Clinical Trials Logo

Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT06276036
Other study ID # CAPID
Secondary ID
Status Active, not recruiting
Phase N/A
First received
Last updated
Start date July 23, 2019
Est. completion date June 1, 2025

Study information

Verified date March 2024
Source Meyer Children's Hospital IRCCS
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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


Recruitment information / eligibility

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

Study Design


Related Conditions & MeSH terms


Intervention

Other:
Identification of specific markers
Analysis of the immunological profile, Genetic analysis using next-generation sequencing (NGS) technology, Bioinformatic analysis, Functional studies.

Locations

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

Sponsors (1)

Lead Sponsor Collaborator
Meyer Children's Hospital IRCCS

Countries where clinical trial is conducted

United States,  Belgium,  Italy, 

Outcome

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
See also
  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