Immune Thrombocytopenia

This is a multinational, open label, single arm study that will evaluate the impact of early multi-immune modulation with rilzabrutinib in adult ITP patients who failed first-line treatment. The study includes a screening period (up to 8 weeks), a primary analysis period (up to 28 weeks), a long-term extension period for selected participants (28 weeks) and a 24-week follow-up period only for eligible participants.

The goal of this clinical trial is to learn if sirolimus, added to standard background therapy (corticosteroids and hydroxychloroquine), works to treat systemic lupus erythematosus-associated immune thrombocytopenia (SLE-ITP) in adults aged 18 to 65 years. The study also aims to evaluate the long-term effectiveness and safety of sirolimus over 48 weeks. The main questions it aims to answer are: 1. Does sirolimus increase the overall response rate (complete or partial remission of thrombocytopenia) at 24 weeks compared with placebo? 2. The safety issues when taking sirolimus over 24 weeks (Phase 1) and up to 48 weeks (Phase 2). 3. How long does the treatment effect last, and what is the relapse rate during open-label extension? In Phase 1 (first 24 weeks), researchers will compare sirolimus to a placebo (an identical-looking capsule containing no active drug) to see if sirolimus works to treat SLE-ITP when both groups also receive standardized prednisone and hydroxychloroquine. In Phase 2 (weeks 24 to 48), all participants will receive open-label sirolimus to assess long-term efficacy and safety. Participants will: 1. Phase 1: Take sirolimus (1.5 mg once daily) or a placebo for 24 weeks, plus standardized prednisone (tapered according to protocol) and hydroxychloroquine (0.4 g daily) 2. Phase 2: After completing Phase 1, take open-label sirolimus (1.5 mg once daily) for an additional 24 weeks, continuing stable or tapering doses of prednisone and hydroxychloroquine 3. Visit the clinic at screening, baseline, weeks 4, 12, 24 (end of Phase 1), and then at weeks 28, 36, and 48 (end of Phase 2) for checkups, blood tests, and disease activity assessments 4. Receive telephone follow-ups at weeks 8, 16, 20, 32, 40, and 44 to report platelet counts, medication adherence, and adverse events

This study aims to describe the treatment patterns and clinical outcomes of treatment with eltrombopag in primary immune thrombocytopenia (ITP) patients treated with prior corticosteroid therapy. This study will use secondary data provided by health care professionals (HCPs) for a sample of primary ITP patients, and primary data from a structured HCP survey. Data will be collected from HCPs working in various clinical centers across the United States (US), United Kingdom (UK), and Germany.

This study will evaluate the safety and efficacy of tafasitamab in adult participants with primary autoimmune blood cell disorders.

Immune thrombocytopenia (ITP) is an autoimmune disease but, paradoxically, and unlike other autoimmune diseases, antiplatelet antibodies are not used either for the diagnosis of the disease or for its prognosis. ITP is a diagnosis of exclusion retained after elimination of other pathologies leading to a thrombocytopenia. No major study has prospectively evaluated the diagnostic value of the presence of anti-platelet antibodies in the etiological investigation of a thrombocytopenia, nor the impact of platelet antibodies on the course of ITP. The gold standard analysis for the determination of platelet antibodies, is the "monoclonal antibody immobilization of platelet antigens" assay (MAIPA), either direct to detect autoantibodies attached to platelets, or indirect to detect circulating antiplatelet antibodies. Therefore, this work aims to study the contribution of the presence of anti-platelet antibodies detected in MAIPA to determine the autoimmune nature of a thrombocytopenia at diagnosis.

Evaluate the safety and tolerance of multiple subcutaneous injections of SHR-2173 in ITP patients

The goal of this clinical trial is to demonstrate GL-2045 safety and efficacy proof of concept by demonstrating intravenous immunoglobulin (IVig)-like platelet responses in adult patients with Immune Thrombocytopenia (ITP).

This is a Phase I, single-arm, open-label, dose-escalation and dose-expansion study. This study evaluates the safety and efficacy of universal STAR-T cells in patients with R/R CTD-associated Immune Thrombocytopenia (CTD-ITP). Approximately 9 patients aged 18-65 will receive infusion of universal STAR-T cells at the starting dose of 3E6 STAR+T cells/kg. The main purpose of exploratory clinical research is to explore the efficacy and safety of universal STAR-T cell and the lymphodepletion regimen. The primary endpoint is observations of types, severity, and frequency of adverse events (AEs) and efficacy assessment. This single-arm, open-label trial will enroll patients across Chinese People's Liberation Army (PLA) General Hospital.

A multicenter, randomized, double-blind placebo-controlled study to report the efficacy and safety of all-trans etinoic acid compared to placebo for the treatment of adults with corticosteriod-resistant/relapsed primary immune thrombocytopenia (ITP).

This randomized, open-label study aim to compare the efficacy and safety of Daratumumab (anti-CD38 monoclonal antibody) with rituximab in ITP patients.This study will be conducted in ITP patients who had not responded to or had relapsed after previous glucocorticoid treatment.

This randomized, open-label study aim to compare the efficacy and safety of rituximab combining anti-CD38 monoclonal antibody with rituximab in ITP patients.This study will be conducted in ITP patients who had not responded to or had relapsed after previous glucocorticoid treatment.

This single-arm, open-label phase II study aim to evaluate the efficacy and safety of Daratumumab (anti-CD38 monoclonal antibody) combined with Rituximab in ITP patients.This study will be conducted in ITP patients who had not responded to or had relapsed after previous glucocorticoid treatment.

This randomized, open-label study aim to compare the efficacy and safety of Daratumumab (anti-CD38 monoclonal antibody) with Rituximab in pediatric ITP patients.This study will be conducted in pediatric ITP patients who had not responded to or had relapsed after previous glucocorticoid treatment.

CARMEN is a national, real-world clinical registry of all adult patients with incident diagnosis of Immune thrombocytopenia (ITP) or Autoimmune Hemolytic anemia (AIHA) patients in France. It is aimed at describing ITP and AIHA clinical features, assessing the real-world risk-benefit ratio of treatments and adherence to guidelines for ITP and AIHA management.

The goal of this clinical trial is to find the best dose of zanubrutinib when used together with high-dose dexamethasone for adults who are newly diagnosed with primary immune thrombocytopenia (ITP), and to learn how safe and effective this combination treatment is.

The purpose of this study is to assess the safety and efficacy of CD19 UCAR-T cell therapy in Subjects with autoimmune diseases.

Primary immune thrombocytopenia (ITP) is an autoimmune- mediated acquired bleeding disorder, defined as a platelet count less than 100×109/L without other causes of isolated thrombocytopenia. The etiology of ITP is complex and heterogeneous, and as no specific biomarkers are indicating its presence, ITP remains a diagnosis of exclusion. The heterogeneous nature of ITP is evident in the differences in clinical presentation and response to regular treatment among patients and the multiple mechanisms that have been forwarded to account for it, such as autoantibodies, T cell dysregulation, and impaired megakaryocytes. Except primary ITP, all forms of immune-mediated thrombocytopenia is defined as secondary ITP. Secondary ITP has several causes, including autoimmune diseases such as systemic lupus erythematosus

This is a single center, open-label, 3+3 dose escalation, early phase 1 study to evaluate the safety, tolerability, and preliminary efficacy of PRG-1801 for patients with relapsed/refractory immune thrombocytopenia (ITP).

To evaluate the safety and efficacy of Anti-CD38 Antibody in the treatment of pediatric primary immune thrombocytopenia in patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including Anti-CD20 Antibody and/or TPO-RA, or those in whom no other second-line treatment options are suitable.

To evaluate the safety and efficacy of Anti-CD38 Antibody in the treatment of primary immune thrombocytopenia in patients who have failed first-line treatment.

To evaluate the safety and efficacy of nicotinamide adenine dinucleotide in the treatment of immune thrombocytopenia in patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including Anti-CD20 Antibody and/or TPO-RA.

The objective of this ITP registry is to collect clinical information, including biosampling, from consenting patients with a variety of ITPs at different points in the course of their disease.

Randomized, open-label, multicenter study to compare the efficacy and safety of Upadacitinib plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).

To evaluate the safety and efficacy of Anti-CD38 Antibody in the treatment of elderly patients with primary immune thrombocytopenia in patients who have failed multiple treatment.

The Internal Medicine Department of Haut-Lévêque Hospital (Pr E LAZARO, Pr JL PELLEGRIN, Pr JF VIALLARD) was accredited in 2017 by the Ministry of Health as a Constitutive Reference Center for Autoimmune Cytopenia. The investigators wish to launch new research projects in autoimmune cytopenia and propose a translational and fundamental research based on collaboration between the clinical department, the biological resource center and the CNRS and INSERM research units ("Bedside to the Bench Strategy"). Thus, in the perspective of future research work, it seems imperative to set up a biological bank for the patients followed in our Reference Center.