Active clinical trials for "Myelodysplastic Syndromes"

The goal of this clinical research study is to find the highest tolerable dose of OPB-31121 that can be given to patients with leukemia or myelodysplastic syndrome (MDS).

This is a prospective, multicenter phase II trial designed to evaluate the safety and activity of the combination of association of arsenic trioxide (ATO) and ascorbic acid in patients with myelodysplastic syndromes

A Phase I, Open-Label, Dose-Escalation Study of CC-11006 In Subjects With Low- or Intermediate-1 Risk Myelodysplastic Syndromes.

The purpose of this study is to evaluate the efficacy of sorafenib in patients with Myelodysplastic Syndrome (MDS). Eligible subjects will receive Sorafenib administered at 400mg orally twice a day, given on days 1-28 of a 28-day cycle. Patients will be evaluated for hematological response after 2 cycles and then every 3 cycles thereafter for a maximum of 5 years from study entry. If a patient achieves a complete response they may receive an additional 6 cycles of therapy beyond documentation of complete response unless unacceptable toxicity occurs. For patients with partial response, hematological improvement or stable disease they will continue treatment until relapse, progression of disease, or unacceptable toxicity occurs.

Patients are being asked to participate in this study because they have a malignant blood disease such as Myelodysplastic Syndrome (MDS), Myeloproliferative Disorder (MPD), Acute Myelogenous Leukemia (AML) or Chronic Myelogenous Leukemia (CML). We feel that patients could benefit from an allogeneic (meaning the cells come from a donor other than themself) stem cell transplant. The donor would be a family member or an unrelated person that is felt to be a good match for the patient. Stem cells are cells that are made in the bone marrow (spongy material that fills the middle of the bones). As the stem cells grow, they change into different types of blood cells that they need. This includes red blood cells that carry oxygen around the body, white blood cells that help to fight infections, and platelets that help to prevent and stop bleeding. Usually, patients are given high doses of chemotherapy before a stem cell transplant. High doses of chemo destroy the bone marrow. Healthy stem cells from a donor are then given to replace the patient's unhealthy cells. However, because of complications with the patient's disease, they have a high risk of having life-threatening side effects. These include serious damage to organs such as the lung, liver, kidney and heart. There is also an increased risk of bacterial, fungal, and viral infections. The other major problem is when a donor's stem cells (also called the graft) find that the patient's cells ( the host cells) are not the same. The donor cells may try to destroy the host's cells. The cells at high risk are those of the skin, liver and intestines. This is called graft versus host disease (GVHD) and it can be fatal. Recently, doctors have been able to use less toxic chemotherapy treatments before patients receive their transplants. This less toxic treatment helps reduce some of the treatment related problems mentioned above. Patient's are being asked to be involved in a research study that uses this approach. One major risk of this low dose treatment is that the patient's body may reject the donor cells. This is called graft rejection. This study is designed to see if this low dose treatment is safe and effective. This treatment plan adds CAMPATH 1H (a special protein called an antibody) to a low dose chemotherapy regimen. After chemo, the patient will receive an allogeneic (cells come from a donor) stem cell transplant. Adding CAMPATH 1H to the transplant medicines may help in treating the disease. CAMPATH 1H may reduce life-threatening and treatment related side effects like GVHD. CAMPATH 1H stays active in the body for a long time which means it may work longer to prevent GVHD. CAMPATH 1H destroys lymphocytes, a type of white cells that help fight infection, and this helps prevent graft rejection. We want to see if the addition of CAMPATH 1H to the patient's pre-transplant low dose chemotherapy will decrease the side effects from an allogeneic stem cell transplant, while providing a curative treatment for patients with blood disorders.

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Tretinoin may help hematologic cancer cells develop into normal white blood cells. PURPOSE: Phase I/II trial to study the effectiveness of arsenic trioxide with or without tretinoin in treating patients who have hematologic cancer that has not responded to previous therapy.

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: This phase I trial is studying the side effects and best dose of 12-O-tetradecanoylphorbol-13-acetate in treating patients with hematologic cancer or bone marrow disorder that has not responded to previous treatment.

RATIONALE: Bone marrow transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy used to kill tumor cells. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Eliminating the T cells from the donor cells before transplanting them may prevent this from happening. PURPOSE: Phase II trial to study the effectiveness of T cell removal to prevent graft-versus-host disease in patients who are undergoing bone marrow transplantation from a donor.

The primary purpose of the study is to evaluate the preliminary safety and efficacy of oral decitabine/cedazuridine in combination with magrolimab.

Myelodysplastic syndromes (MDS) comprise a heterogeneous group of clonal bone marrow neoplasms that predominate in the elderly, with a median age at diagnosis of 70 years. The diagnosis of MDS relies on peripheral blood cytopenia and morphologic dysplasia for one or more hematopoietic cell lineage. Cytopenia is evidenced with hemogram while dysplasia requires bone marrow aspirate, which is an invasive procedure . Considering the low prevalence of disease among subjects referred for suspected MDS, many patients are exposed to unnecessary bone marrow aspiration-related discomfort and harms. Therefore, an objective assay based on a peripheral blood sample that accurately discriminates MDS from other cytopenia etiologies is highly desirable. We have previously developed and refined a flow cytometric analysis protocol for quantifying neutrophil MPO expression in peripheral blood at three university-affiliated hospitals (i.e., Clermont-Ferrand, Saint-Etienne, and Grenoble) (Raskovalova et al, Hematologica 2019). We found that the robust coefficient of variation (RCV, computed as the robust standard deviation divided by the median) within an individual subject was the best parameter in discriminating patients with versus without MDS. Although promising, flow cytometric analysis of neutrophil MPO expression in peripheral blood is technically complex, time consuming, and not standardized. Hence, its performance requires specific expertise and the results show substantial variability. A single ready-to-use tube with lyophilized antibodies would have the potential to standardize the measurement of neutrophil MPO expression in peripheral blood across laboratories, with results available within 30-60 min in routine practice. In this study, the investigators hypothesize that a standardized and semi-automatic flow cytometric assay of neutrophil MPO expression in peripheral blood could accurately rule out MDS and obviate the need for bone marrow aspiration and biopsy, with sensitivity and negative predictive value estimates approaching 100%. In this observational diagnostic accuracy study, burden will be null for recruited patients. No specific intervention is assigned to participants. All diagnostic testing, procedures, and medication ordering are performed at the discretion of attending physicians. A test result will have no impact on patient management. .Compliance with current guidelines disseminated by the French Haute Autorité de Santé (HAS) will be advocated for the diagnostic work-up of patients with suspected MDS. No follow-up visits are planned in this cross-sectional study.