Ignite Creation Date:
2025-12-25 @ 4:35 AM
Ignite Modification Date:
2025-12-26 @ 3:37 AM
Study NCT ID:
NCT05931718
Status:
RECRUITING
Last Update Posted:
2024-10-15
First Post:
2023-05-29
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000744', 'term': 'Anemia, Hemolytic, Autoimmune'}, {'id': 'D016553', 'term': 'Purpura, Thrombocytopenic, Idiopathic'}, {'id': 'D009190', 'term': 'Myelodysplastic Syndromes'}], 'ancestors': [{'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D011696', 'term': 'Purpura, Thrombocytopenic'}, {'id': 'D011693', 'term': 'Purpura'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D057049', 'term': 'Thrombotic Microangiopathies'}, {'id': 'D013921', 'term': 'Thrombocytopenia'}, {'id': 'D001791', 'term': 'Blood Platelet Disorders'}, {'id': 'D000095542', 'term': 'Cytopenia'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D006470', 'term': 'Hemorrhage'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D012877', 'term': 'Skin Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000069467', 'term': 'Fecal Microbiota Transplantation'}, {'id': 'D004921', 'term': 'Erythropoietin'}, {'id': 'C000621232', 'term': 'luspatercept'}, {'id': 'D016179', 'term': 'Granulocyte Colony-Stimulating Factor'}], 'ancestors': [{'id': 'D001691', 'term': 'Biological Therapy'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D003115', 'term': 'Colony-Stimulating Factors'}, {'id': 'D006023', 'term': 'Glycoproteins'}, {'id': 'D006001', 'term': 'Glycoconjugates'}, {'id': 'D002241', 'term': 'Carbohydrates'}, {'id': 'D016298', 'term': 'Hematopoietic Cell Growth Factors'}, {'id': 'D016207', 'term': 'Cytokines'}, {'id': 'D036341', 'term': 'Intercellular Signaling Peptides and Proteins'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D001685', 'term': 'Biological Factors'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'peripheral blood for cytokines and next generation sequencing, bone marrow sample for single cell analysis, fecal sample for microbiome'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 200}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2019-06-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-10', 'completionDateStruct': {'date': '2035-06', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-10-08', 'studyFirstSubmitDate': '2023-05-29', 'studyFirstSubmitQcDate': '2023-07-03', 'lastUpdatePostDateStruct': {'date': '2024-10-15', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-07-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2030-09', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'sensitivity of autoantibody testing in autoimmune cytopenias', 'timeFrame': '2021-2026', 'description': 'to define the sensitivity of autoantibody testing in autoimmune cytopenias'}, {'measure': 'specificity of autoantibody testing in autoimmune cytopenias', 'timeFrame': '2021-2026', 'description': 'to define the specificity of autoantibody testing in autoimmune cytopenias'}, {'measure': 'sensitivity of bone marrow trephine in autoimmune cytopenias', 'timeFrame': '2021-2026', 'description': 'to define the sensitivity of bone marrow trephine in autoimmune cytopenias'}], 'secondaryOutcomes': [{'measure': 'overall response rate', 'timeFrame': '2021-2026', 'description': 'to define response rates to treatment of autoimmune cytopenias and myelodysplastic syndromes'}, {'measure': 'Evaluation of somatic mutations', 'timeFrame': '2021-2026', 'description': 'to define somatic mutations in autoimmune cytopenias and myelodysplastic syndromes by NGS'}, {'measure': 'Evaluation of pyruvate kinase activity', 'timeFrame': '2021-2026', 'description': 'to define pyruvate kinase activity in myelodysplastic syndromes'}, {'measure': 'Evaluation of microbiome', 'timeFrame': '2021-2026', 'description': 'to define microbiome composition in autoimmune cytopenias and myelodysplastic syndromes'}, {'measure': 'Single cell RNA expression', 'timeFrame': '2021-2026', 'description': 'to define bone marrow composition by single cell analysis in autoimmune cytopenias and myelodysplastic syndromes'}, {'measure': 'Evaluation of cytokine levels', 'timeFrame': '2021-2026', 'description': 'to define cytokine levels in autoimmune cytopenias and myelodysplastic syndromes by ELISA'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['autoimmune hemolytic anemia', 'immune thrombocytopenia', 'chronic idiopathic neutropenia', 'autoimmune neutropenia', 'cold agglutinin disease', 'myelodysplastic syndromes', 'rituximab', 'thrombopoietin receptor agonists', 'erythropoietin', 'steroids', 'splenectomy', 'red cell metabolism', 'cytokines', 'luspatercept', 'microbiome', 'single cell analysis', 'next generation sequencing'], 'conditions': ['Autoimmune Hemolytic Anemia', 'Immune Thrombocytopenia', 'Chronic Idiopathic Neutropenia', 'Autoimmune Neutropenia', 'Myelodysplastic Syndromes', 'Cold Agglutinin Disease']}, 'referencesModule': {'references': [{'pmid': '38029356', 'type': 'DERIVED', 'citation': 'Fattizzo B, Pedone GL, Brambilla C, Pettine L, Zaninoni A, Passamonti F, Barcellini W. Recombinant erythropoietin in autoimmune hemolytic anemia with inadequate bone marrow response: a prospective analysis. Blood Adv. 2024 Mar 12;8(5):1322-1327. doi: 10.1182/bloodadvances.2023011798.'}, {'pmid': '38013593', 'type': 'DERIVED', 'citation': 'Versino F, Revelli N, Villa S, Pettine L, Zaninoni A, Prati D, Passamonti F, Barcellini W, Fattizzo B. Transfusions in autoimmune hemolytic anemias: Frequency and clinical significance of alloimmunization. J Intern Med. 2024 Mar;295(3):369-374. doi: 10.1111/joim.13753. Epub 2023 Nov 27.'}]}, 'descriptionModule': {'briefSummary': '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.\n\nThe main aims to answer are:\n\n* evaluation of traditional and novel diagnostic tools including immunohematology, cytokine essays, bone marrow studies, molecular findings, and fecal microbiome.\n* evaluation of type and sequence of the therapies administered, the response rates, and the adverse events.\n* evaluation of clinical and laboratory (immunologic, molecular, and morphologic) predictors of outcome.\n* evolution of autoimmune cytopenias into myelodysplastic syndromes.\n* a subgroup of patients with myelodysplastic syndromes will be included to evaluate the presence of immunologic events, autoimmune activation, and red cell metabolism.\n\nParticipants 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.', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'PROBABILITY_SAMPLE', 'studyPopulation': "Patients with anemia (Hb \\<12 g/dL), thrombocytopenia (PLT\\<100.000/mmc) and/or neutropenia (neutrophils\\<1000/mmc) attending the inpatient or outpatient facility of Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.\n\nCytopenia diagnosis will be based on current guidelines for AIHA (evidence of hemolysis and positive direct Coombs test or negative once excluded other causes), for ITP (exclusion of other thrombocytopenia causes) and for CIN/AIN (exclusion of other neutropenia causes). Patients will be classified according to the degree of the cytopenia and, for AIHA, according to thermal characteristics (warm, cold, mixed, atypical).\n\nFor the substudy of patients with MDS patients will be stratified according to WHO 2022 classification of myeloid neoplasms.", 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of autoimmune cytopenias (AIHA/ITP/CIN/AIN)\n* age \\>/= 18 years\n* ability to sign informed consent\n* availability to undergo 3 year follow up\n* 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.\n\nExclusion Criteria:\n\n* any condition impeding the acquisition of the informed consent\n* immune cytopenia diagnosis preceding \\>/= 6 months the enrolment'}, 'identificationModule': {'nctId': 'NCT05931718', 'acronym': 'AIHA ITP CIN', 'briefTitle': 'Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia', 'organization': {'class': 'OTHER', 'fullName': "Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico"}, 'officialTitle': 'Evaluation of the Diagnostic/Therapeutic Course of Patients With Autoimmune Cytopenias (Autoimmune Hemolytic Anemia AIHA, Immune Thrombocytopenia ITP, Chronic Idiopathic/Autoimmune Neutropenia CIN/AIN) and Identification of Predictive and Prognostic Markers.', 'orgStudyIdInfo': {'id': 'CYTOPAN'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Autoimmune hemolytic anemia', 'description': 'AIHA patients will be enrolled at diagnosis and stratified according to AIHA type (i.e. warm, cold, mixed, and atypical forms), sampled for peripheral blood for cytokine and NGS studies, and for feces for microbiome studies. If clinically indicated, bone marrow evaluation will be performed and a sample for single cell analysis will be collected. Patients will be followed up to collect treatments, responses, relapses, and complications (particularly thromboses and infections).', 'interventionNames': ['Biological: cytokine essays', 'Biological: NGS', 'Biological: Fecal microbiome', 'Drug: Erythropoietin']}, {'label': 'Immune thrombocytopenia', 'description': 'ITP patients will be enrolled at diagnosis and sampled for peripheral blood for cytokine and NGS studies, and for feces for microbiome studies. If clinically indicated, bone marrow evaluation will be performed and a sample for single cell analysis will be collected.Patients will be followed up to collect treatments, responses, relapses, and complications (particularly thromboses and infections).', 'interventionNames': ['Biological: cytokine essays', 'Biological: NGS', 'Biological: Fecal microbiome', 'Drug: Thrombopoietin Receptor Agonist']}, {'label': 'Chronic idiopathic neutropenia/Autoimmune neutropenia', 'description': 'CIN/AIN patients will be enrolled at diagnosis and sampled for peripheral blood for cytokine and NGS studies, and for feces for microbiome studies. If clinically indicated, bone marrow evaluation will be performed and a sample for single cell analysis will be collected.Patients will be followed up to collect treatments, responses, relapses, and complications (particularly infections).', 'interventionNames': ['Biological: cytokine essays', 'Biological: NGS', 'Biological: Fecal microbiome', 'Drug: G-CSF']}, {'label': 'Myelodysplastic syndromes', 'description': 'MDS patients will be enrolled at diagnosis and sampled for peripheral blood for cytokine and NGS studies, and to evaluate red cell metabolism. If clinically indicated, bone marrow evaluation will be performed and a sample for single cell analysis will be collected. Patients will be followed up to collect treatments, responses, relapses, and outcome.', 'interventionNames': ['Biological: cytokine essays', 'Biological: NGS', 'Biological: Fecal microbiome', 'Drug: Luspatercept']}], 'interventions': [{'name': 'cytokine essays', 'type': 'BIOLOGICAL', 'description': 'evaluation of immunomodulatory cytokines by ELISA kits on peripheral blood samples', 'armGroupLabels': ['Autoimmune hemolytic anemia', 'Chronic idiopathic neutropenia/Autoimmune neutropenia', 'Immune thrombocytopenia', 'Myelodysplastic syndromes']}, {'name': 'NGS', 'type': 'BIOLOGICAL', 'description': 'evaluation of somatic mutations commonly associated with myeloid neoplasm and immunodeficiencies by next generation sequencing on peripheral blood samples', 'armGroupLabels': ['Autoimmune hemolytic anemia', 'Chronic idiopathic neutropenia/Autoimmune neutropenia', 'Immune thrombocytopenia', 'Myelodysplastic syndromes']}, {'name': 'Fecal microbiome', 'type': 'BIOLOGICAL', 'description': 'evaluation of fecal microbiome on fecal samples', 'armGroupLabels': ['Autoimmune hemolytic anemia', 'Chronic idiopathic neutropenia/Autoimmune neutropenia', 'Immune thrombocytopenia', 'Myelodysplastic syndromes']}, {'name': 'Erythropoietin', 'type': 'DRUG', 'description': 'evaluation of recombinant erythropoietin use, safety and efficacy in patients with autoimmune hemolytic anemia according to clinical practice', 'armGroupLabels': ['Autoimmune hemolytic anemia']}, {'name': 'Luspatercept', 'type': 'DRUG', 'description': 'evaluation of cytokine levels, molecular profile and bone marrow microenvironment by single cell analysis in patients treated with luspatercept according to clinical practice', 'armGroupLabels': ['Myelodysplastic syndromes']}, {'name': 'Thrombopoietin Receptor Agonist', 'type': 'DRUG', 'description': 'evaluation of TPO-RA use, safety and efficacy in patients with ITP according to clinical practice', 'armGroupLabels': ['Immune thrombocytopenia']}, {'name': 'G-CSF', 'type': 'DRUG', 'description': 'evaluation of G-CSF use, safety and efficacy in patients with CIN/AIN according to clinical practice', 'armGroupLabels': ['Chronic idiopathic neutropenia/Autoimmune neutropenia']}]}, 'contactsLocationsModule': {'locations': [{'zip': '20100', 'city': 'Milan', 'status': 'RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Bruno Fattizzo, MD', 'role': 'CONTACT', 'email': 'brunofattizzo@gmail.com', 'phone': '+390255033477'}], 'facility': "Fondazione Irccs Ca' Granda Ospedale Maggiore Policlinico", 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': "Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico", 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}