Dose Escalation/ Expansion Study of CA-4948 as Monotherapy in Patients With AML or MDS
Acute Myelogenous LeukemiaMyelodysplastic SyndromeThis is a multicenter, open-label, Phase 1/2a dose escalation and expansion study of orally administered emavusertib (CA-4948) monotherapy in adult patients with Acute Myelogenous Leukemia (AML) or high risk Myelodysplastic Syndrome (MDS). Patients enrolling in the Phase 1 portion of the study must meet one of the following criteria prior to consenting to the study: R/R AML with FLT3 mutations who have been previously treated with a FLT3 inhibitor R/R AML with spliceosome mutations of SF3B1 or U2AF1 R/R hrMDS with spliceosome mutations of SF3B1 or U2AF1 Number of pretreatments: 1 or 2 The Phase 2a Dose Expansion will be in 3 Cohorts of patients: R/R AML with FLT3 mutations who have been previously treated with a FLT3 inhibitor; R/R AML with spliceosome mutations of SF3B1 or U2AF1; and R/R hrMDS (IPSS-R score > 3.5) with spliceosome mutations of SF3B1 or U2AF1. All patients above have had ≤ 2 lines of prior systemic anticancer treatment. In previous versions of this protocol there was a Phase 1b portion of the study, in which patients with AML or hrMDS received CA-4948 in combination with venetoclax. This part of the study is no longer open for enrollment.
Tagraxofusp in Patients With CD123+ or With BPDCN-IPh-like Acute Myeloid Leukemia
Acute Myeloid LeukemiaNon-randomized, open-label, multicenter phase II Study for the treatment of 25 R/R BPDCN-IF (CD123/CD4/CD56 positive) AML patients and 25 patients presenting R/R AML CD123+, but negative for either, or both, CD4 and CD56. Patients will be treated with 12 mcg/kg/day of tagraxofusp for 5 days, for at least 4 cicles.
Ivosidenib and Combination Chemotherapy for the Treatment of IDH1 Mutant Relapsed or Refractory...
Recurrent Acute Myeloid LeukemiaRecurrent Myelodysplastic Syndrome3 moreThis phase I trial studies the side effects and best dose of ivosidenib when given together with combination chemotherapy for the treatment of 1DH1 mutant acute myeloid leukemia that has come back (relapsed) or does not respond to treatment (refractory). Ivosidenib may stop the growth of cancer cells by blocking the IDH1 mutation and some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as fludarabine phosphate, cytarabine, and filgrastim, 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. Giving ivosidenib with combination chemotherapy may work better in treating patients with acute myeloid leukemia compared to chemotherapy alone.
RVU120 in Patients With Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome
Acute Myeloid LeukemiaHigh-risk Myelodysplastic SyndromeThis first-in-human study will evaluate RVU120 (SEL120), a novel small molecule CDK8/19 inhibitor, in patients with Acute Myeloid Leukemia (AML) or High-risk Myelodysplastic Syndrome (HR-MDS), in terms of selection of the recommended dose for further clinical development and assessment of safety, tolerability, preliminary anti-leukemic activity, as well as pharmacokinetic and pharmacodynamic profiles.
Pembrolizumab and Decitabine With or Without Venetoclax in Treating Patients With Acute Myeloid...
Acute Myeloid LeukemiaMyelodysplastic Syndrome4 moreThis phase Ib trial studies the side effects and best dose of pembrolizumab and how well it works in combination with decitabine with or without venetoclax in treating patients with acute myeloid leukemia or myelodysplastic syndrome that is newly-diagnosed, has come back (recurrent), or does not respond to treatment (refractory). 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. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. This trial may help doctors find the best dose of pembrolizumab that can be safely given in combination with decitabine with or without venetoclax, and to determine what side effects are seen with this treatment.
A Clinical Study of VA-CAG as Induction Therapy in Newly Diagnosed AML Patients
Acute Myeloid LeukemiaThis study aims to assess the therapeutic efficacy and safety of venetoclax in combination with azacitidine and CAG(VA-CAG) as induction regimen in newly diagnosed young patients with acute myeloid leukemia(AML).
Palbociclib or Tazemetostat in Combination With CPX-351 for the Treatment of Relapsed or Refractory...
Recurrent Acute Myeloid LeukemiaRefractory Acute Myeloid LeukemiaThis phase I trial tests the safety, side effects, and best dose of palbociclib or tazemetostat in combination with CPX-351 in treating patients with acute myeloid leukemia (AML) that has come back (relapsed) or does not respond to treatment (refractory). CPX-351 is a combination of the chemotherapy drugs, daunorubicin and cytarabine, which is the standard of care for AML. Chemotherapy drugs 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. Palbociclib and tazemetostat are enzyme inhibitor drugs that are approved for treating certain cancers but not AML. These drugs may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving CPX-351 chemotherapy with enzyme inhibitors palbociclib or tazemetostat may kill more cancer cells.
Combined Evaluation of Epigenetic and Sensitising Therapy in AML and MDS
Myelodysplastic SyndromesChronic Myelomonocytic Leukemia1 moreThe goal of this project is to see if two new potential treatments (defactinib and the combination tablet of decitabine/cedazuridine) can safely be combined to improve outcomes in people with high-risk myelodysplastic syndrome (MDS), certain forms of Acute Myeloid Leukaemia (AML), and Chronic Myelomonocytic Leukaemia (CMML). Decitabine/cedazuridine is approved for use by the Australian Therapeutics Goods Administration (TGA) as treatment for MDS. Defactinib is an experimental treatment. This means it is not an approved treatment for MDS in Australia. So far it has been given to over 625 patients in studies across the world. All study participants will receive active treatment, there is no placebo. Participants will take the decitabine/cedazuridine treatment once a day for 5 days in a row (day 1 to day 5) on its own for the first month (cycle). From month 2 participants will take the decitabine/cedazuridine treatment and will also take the defactinib treatment, both for 5 days in a row on days 1 to day 5 each month (cycle). Defactinib is taken twice a day.
Treatment With ABT-199 (Venetoclax) and Purine Analogues in Relapsed/Refractory Acute Myeloid Leukemia...
Acute Myeloid Leukemiain Relapse1 moreNon-commercial, open-label interventional phase Ib study to assess the effectivity of the combination of venetoclax and 6-mercaptopurine in patients with relapsed or refractory AML.
Study of Iadademstat and Gilteritinib in Patients With R/R AML With FMS-like Tyrosine Kinase Mutation...
Acute Myeloid Leukemiain Relapse1 moreThis is an escalation/expansion, open label, multicenter study to investigate the safety and the RP2D of the combination of iadademstat with gilteritinib in FLT3-mutated R/R AML.