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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05931718
Other study ID # CYTOPAN
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date June 1, 2021
Est. completion date June 2030

Study information

Verified date May 2023
Source Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

The goal of this observational study is to characterize the diagnostic and therapeutic management of autoimmune cytopenias including autoimmune hemolytic anemia, immune thrombocytopenia, and chronic idiopathic/autoimmune neutropenia. The main aims to answer are: - evaluation of traditional and novel diagnostic tools including immunohematology, cytokine essays, bone marrow studies, molecular findings, and fecal microbiome. - evaluation of type and sequence of the therapies administered, the response rates, and the adverse events. - evaluation of clinical and laboratory (immunologic, molecular, and morphologic) predictors of outcome. - evolution of autoimmune cytopenias into myelodysplastic syndromes. - a subgroup of patients with myelodysplastic syndromes will be included to evaluate the presence of immunologic events, autoimmune activation, and red cell metabolism. Participants will receive a clinical/laboratory diagnostic workup as per current clinical practice. Furthermore They will be sampled at baseline (peripheral blood and feces for microbiome) and followed up for at least 3 years to evaluate their clinical course, therapeutic management and outcome.


Description:

This observational study will characterize the diagnostic and therapeutic management of autoimmune cytopenias including autoimmune hemolytic anemia, immune thrombocytopenia, and chronic idiopathic/autoimmune neutropenia to evaluate predictors of outcome. Additionally, a subgroup of patients with myelodysplastic syndromes (diagnosed according to current WHO 5th edition 2022) will be included to evaluate the presence of autoimmune activation, and red cell metabolism.


Recruitment information / eligibility

Status Recruiting
Enrollment 200
Est. completion date June 2030
Est. primary completion date September 2026
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria: - Diagnosis of autoimmune cytopenias (AIHA/ITP/CIN/AIN) - age >/= 18 years - ability to sign informed consent - availability to undergo 3 year follow up - for the subgroup of patients with myelodysplastic syndrome: bone marrow evaluation showing >/= 10% dysplastic features of at least one lineage along with MDS defining cytopenia and/or MDS defining cytogenetics. Exclusion Criteria: - any condition impeding the acquisition of the informed consent - immune cytopenia diagnosis preceding >/= 6 months the enrolment

Study Design


Intervention

Biological:
cytokine essays
evaluation of immunomodulatory cytokines by ELISA kits on peripheral blood samples
NGS
evaluation of somatic mutations commonly associated with myeloid neoplasm and immunodeficiencies by next generation sequencing on peripheral blood samples
Fecal microbiome
evaluation of fecal microbiome on fecal samples
Drug:
Erythropoietin
evaluation of recombinant erythropoietin use, safety and efficacy in patients with autoimmune hemolytic anemia according to clinical practice
Luspatercept
evaluation of cytokine levels, molecular profile and bone marrow microenvironment by single cell analysis in patients treated with luspatercept according to clinical practice
Thrombopoietin Receptor Agonist
evaluation of TPO-RA use, safety and efficacy in patients with ITP according to clinical practice
G-CSF
evaluation of G-CSF use, safety and efficacy in patients with CIN/AIN according to clinical practice

Locations

Country Name City State
Italy Fondazione Irccs Ca' Granda Ospedale Maggiore Policlinico Milano

Sponsors (1)

Lead Sponsor Collaborator
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Country where clinical trial is conducted

Italy, 

Outcome

Type Measure Description Time frame Safety issue
Primary sensitivity of autoantibody testing in autoimmune cytopenias to define the sensitivity of autoantibody testing in autoimmune cytopenias 2021-2026
Primary specificity of autoantibody testing in autoimmune cytopenias to define the specificity of autoantibody testing in autoimmune cytopenias 2021-2026
Primary sensitivity of bone marrow trephine in autoimmune cytopenias to define the sensitivity of bone marrow trephine in autoimmune cytopenias 2021-2026
Secondary overall response rate to define response rates to treatment of autoimmune cytopenias and myelodysplastic syndromes 2021-2026
Secondary Evaluation of somatic mutations to define somatic mutations in autoimmune cytopenias and myelodysplastic syndromes by NGS 2021-2026
Secondary Evaluation of pyruvate kinase activity to define pyruvate kinase activity in myelodysplastic syndromes 2021-2026
Secondary Evaluation of microbiome to define microbiome composition in autoimmune cytopenias and myelodysplastic syndromes 2021-2026
Secondary Single cell RNA expression to define bone marrow composition by single cell analysis in autoimmune cytopenias and myelodysplastic syndromes 2021-2026
Secondary Evaluation of cytokine levels to define cytokine levels in autoimmune cytopenias and myelodysplastic syndromes by ELISA 2021-2026
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