Active clinical trials for "Myelodysplastic Syndromes"

This is a clinical trial to determine the safety, recommended dose level (RDL), and infection control of BLEX 404 Oral Liquid in combination with azacitidine in patients with International Prognostic Scoring System (IPSS) intermediate-1 (int-1), intermediate-2 (int-2) or high-risk myelodysplastic syndrome (MDS) and Chronic Myelomonocytic Leukemia (CMML).

Indication:Relapsed or refractory AML in patients for whom no established treatment options are available (this indication will heretofore be referred to as the protocol AML indication), or adult patients with MDS who are classified as high risk or very high risk according to the Revised International Prognosis Scoring System (IPSS-R). Number of Investigators and Study Centers:Up to 5 Investigators in the US. Objectives:Dose Escalation Part Primary Objective: To determine the maximum tolerated dose (MTD) of BS HH 002.SA administered subcutaneously once per day for 12 days of a 28-day cycle. Secondary Objectives: To provide an initial safety profile of single and multiple cycles of BS HH 002.SA. To assess the pharmacokinetic (PK) profile of BS HH 002.SA. To explore the anti-tumor activity of BS HH 002.SA in patients with the protocol AML indication or high-risk MDS. To explore cytogenetics of the malignant cells in relation to response to BS HH 002.SA. Cohort Expansion Part Primary Objectives: To evaluate safety and tolerability of BS HH 002.SA at MTD and/or lower dose level (DL) in selected cohorts of patients with the protocol AML indication or high-risk MDS. To evaluate preliminary anti-tumor activity of BS HH 002.SA at MTD and/or lower DL in selected cohorts of patients with the protocol AML indication or high-risk MDS. Secondary Objectives: To assess the PK profile of BS HH 002.SA. To explore cytogenetics of the malignant cells in relation to response to BS HH 002.SA. Study Population:Adult patients with the protocol AML indication or high-risk MDS.

1703: The study is designed as a randomized, phase III, multicenter trial comparing two acute graft-versus-host disease (aGVHD) prophylaxis regimens: tacrolimus/methotrexate (Tac/MTX) versus post-transplant cyclophosphamide/tacrolimus/mycophenolate mofetil (PTCy/Tac/MMF) in the setting of reduced intensity conditioning (RIC) allogeneic peripheral blood stem cell (PBSC) transplantation. 1801: The goal of this protocol is to test the primary hypothesis that the engraftment stool microbiome diversity predicts one-year non-relapse mortality in patients undergoing reduced intensity allogeneic HCT.

At present, the main therapies for myelodysplastic syndromes with ring sideroblasts (MDS-RS) are red blood cell and platelet transfusion, erythropoietin (EPO), androgen, and iron chelation therapy. Lenalidomide is an immunomodulator with multiple mechanisms, including direct targeting of MDS clones, immunomodulation, erythropoiesis restoration, and angiogenesis inhibition. A Phase III, randomized, placebo-controlled trial of oral azacitidine (AZA) in lower-risk MDS reported higher rates of hemoglobin and platelet hematological improvement in patients with AZA monotherapy. Therefore, this study intended to investigate the efficacy and safety of lenalidomide and sequential AZA in the treatment of refractory MDS-RS versus azacitidine monotherapy.

In a randomized controlled phase II/III clinical trial, 58% of patients with lower-risk MDS had at least a 50% reduction in red blood cell (RBC) transfusion units every 8 weeks after roxadustat treatment. In a randomized controlled phase III clinical trial, luspatercept significantly improved transfusion dependence in erythropoietin-stimulating agents (ESA)-refractory MDS-RS and improved hemoglobin response and quality of life, compared to placebo. This study aimed to evaluate the efficacy and safety of roxadustat combined with luspatercept versus luspatercept monotherapy in the treatment of refractory MDS-RS.

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. The main aims to answer are: evaluation of traditional and novel diagnostic tools including immunohematology, cytokine essays, bone marrow studies, molecular findings, and fecal microbiome. evaluation of type and sequence of the therapies administered, the response rates, and the adverse events. evaluation of clinical and laboratory (immunologic, molecular, and morphologic) predictors of outcome. evolution of autoimmune cytopenias into myelodysplastic syndromes. a subgroup of patients with myelodysplastic syndromes will be included to evaluate the presence of immunologic events, autoimmune activation, and red cell metabolism. Participants 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.

This phase II trial evaluates how a curcumin supplement (C3 complex/Bioperine) changes the inflammatory response and symptomatology in patients with clonal cytopenia of undetermined significance (CCUS), low risk myelodysplastic syndrome (LR-MDS), and myeloproliferative neoplasms (MPN). Chronic inflammation drives disease development and contributes to symptoms experienced by patients with CCUS, LR-MDS, and MPN. Curcumin has been shown to have anti-inflammatory and anti-cancer properties and has been studied in various chronic illnesses and hematologic diseases.

Myelodysplastic syndromes (MDS) are clonal bone marrow neoplasms characterized by dysplasia and ineffective hematopoiesis leading to peripheral blood cytopenias, with an increased risk of progression to acute myeloid leukemia. The conventional diagnostic work-up of MDS relies on cytomorphological evaluation of bone marrow, which may be complemented by conventional cytogenetic, flow cytometry, and molecular analysis by next generation sequencing techniques. Suspicion of MDS is the commonest reason for bone marrow aspirate in older patients with unexplained peripheral blood cytopenias. Yet many patients are exposed to unnecessary bone marrow aspiration-related discomfort and harms, because of the limited prevalence of disease among subjects referred for suspected MDS. In this context, a valid and reliable assay based on peripheral blood sample that accurately discriminates MDS from other cytopenia etiologies without requiring invasive bone marrow aspiration is warranted. The accuracy of peripheral blood neutrophil myeloperoxidase expression quantified by flow cytometric analysis for the diagnosis of MDS is supported by three primary studies totaling 211 individuals. An intra-individual robust coefficient of variation (RCV) value for neutrophil myeloperoxidase expression lower than 30.0% accurately ruled out MDS, with both sensitivity and negative predictive value point estimates of 100%, in consecutive patients with suspected disease. This biomarker might obviate the need for cytomorphological evaluation of bone marrow aspirate for up to 35% of patients referred for suspected MDS. Although promising, these preliminary results require replication in an independent external validation sample. The broad aim of the multicenter MPO-MDS-Valid study project is to prospectively validate the diagnostic accuracy of intra-individual RCV for peripheral blood neutrophil myeloperoxidase expression quantified by flow cytometric analysis among consecutive patients referred for suspected MDS.

This study will assess the safety, efficacy, and feasibility of ⍺/β CD3+ T-cell and CD19+ B-cell depletion in allogeneic stem cell transplantation in patients with acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML), juvenile myelomonocytic leukemia (JMML), high risk myelodysplastic syndrome (MDS), chronic myeloid leukemia (CML) and lymphoma. Subjects will receive an allogeneic stem cell transplant that has been depleted of ⍺/β CD3+ T-cells and CD19+ B-cells using the Miltenyi CliniMACS Prodigy® system.

This is an open label Phase I-II study to determine the safe doses of bortezomib, sitagliptin, and PTCy (Phase I) with expansion into a phase II trial to determine efficacy in improving survival.