Active clinical trials for "Myelodysplastic Syndromes"

This study assesses feasibility and patient acceptability of using a Fitbit to monitor step count and heart rate in transfusion dependent patients with myelodysplastic syndrome. Information from this study may help researchers understand if there is any correlation between activity level and anemia.

The investigators want to compare the global response rate of patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) after six months of treatment with 5-azacitidine on two different doses. First group of 50 mg/m2 for 10 days each 28 days versus 75 mg/m2 for 7 days on 28 days cycles.

Single patient expanded access program to provide galinpepimut-S for eligible patients with AML or MDS who have no other treatment option.

Allogeneic hematopoietic stem cell transplantation (HSCT) is an established form of treatment for hematological abnormalities. Poor graft function, occurs when there poor donor engraftment. A second infusion of unselected donor hematopoietic stem cells (HSC) can result in improvement, but can potentially increase the incidence of graft versus host disease. Cluster of differentiation 34+ (CD34+) selected stem cells depleted of T-cells is an attractive alternative for treatment of poor graft function as it may be associated with less Graft versus Host Disease (GVHD) and enhanced count recovery. The investigators are using the Miltenyi CliniMACS device and CD34 cell selection reagents for the preparation of allogeneic hematopoietic progenitor cell (HPC) transplants for patients who have had prior stem cell transplants and require a stem cell "boost" from the original donor.

The objective of this expanded access protocol (EAP) is to provide access to treatment with imetelstat, the Investigational Product (IP), for eligible adult participants diagnosed with very low, low, intermediate risk (by Revised International Prognostic Scoring System, IPSS-R) myelodysplastic syndromes (MDS) who are red blood cell (RBC) transfusion-dependent, have failed to respond or have lost response or are ineligible for ESAs, had not received prior treatment with either a hypomethylating agent or lenalidomide and were non-del(5q), until such time that imetelstat becomes commercially available.

This is an expanded access program (EAP) for eligible participants designed to provide access to ACE-011.

This protocol provides expanded access to bone marrow transplants for children who lack a histocompatible (tissue matched) stem cell or bone marrow donor when an alternative donor (unrelated donor or half-matched related donor) is available to donate. In this procedure, some of the blood forming cells (the stem cells) are collected from the blood of a partially human leukocyte antigen (HLA) matched (haploidentical) donor and are transplanted into the patient (the recipient) after administration of a "conditioning regimen". A conditioning regimen consists of chemotherapy and sometimes radiation to the entire body (total body irradiation, or TBI), which is meant to destroy the cancer cells and suppress the recipient's immune system to allow the transplanted cells to take (grow). A major problem after a transplant from an alternative donor is increased risk of Graft-versus-Host Disease (GVHD), which occurs when donor T cells (white blood cells that are involved with the body's immune response) attack other tissues or organs like the skin, liver and intestines of the transplant recipient. In this study, stem cells that are obtained from a partially-matched donor will be highly purified using the investigational CliniMACS® stem cell selection device in an effort to achieve specific T cell target values. The primary aim of the study is to help improve overall survival with haploidentical stem cell transplant in a high risk patient population by limiting the complication of GVHD.

The project's objective is to identify and characterize somatic mutations in cases of idiopathic cytopenia of undetermined significance (ICUS) on the basis of molecular defects found in myelodysplastic syndrome (MDS), in order to validate the hypothesis whereby ICUS may be a precursor of MDS

Developing a molecular genetic test that can identify prognostic factors which can predict response to treatment, and to contribute to the establishment of future therapeutic strategies base on prognostic factors by undergoing peripheral blood and bone marrow examination of Myelodysplastic Syndrome (MDS) patients at diagnosis and relapse.

Study type An observational study conducted in different hematological centers in Belgium. Study objectives Primary objective: To assess the impact of newly started treatments on the QOL of patients suffering from myelodysplastic syndromes. Secondary objectives: To assess the impact of newly started therapy on disease perception in MDS patients To study the relation between disease perception and quality of life To examine which clinical and disease specific factors determine QOL in MDS patients Collect information on the transfusion threshold in Belgian hematological centers and evaluate the impact on quality of life. To evaluate whether changes in QOL are related to hematological respons. Study design Newly diagnosed MDS patients who are about to start a treatment or previously diagnosed MDS patients who are starting with a new line of therapy. QOL assessment with the QUALMS. Disease perception measurement using the B-IPQ. Measurement at diagnosis/before start of therapy, at 4 weeks, 12 weeks, and at 24 weeks into treatment. Study endpoints Primary endpoint: Change in QUALMS score at visit timepoints 4 - 12 - 24 weeks after the start of a new treatment. Secondary endpoint: Change in B-IPQ score at visit timepoints 4 - 12 - 24 weeks after the start of a new treatment Association between B-IPQ and QUALMS score. Association between clinical and disease specific factors and QUALMS score Association between transfusion threshold and QUALMS score. Association between hematological response and QUALMS score Summary of eligibility criteria Adult patients with a new diagnosis of MDS (according to WHO 2016 definitions (3) or known patients with MDS who are about to start a new treatment. Signed informed consent. Patients enrolled in an unblinded interventional therapeutic trial are eligible. Exclusion criteria Patients with acute leukemia defined as >20% bone marrow blasts. Patients suffering from an overlap syndrome myelodysplastic/myeloproliferative disease. Patients in post allogeneic transplant setting. Patients enrolled in a blinded interventional therapeutic trial. Patients starting with multiple treatments under investigation at the same moment apart from intensive chemotherapy. Newly diagnosed patients who do not start with treatment. Patients who started a previous treatment less then 12 weeks ago apart from packed cell transfusion (up to 4 weeks allowed). Diagnosis of any previous or concomitant malignancy except when the patient successfully completed treatment (chemotherapy and/or surgery and/or radiotherapy) with curative intent for this malignancy at least 3 months prior to inclusion. Patients refusing to sign informed consent.