Phase 1 Trial of ASTX727 in Subjects With Lower-risk Myelodysplastic Syndromes
Lower-risk MyelodysplasticTo investigate the tolerability and safety of ASTX727 in Japanese subjects with lower-risk MDS.
CPX-351 (Vyxeos™) for Transplant Eligible, Higher Risk Patients With Myelodysplastic Syndrome
Myelodysplastic SyndromesThis is a pilot and feasibility study of transplant eligible, higher risk myelodysplastic syndrome (MDS) patients to determine the safety and tolerability of a lower -dose and higher-dose CPX-351 regimen, with secondary objectives including complete remission (CR) rates and proportion of patients proceeding to transplant.
A Study of JNJ-64619178, an Inhibitor of PRMT5 in Participants With Advanced Solid Tumors, NHL,...
NeoplasmsSolid Tumor3 moreThe purpose of the study is to identify the maximum tolerated dose (MTD) of JNJ-64619178 in participants with relapsed/refractory B cell non-Hodgkin lymphoma (NHL) or advanced solid tumors and also to identify the recommended Phase 2 dose(s) (RP2Ds) of JNJ-64619178 for NHL and advanced solid tumors (Part 1) and to confirm the tolerability of JNJ-64619178 in participants with lower risk myelodysplastic syndromes (MDS) (Part 2).
A Study of AG-946 in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS)...
Myelodysplastic SyndromesThis purpose of this study is to establish proof of concept of AG-946 in participants with LR-MDS in Phase 2a and to compare the effect of AG-946 versus placebo and to detect a dose response for erythroid response in participants with LR-MDS in Phase 2b.
DS-1594b With or Without Azacitidine, Venetoclax, or Mini-HCVD for the Treatment of Relapsed or...
Hematopoietic and Lymphoid Cell NeoplasmRecurrent Acute Lymphoblastic Leukemia7 moreThis phase I/II trial studies the effect of DS-1594b with or without azacitidine, venetoclax, or mini-HCVD in treating patients with acute myeloid leukemia or acute lymphoblastic leukemia that has come back (recurrent) or not responded to treatment (refractory). Chemotherapy drugs, such as azacitidine, venetoclax, and mini-HCVD, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. DS-1594b may inhibit specific protein bindings that cause blood cancer. Giving DS-1594b, azacitidine, and venetoclax, or mini-HCVD may work better in treating patients with acute myeloid leukemia or acute lymphoblastic leukemia.
A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124...
Non-transfusion-dependent ThalassemiaLow Risk Myelodysplastic Syndrome1 moreThis study will investigate the safety and tolerability of SLN124 in patients with Thalassaemia or patients with Very Low- and Low-risk Myelodysplastic Syndrome (MDS) after single ascending s.c. doses and multiple doses in healthy male and female subjects. Up to 7 cohorts of 56 patients with Thalassaemia and up to 7 cohorts of 56 patients with MDS will be enrolled. Each subject will receive single or multiple doses of SLN124 or placebo given by subcutaneous (s.c) injection.
TPO-Mimetic Use in Children for Hematopoietic Failure
Bone Marrow Failure DisordersAplastic Anemia3 moreThis is an open label, prospective Pilot interventional study will investigate the safety and efficacy of Romiplostim, thrombopoietin (TPO) mimetic, in children (ages: 0 to 21 years) with broad scope of bone marrow failure disorders including acquired and inherited conditions as a first line of therapy along with standard of care.
Study of 3D189 in Patients With Hematologic Malignancies
Acute LeukemiaMultiple Myeloma2 moreTo assess the safety, immunogenicity and preliminary efficacy of 3D189 in patients with hematological malignancies.
A Trial of Epigenetic Priming in Patients With Newly Diagnosed Acute Myeloid Leukemia
Acute Myeloid LeukemiaMyelodysplastic SyndromesThe overall aim of this study is to determine if epigenetic priming with a DNA methyltransferase inhibitor (DMTi) prior to chemotherapy blocks is tolerable and carries evidence of a clinical efficacy signal as determined by minimal residual disease (MRD), event-free survival (EFS), and overall survival (OS). Tolerability for each of the agents, as well as total reduction in DNA methylation and outcome assessments will be done to simultaneously obtain preliminary biological and clinical data for each DMTi in parallel. PRIMARY OBJECTIVES: Evaluate the tolerability of five days of epigenetic priming with azacitidine and decitabine as a single agent DMTi prior to standard AML chemotherapy blocks. Evaluate the change in genome-wide methylation burden induced by five days of epigenetic priming and the association of post-priming genome-wide methylation burden with event-free survival among pediatric AML patients. SECONDARY OBJECTIVES Describe minimal residual disease levels following Induction I chemotherapy in patients that receive DMTi. Estimate the event-free survival and overall survival of patients receiving a DMTi prior to chemotherapy courses.
Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Myelodysplastic Syndrome Low...
MDSComparison of survival in patients with or without a matched donor at 36 months