Cytopenia Clinical Trial
Official title:
Autoimmune Cytopenias as a Sign of Primary Immunodeficiency: Immunological and Molecular Approach to Optimize the Diagnostic-therapeutic Pathway
Verified date | March 2024 |
Source | Meyer Children's Hospital IRCCS |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
Autoimmune cytopenias resistant to treatment are among the most common clinical manifestations observed in patients with congenital alterations of the immune system, such as primary immunodeficiencies (PI). The exact contribution of immune system alterations to the pathogenesis of autoimmune cytopenias has not yet been fully elucidated. Moreover, conventionally employed therapeutic strategies often fail, leading to increased healthcare costs, high morbidity, and even mortality. Therefore, there is a need to establish clinical guidelines for diagnosis and to identify early biomarkers capable of identifying individuals responsive to therapy. Thus, a systematic approach to the study of such pathologies will allow for the identification of early biomarkers and facilitate the development of targeted therapeutic strategies
Status | Active, not recruiting |
Enrollment | 53 |
Est. completion date | June 1, 2025 |
Est. primary completion date | December 1, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Year to 17 Years |
Eligibility | Inclusion Criteria: - Clinical and hematological diagnosis of autoimmune cytopenia Additional inclusion criteria for classification into responder vs. non-responder: - For immune thrombocytopenic purpura: platelet count increase >30,000 with at least a twofold increase from pre-treatment value - For autoimmune hemolytic anemia: Hb =10 g/dL with an increase of at least 2 g/dL compared to baseline Exclusion Criteria: - Transient cytopenia without confirmation of autoimmunity where frontline treatment is not necessary |
Country | Name | City | State |
---|---|---|---|
Belgium | Ghent University Hospital | Ghent | |
Italy | Clinica Pediatrica - Università di Catania | Catania | |
Italy | Meyer Children's Hospital IRCCS | Florence | |
Italy | IRCCS Istituto Giannina Gaslini | Genova | |
Italy | Azienda Ospedaliero Universitaria Pisana | Pisa | |
United States | University of South Florida | Tampa | Florida |
Lead Sponsor | Collaborator |
---|---|
Meyer Children's Hospital IRCCS |
United States, Belgium, Italy,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Identification of specific markers | The primary goal is to identify potential primary immunodeficiencies (PI) as responsible for autoimmune cytopenias through a comprehensive examination of clinical manifestations and the study of immune system cells in the patient cohort under investigation. The study will involve immunophenotypic investigation of T and B cell subsets using flow cytometry, the application of genomic approaches, and possibly the use of specific functional immunological tests to confirm and validate genetic results. | Every three to six months |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT02104440 -
Clinical Trial In The Treatment Of Allogeneic Post-Transplant Cytopenias With Sequential Infusion Of Allogeneic Mesenchymal Cells Expanded In Vitro
|
Phase 2 | |
Active, not recruiting |
NCT03682029 -
Epigenetics, Vitamin C, and Abnormal Blood Cell Formation - Vitamin C in Patients With Low-Risk Myeloid Malignancies
|
N/A | |
Recruiting |
NCT03026751 -
Improving Diagnosis in Idiopathic Cytopenia Using Gene Sequencing
|
N/A | |
Recruiting |
NCT06430788 -
A Study of Emapalumab for Pediatric Aplastic Anemia
|
Phase 2 | |
Recruiting |
NCT04873102 -
Danazol for Treatment of Cytopenias in Patients With Cirrhosis
|
Phase 2 | |
Not yet recruiting |
NCT06285825 -
A Pilot Study of Emapalumab for the Treatment of CAR T-Cell Therapy-Associated Prolonged Cytopenia
|
Early Phase 1 | |
Terminated |
NCT03733249 -
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
|
Phase 1/Phase 2 | |
Recruiting |
NCT02958462 -
Pre-myeloid Cancer and Bone Marrow Failure Clinic Study
|
N/A | |
Recruiting |
NCT05236764 -
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
|
N/A | |
Recruiting |
NCT04741945 -
Repurposing Metformin as a Leukemia-preventive Drug in CCUS and LR-MDS
|
Phase 2 | |
Completed |
NCT01928537 -
Efficacy and Safety of IV Rigosertib in MDS Patients With Excess Blasts Progressing After Azacitidine or Decitabine
|
Phase 3 | |
Recruiting |
NCT04419649 -
A Study of KER-050 to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes
|
Phase 2 | |
Completed |
NCT04070612 -
National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia.
|
||
Active, not recruiting |
NCT03301168 -
Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant
|
Phase 1/Phase 2 | |
Terminated |
NCT02065869 -
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
|
Phase 1/Phase 2 | |
Completed |
NCT00367588 -
Low Bacterial Diet in Patients With Cytopenia
|
N/A |