Severe Immune Cytopenia Registry Www.Sic-reg.Org | NCT03576742

Studies
Severe Immune Cytopenia Registry Www.Sic-reg.Org

NCT03576742

Sponsor

Medical University of Graz

Status

Completed

Last Update Posted

Nov 21, 2025Actual

Enrollment

80Actual

Phase

Not provided

Conditions

Immune Thrombocytopenia
Autoimmune Hemolytic Anemia
Evans Syndrome
Primary Immunodeficiency

Interventions

potential biomarkers

Countries

Austria

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Oct 2025

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Mar 9, 2018Actual

Primary Completion Date

Dec 31, 2023Actual

Completion Date

Dec 31, 2024Actual

First Submitted Date

Jun 8, 2018

First Submission Date that Met QC Criteria

Jun 21, 2018

First Posted Date

Jul 3, 2018Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Nov 18, 2025

Last Update Posted Date

Nov 21, 2025Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

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).

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Yes

Target Follow-Up Duration

4 Years

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

Retention

Enrollment

80Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
patients		all who fulfil inclusion criteria and consent to participation; potential biomarkers will be documented	Diagnostic Test: potential biomarkers

Interventions

Name	Type	Description	Arm Group Labels	Other Names
potential biomarkers	Diagnostic Test	facs analysis, microbiome analysis	patients

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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 Outcomes

Measure	Description	Time Frame
Clinical course	Documentation of physician-reported clinical symptoms including outcome measures/performance scores	0-4 years
Biomarkers - Blood	Identification of novel biomarkers by flow cytometry of leukocytes

Other Outcomes

Not provided

Eligibility Module

Eligibility 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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Months

Maximum Age

25 Years

Standard Ages

ChildAdult

Study Population

consecutive registration of pediatric, adolescent, and young adult patients with severe immune cytopenia who consent

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Seidel	Medical University of Graz	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Pediatric Hematology-Oncology Outpatient Clinic		Graz	Styria	8036	Austria

IPD Sharing Statement Module

Plan to Share IPD

Undecided

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D016553	Purpura, Thrombocytopenic, Idiopathic
D000744	Anemia, Hemolytic, Autoimmune
C536380	Evans Syndrome
D000081207	Primary Immunodeficiency Diseases
D010198	Pancytopenia
D000741	Anemia, Aplastic
D009190	Myelodysplastic Syndromes

Ancestor Terms

ID	Term
D011696	Purpura, Thrombocytopenic
D011693	Purpura
D001778	Blood Coagulation Disorders
D006402	Hematologic Diseases

Browse Leaves

Not provided

Browse Branches

Not provided