Clinical Trials

Multi-center study enrolling patients suspected or newly diagnosed with myelodysplastic syndromes (MDS), myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) overlap disorder, or idiopathic cytopenia of undetermined significance (ICUS). Participants will be followed long term. Clinical data, blood, and tissue samples will be collected to establish a biorepository to facilitate the study of the natural history of MDS.

The purpose of phase 2a is to establish proof-of-concept (POC) for AG-946 in participants with lower-risk myelodysplastic syndromes (LR-MDS) and to evaluate the safety, effect, and pharmacokineticsof AG-946 on additional measures of anemia. Additionally to evaluate the effect of AG-946 on transfusion burden (participants with LTB only) and the effect of AG-946 on pharmacodynamic biomarkers.

The purpose of phase 2b is to compare the effect of AG-946 versus placebo and to detect a doseresponse for erythroid response in participants with LR-MD

This study will determine the safety profile, maximum tolerated dose (MTD), dose-limiting toxicities (DLT), and recommended Phase 2 dose (RP2D) of SX-682 in the treatment of patients with Myelodysplastic Syndromes (MDS).

The purpose of this study of various ASTX727 LD doses and schedules to assess safety, pharmacodynamics, pharmacokinetics, and hematologic response in subjects with International Prognostic Scoring System (IPSS) risk category of low-risk or Intermediate-1 Myelodysplastic Syndromes (MDS). This study will be conducted in two phases. In phase 1 subjects will be randomized into 3 cohorts in a 28-day cycles. Phase 2, 80 new subjects will be randomized in a 1:1 ratio into 2 doses/schedules.

The purpose of this study is to evaluate the safety and effectiveness of MBG453, or placebo added to azacitidine, in adult subjects with intermediate, high or very high risk myelodysplastic syndrome (MDS) as per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2) who have an indication for treatment with azacitidine in first-line setting and are not eligible for intensive chemotherapy or hematopoietic stem cell transplantation (HSCT) according to medical judgment by the investigator.

The purpose of this study is to evaluate ALX148 in combination with azacitidine for the treatment of patients with higher risk myelodysplastic syndrome (MDS).

The primary objective of this study is to generate evidence for the content validity of the Functional Assessment of Cancer Therapy - Anemia (FACT-An) and the Quality of Life in Myelodysplasia Scale (QUALMS) for use in an MDS population. Specifically, the study aims to gain an understanding of the experience of patients with MDS, including the key symptoms and impacts, as well as assess the patient understanding and relevance of the FACT-An and QUALMS for use in patients with MDS such that they may be used as trial endpoints.

This is a Phase II, open-label, two strata, multicenter, prospective study of plerixafor-mobilized HLA-identical sibling allografts in recipients with hematological malignancies. This study will establish the safety and efficacy of subcutaneous plerixafor for this purpose.

This 2-stage, open-label study will evaluate safety and pharmacokinetics of ASTX727, as well as determine the dose for the study's second stage. In the second stage the selected dose will be confirmed and evaluated for clinical activity, including response rate.

The purpose of this study is to compare the 1-year cumulative incidence of severe GVHD (from day of HCT) defined as Grade III-IV acute GVHD (aGVHD) and/or chronic GVHD (cGVHD) that requires systemic immunosuppression and to compare the disease free survival (DFS) (from time of randomization) in children and young adults (AYA) with acute myeloid leukemia (AML), acute lymphoid leukemia (ALL), and Myelodysplastic Syndrome (MDS) who are randomly assigned to haploHCT or to an 8/8 adult MUD HCT.

This is a Phase II therapeutic trial combining Decitabine days 1-5 with oral Vorinostat twice daily days 6-15 followed by a single infusion of CD3-/CD19- enriched donor natural killer (NK) cells on day 17 and a short course of Interleukin-2 (IL-2) to facilitate NK cell survival and expansion. Two courses of treatment will be given separated by 6-8 weeks. The intent is to administer all treatment in the outpatient setting.

The purpose of this study is to document the erythroid response rate assessed as per the 2015 International Working Group (IWG) MDS/MPN response criteria.

In this study we want to find out more about the side effects of a new drug combination for MDS/MPN-RS-T and MDS/MPN-U with RS, luspatercept with or without hydroxyurea and what doses of this combination are safe for people to take and how the disease responds to the treatment. There are 2 groups of patients being studied in this trial. One group of patients will receive luspatercept. The other group will receive luspatercept and hydroxyurea. ...

The purpose of this study is to determine how well cytarabine and idarubicin or daunorubicin with or without pembrolizumab work in treating patients with newly-diagnosed acute myeloid leukemia. Drugs used in chemotherapy, such as cytarabine, idarubicin, and daunorubicin, 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. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving induction chemotherapy with pembrolizumab may work ...

The main objective of this study is to describe and evaluate safety and effectiveness of MBG453 (sabatolimab) in combination with FDA approved Hypomethylating Agents (HMAs) of investigator's choice (IV Decitabine or Azacitidine /SC Azacitidine /Oral Decitabine (cedazuridine combination (INQOVI)).

Phase I Dose Escalation: To investigate the MTD, safety, efficacy and pharmacokinetics of volasertib in combination with decitabine in patients ≥ 65 years of age with newly diagnosed previously untreated AML, relapsed or refractory AML or high risk MDS, and considered ineligible for standard intensive therapy. Phase I MTD Extension: To collect additional data on safety, efficacy and pharmacokinetics and to confirm the Recommended Phase II Dose (RP2D), of volasertib in combination with decitabine in previously untreated patients with AML ≥ 65 years of age and considered ineligible for standard intensive therapy. Phase IIa: Efficacy of volasertib in combination with ...

The purpose of this study is to evaluate the safety and tolerability of REM-422 in patients with relapsed/refractory acute myeloid leukemia or higher-risk myelodysplastic syndromes.

The primary purpose of this study is to assess the safety and tolerability of NKX101 including dose-limiting toxicities (DLTs), and to identify the recommended Phase 2 dose (RP2D) and/or maximum tolerated dose (MTD) of NKX101.

The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in advanced hematologic malignancies that harbor an IDH1 mutation. The first portion of the study is a dose escalation phase where cohorts of patients will receive ascending oral doses of AG-120 to determine maximum tolerated dose (MTD) and/or the recommended Phase II dose. The second portion of the study is a dose expansion phase where three cohorts of patients will receive AG-120 to further evaluate the safety, tolerability, and clinical activity of the recommended Phase II dose. Study will not ...

The purpose of this study is to assess tumor cells from blood and bone marrow from patients with myeloid neoplasms for epigenetic dysregulation and abnormalities of chromatin and for immune activation and exhaustion.

The purpose of this study is to characterize the safety, tolerability and confirm the dose for select single agents and combinations in patients with lower risk (very low, low, and intermediate risk) Myelodysplastic Syndrome (MDS).

The purpose of this study is to determine the safety profile, maximum tolerated dose (MTD), dose-limiting toxicities (DLT), and recommended Phase 2 dose (RP2D) of SX-682 in the treatment of patients with Myelodysplastic Syndromes (MDS).

The purpose of Phase I of this study is to evaluate the safety and tolerability of CTX-712 in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) / higher risk myelodysplastic syndromes (HR-MDS) / MDS/MPN to determine the recommended dose of CTX-712. The purpose of Phase II of this study is to assess the effiectiveness of CTX-712 in patients with R/R AML/HR-MDS by determination of complete remission (CR) rates.

This is a Phase 1, open-label, study of TGR-1202, a PI3K delta inhibitor, administered together with ruxolitinib in patients with myeloproliferative neoplasms (specifically: polycythemia vera, primary myelofibrosis, PPV-MF or PET-MF) and MDS/MPN.

This study is a Phase 1 multicenter, open-label study evaluating the safety and efficacy of escalating doses of MT-401-OTS in 2 participant populations: 1) Those with intermediate or high-risk AML per 2022 ELN criteria who have evidence of MRD and/or \

The purpose of this study is to determine the safety and best dose of PRGN-3006 T Cells to treat relapsed/refractory Acute Myeloid Leukemia and High Risk Myelodysplastic Syndrome.

This study will include a Phase 1 dose-finding portion (Cohorts A and B) and a four-arm expansion portion. The primary objectives of the study are to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of H3B-8800 administered orally in participants with Myelodysplastic Syndromes (MDS), Acute Myeloid Leukemia (AML), or Chronic Myelomonocytic Leukemia (CMML) and to assess the safety and tolerability of H3B-8800 as a single agent administered orally once daily on a 5 days on/9 days off repeated dosing schedule in 28-day cycles in participants with MDS, AML, or CMML.

The purpose of this study is to determine whether the combination of pevonedistat and azacitidine improves overall response rate (ORR) by Cycle 6 and whether the combination of pevonedistat and azacitidine improves event-free survival (EFS) when compared with single-agent azacitidine.

The purpose of this study is to test whether decitabine and cedazuridine (ASTX727) in combination with venetoclax work better than ASTX727 alone at decreasing symptoms of bone marrow cancer in patients with chronic myelomonocytic leukemia (CMML), myelodysplastic syndrome/myeloproliferative neoplasm (MDS/MPN) with excess blasts.  

This MyeloMATCH Master Screening and Reassessment Protocol (MSRP) evaluates the use of a screening tool and specific laboratory tests to help improve participants' ability to register to clinical trials throughout the course of their myeloid cancer (acute myeloid leukemia or myelodysplastic syndrome) treatment. This study involves testing patients' bone marrow and blood for certain biomarkers. A biomarker (sometimes called a marker) is any molecule in the body that can be measured. Doctors look at markers to learn what is happening in the body. Knowing about certain markers can give doctors more information about what is driving the cancer and how ...

This is an open-label study of the safety, biodynamics, and anti-cancer activity of SENTI-202 (an off-the-shelf logic gated CAR NK cell therapy) in patients with CD33 and/or FLT3 expressing blood cancers, including AML and MDS.

This study is being done to store blood, buccal (cheek) cells, genetic material including DNA (deoxyribonucleic acid) and RNA (ribonucleic acid), and bone marrow so that they can be used for laboratory studies that may contribute to finding the causes of disease and factors that may determine disease progression and treatment response.

The current study is a minimal risk study that involves abstraction of clinical and laboratory information from patients with myeloid disorders with the intent to accurately define the natural history of a specific disease. The information includes survival, cause of death, disease complications, treatment, and other issues.

The primary purpose of this study is to compare 1 year graft-versus-host disease (GVHD)-free, graft relapse-free survival (GRFS) between the two GVHD prophylaxis regimens.

The primary objective of the United States Food and Drug Administration (FDA) for this study is to demonstrate non-inferiority in subjects who received an allogeneic BMT for subjects randomized to Rezafungin for Injection compared to subjects randomized to the standard antimicrobial regimen (SAR) for fungal-free survival at Day 90 (±7 days).

The primary objective of the European Medicines Agency (EMA) for this study is to demonstrate superiority in subjects who received an allogeneic BMT randomized to Rezafungin for Injection compared to subjects randomized to the SAR for fungal-free survival at Day 90 (±7 days).

The purpose of this study is to develop a data-driven approach that enables healthcare providers to “prescribe” exercise in the appropriate dose in a manner analogous to prescribing a drug.