 21 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Register für Schwere Immunzytopenien - Severe Immune Cytopenia Registry (SIC-Reg.Org)
- ClinicalTrials.gov ID
- NCT03576742
- Acronym / sponsor ID
- sic-reg / 30-155 ex 17/18
- Conditions
- Immune Thrombocytopenia, Autoimmune Hemolytic Anemia, Evans Syndrome, Primary Immunodeficiency
- Keywords
- pancytopenia, aplastic anemia, myelodysplastic syndrome, primary immunodeficiency, immune dysregulation, autoimmune cytopenia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 80 participants (actual)
- Lead sponsor
- Medical University of GrazUniversity, hospital or other
- Study start
- 9 Mar 2018 (actual)
- Primary completion
- 31 Dec 2023 (actual)
- Study completion
- 31 Dec 2024 (actual)
- First posted
- 3 Jul 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 4 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| patients | – | Diagnostic Test: potential biomarkersall who fulfil inclusion criteria and consent to participation; potential biomarkers will be documented |
Who may qualify
- Ages
- 6 months to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- consecutive registration of pediatric, adolescent, and young adult patients with severe immune cytopenia who consent
Key inclusion criteria (3 listed)
- Autoimmune hemolytic anemia (AIHA)
- Evans syndrome (ES)
- Persistent or chronic immune thrombocytopenia (ITP; >6 months after first manifestation)
Key exclusion criteria (2 listed)
- (history of) malignancies
- (history of) hematopoietic stem cell transplantation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| potential biomarkersfacs analysis, microbiome analysis | Diagnostic test | patients |
Primary outcome
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
5 secondary outcomes
Clinical course
Time frame: 0-4 years
Biomarkers - Blood
Time frame: 0-4 years
Biomarkers - Stool
Time frame: 0-4 years
Routine laboratory parameters
Time frame: 0-4 years
Number of participants with the diagnosis of severe immune cytopenia per participating centre and per year
Time frame: 0-4 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Nov 2025.
- Individual participant dataSharing planned: Undecided
Related trials for Immune Thrombocytopenia
RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Seidel principal investigator
Medical University of Graz
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Immune Thrombocytopenia and Autoimmune Hemolytic Anemia, run by Medical University of Graz at 1 site in Austria.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Austria. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
underlying disease that causes or is associated with severe immune cytopenia (time frame: 0-4 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Nov 2025.