ITP Registry and Accompanying Biospecimen Collection
Immune ThrombocytopeniaThe 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.
An Exploratory Study of Herombopag for Thrombocytopenia Induced by Chemotherapy Combined With Immunotherapy...
Non-small Cell Lung CancerThe purpose of this study is to explore the safety and efficacy of herombopag olamine tablets for thrombocytopenia induced by chemotherapy combined with immunotherapy in non-small cell lung cancer
Investigation of the Genetics of Hematologic Diseases
Bone Marrow Failure SyndromesErythrocyte Disorder11 moreThe purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases. This is not a therapeutic or diagnostic protocol for clinical purposes. Blood, bone marrow, hair follicles, nail clippings, urine, saliva and buccal swabs, left over tissue, as well as health information will be used to study and learn about blood diseases by using genetic and/or genomic research. In general, genetic research studies specific genes of an individual; genomic research studies the complete genetic makeup of an individual. It is not known why many people have blood diseases, because not all genes causing these diseases have been found. It is also not known why some people with the same disease are sicker than others, but this may be related to their genes. By studying the genomes in individuals with blood diseases and their family members, the investigators hope to learn more about how diseases develop and respond to treatment which may provide new and better ways to diagnose and treat blood diseases. Primary Objective: Establish a repository of DNA and cryopreserved blood cells with linked clinical information from individuals with non-malignant blood diseases and biologically-related family members, in conjunction with the existing St. Jude biorepository, to conduct genomic and functional studies to facilitate secondary objectives. Secondary Objectives: Utilize next generation genomic sequencing technologies to Identify novel genetic alternations that associate with disease status in individuals with unexplained non-malignant blood diseases. Use genomic approaches to identify modifier genes in individuals with defined monogenic non-malignant blood diseases. Use genomic approaches to identify genetic variants associated with treatment outcomes and toxicities for individuals with non-malignant blood disease. Use single cell genomics, transcriptomics, proteomics and metabolomics to investigate biomarkers for disease progression, sickle cell disease (SCD) pain events and the long-term cellular and molecular effects of hydroxyurea therapy. Using longitudinal assessment of clinical and genetic, study the long-term outcomes and evolving genetic changes in non-malignant blood diseases. Exploratory Objectives Determine whether analysis of select patient-derived bone marrow hematopoietic progenitor/stem (HSPC) cells or induced pluripotent stem (iPS) cells can recapitulate genotype-phenotype relationships and provide insight into disease mechanisms. Determine whether analysis of circulating mature blood cells and their progenitors from selected patients with suspected or proven genetic hematological disorders can recapitulate genotype-phenotype relationships and provide insight into disease mechanisms.
Telitacicept for the Treatment of Connective Tissue Disease-associated Thrombocytopenia
Connective Tissue DiseasesThrombocytopeniaThe goal of this clinical trial is to evaluate the efficacy and safety of Telitacicept for the treatment of connective tissue disease-associated thrombocytopenia.
A Study of Selinexor Monotherapy in Subjects With JAK Inhibitor (JAKi)-naïve Myelofibrosis and Moderate...
MyelofibrosisModerate ThrombocytopeniaThe main purpose of this Phase 2 study is to evaluate the efficacy of selinexor in participants with myelofibrosis (MF) and moderate thrombocytopenia based on spleen volume reduction (SVR). Additional efficacy parameters and safety will also be assessed during the study.
Characterization and Quantification of Platelet/Leukocyte Aggregates in Patients With Suspected...
Heparin-induced ThrombocytopeniaHIT6 moreThe investigators would like to analyze platelet/leukoplak activation, quantify and morphologically characterize these aggregates using in vitro analysis, directly on whole blood from adult patients with suspected HIT. This would enable to better describe the in vivo pathophysiology of the HIT-suspect patient, and eliminate the need for platelet donors to perform the usual confirmatory techniques, whose inter- and intra-individual variability is very high. What's more, the SRA test, evaluated as the reference test, requires the use of radioisotopes and is therefore only carried out in a few biological expertise centers, resulting in a very long delay in the delivery of results. It should also be noted that, in practice, HÉPARINE is immediately stopped in patients with suspected HIT, and they are put on an expensive anticoagulant (DANAPAROIDE SODIQUE or ARGATROBAN) in curative doses until the results of the confirmatory tests are back. In this serious, life-threatening condition, it is essential to have a sensitive, specific test to confirm HIT as quickly as possible. Each patient with suspected HIT (rapid >30% reduction in platelet count after initiation of heparin therapy) should have 4 tubes of 3.2% citrated whole blood (2.7ml) collected at the time of suspected HIT (D0) and before any therapeutic switch (or 24 h max after switch). A new sample (4 citrated tubes) will be taken at D4, D7 and D14 during hospitalization, in patients who test positive for anti-PF4/H Ac. All patients with suspected HIT will follow the standard diagnostic pathway, i.e. a screening test (immunological test for anti-PF4/heparin antibodies, total Ig, ACL TOP, Werfen) followed by a confirmatory test (platelet aggregation on TA-8V, STAGO and/or SRA). Doppler ultrasonography of the lower limbs will be performed in HIT-suspect patients with a positive anti-PF4/H antibody test, as currently performed as part of routine care.
IVIg Plus Low Dose rhTPO for ITP in Pregnancy
Immune ThrombocytopeniaPregnancy RelatedRandomized, open-label, multicenter study to investigate the efficacy and safety of IVIg plus low-dose recombinant thrombopoietin in pregnant patients with corticosteroid or IVIg monotherapy-resistant ITP.
A Clinical Trial to Assess Safety and Efficacy of Daratumumab in the Treatment of Primary Immune...
Immune ThrombocytopeniaA single-center, open-label, off-label use investigator-initiated clinical study with safety run-in to explore the clinical activity and safety of daratumumab in adult ITP patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including rituximab and/or TPO-RA.
Zanubrutinib and Eltrombopag as Second-line Treatment in Adults With Primary Immune Thrombocytopenia...
Immune ThrombocytopeniaRandomized, open-label, multicenter study to compare the efficacy and safety of zanubrutinib plus eltrombopag compared to eltrombopag monotherapy for the first-line treatment of adults with chronic immune thrombocytopenia (ITP).
A Clinical Study of Hetrombopag Olamine Tablets for Thrombocytopenia Induced by Chemotherapy in...
Breast CancerThis study now plans to explore the efficacy and safety of hetrombopag in chemotherapy-induced thrombocytopenia in advanced breast cancer, so as to further guide the clinical application of hetrombopag in chemotherapy-induced platelets.