Myelodysplastic Syndromes

Clinical trials for patients with myelodysplastic syndromes (MDS). MDS are a group of cancers that affect the production of normal blood cells in the bone marrow.

If you would like to be considered for a clinical trial you will require a referral from your current treating team. Visit the Joining a Clinical Trial page for referral information. 

For more information on clinical trials, get in contact with our cancer clinical trials enquiry coordinator.

Clinical Trials Enquiry Coordinator

Business hours, Mon to Friday between 9am - 2pm
Email Clinical Trial Enquiries
Phone (03) 8559 7456

Open and Recruiting Clinical Trials

Study of using Cyclophosphamide After Sibling-donor allogeneic stem-cell Transplantation (CAST) in patients with acute leukaemia and myelodysplasia: a randomised study comparing cyclosporin and methotrexate to cyclosporin and post-transplantation cyclophosphamide for graft-versus-host disease prophylaxis

Trial ID

ACTRN12618000505202

Cancer type

Acute leukaemia and myelodysplasia (MDS)

Status

Open and recruiting

Phase

Three: A phase three clinical trial follows a phase two, in a larger group of patients with specific cancer types. The aim of a phase three is to compare the new treatment to existing treatments available for that cancer type.

Brief summary

A clinical trial to test if the treatment combination cyclosporin and post-transplant cyclophosphamide is better than the standard treatment given for acute leukaemia and myelodysplasia cancer to prevent graft-versus-host disease (GVHD).

The treating doctor has recommended allogeneic stem cell transplant, where stem cells given use the donor’s (sibling) immune system to destroy cancer cells. A complication of allogeneic stem cell transplant is GVHD, where the donor’s immune cells do not recognise the recipient’s body as foreign and attack the body (similar to when the immune system fights a virus / infection). The standard treatment given to prevent GVHD is the combination of cyclosporin and methotrexate (chemotherapy treatment) given following transplant. Cyclosporin is taken orally and cyclophosphamide is given as an infusion into the vein.

Patients will be randomly selected to receive one of the following treatment combinations:

Arm A: Cyclosporin and post-transplant cyclophosphamide

Arm B: Post-transplant cyclosporin and methotrexate

Who can participate

Patients who:

  • Are between 18 and 70 years old
  • Have acute leukaemia and myelodysplasia

Clinical trials can have restrictive criteria of who can and can’t participate, talk to your doctor if you are interested in this clinical trial.

For full inclusion / exclusion criteria refer to the Australian Cancer Clinical Trial Registry.

A Randomized, Double-blind MulticenterStudy Comparing Magrolimabin  Combination with Azacitidine versus Azacitidine Plus Placebo in Treatment-naïve Patients with Higher Risk Myelodysplastic Syndrome

Trial ID

NCT04313881

Cancer type

High risk myelodysplastic syndrome

Status

Open and recruiting

Phase

Three: A phase three clinical trial follows a phase two, in a larger group of patients with specific cancer types. The aim of a phase three is to compare the new treatment to existing treatments available for that cancer type.

Brief summary

A clinical trial to test if the treatment Magrolimab given in combination with Azacitadine is safe and better than Azacitadine alone in patients with high-risk myelodysplastic syndrome (MSD).

MDS is a group of cancers that affect the production of normal blood cells in the bone marrow. High-risk MDS is a severe form of MDS where increased number of blast cells (immature white blood cells not normally visible in healthy blood) don’t grow into normal red cells, white cells and platelets, causing low blood count. This can lead to anaemia (low red cell count) and can affect the body’s ability to fight infection and clot blood.

Magrolimab is a type of treatment called monoclonal antibody which targets the CD47 protein. A monoclonal antibody is a type of antibody (protein made by the body as part of the immune response to a foreign substance) produced in a laboratory designed to bind specifically to cancer cells. CD47 is a protein which helps protect cancer cells from being attacked by the immune system. Magrolimab works to stop the CD47 protein from protecting cancer cells and help the immune system to identify and kill cancer cells. Magrolimab is given in liquid by injection into the vein.

Patients will be randomly selected to receive either Magrolimab given with Azacitadine or placebo (pill/liquid that contains no active treatment) given with Azacitadine. Azacitadine is given in liquid form by small injection under the skin.

Who can participate

Patients who:

  • Have high-risk myelodysplastic syndrome
  • Have not been treated for higher risk myelodysplastic syndrome

Clinical trials can have restrictive criteria of who can and can’t participate, talk to your doctor if you are interested in this clinical trial.

For full inclusion / exclusion criteria refer to the Australian Cancer Clinical Trial Registry.

A phase 3, multicenter, double-blind, randomized, placebo-controlled study of ivosidenib or enasidenib in combination with induction therapy and consolidation therapy followed by maintenance therapy in patients with newly diagnosed acute myeloid leukemia or myelodysplastic syndrome with excess blasts-2, with an IDH1 or IDH2 mutation, respectively, eligible for intensive chemotherapy

Trial ID

NCT03839771

Cancer type

Acute myeloid leukemia or myelodysplastic syndrome

Status

Open and recruiting

Phase

Three: A phase three clinical trial follows a phase two, in a larger group of patients with specific cancer types. The aim of a phase three is to compare the new treatment to existing treatments available for that cancer type.

Brief summary

A clinical trial to test if the treatment Ivosidenib or Enasidenib in combination with chemotherapy is safe and better than the standard treatment given for acute myeloid leukemia or myelodysplastic syndrome.

Patients with excess blast cells (immature white blood cells not normally visible in healthy blood) and specific changes in the genes of their cancers (called IDH1 and IDH2) are eligible for this clinical trial. Changes can help the growth of cancer cells and are seen in approximately 20% of patients with newly diagnosed acute myeloid leukemia. Ivosidenib and Enasidenib work by stopping IDH1 and IDH2 changes to reduce the growth of cancer. Ivosidenib and Enasidenib are given in tablet form by mouth.

Patients with IDH1 changes will be randomly selected to receive either:

  • Ivosidenib given with chemotherapy
  • Placebo (pill/liquid that contains no active treatment) given with chemotherapy

Patients with IDH2 changes will be randomly selected to receive either:

  • Ivosidenib given with chemotherapy
  • Enasidenib given with chemotherapy
Who can participate

Patients who:

  • Have excess blast cells (immature white blood cells not normally visible in healthy blood)
  • Have IDH1 or IDH2 changes

Clinical trials can have restrictive criteria of who can and can’t participate, talk to your doctor if you are interested in this clinical trial.

For full inclusion / exclusion criteria refer to the Australian Cancer Clinical Trial Registry.

A Phase 1b Open-label Study Investigating the Safety, Tolerability, Pharmacokinetics, and Efficacy of Administration of Blinatumomab in Combination With AMG 404 for the Treatment of Adults With Relapsed or Refractory B cell Precursor Acute Lymphoblastic Leukemia (ALL)

Trial ID

NCT04524455

Cancer type

Acute lymphoblastic leukemia

Status

Open and recruiting

Phase

One: A phase one clinical trial tests new treatments sometimes for the first time in humans, usually in a small group of patients. The aim of a phase one clinical trial is to test the safety of the new treatment and find the best dose to give patients.

Brief summary

A clinical trial to test the safety and effectiveness of AMG 404 given in combination with Blinatumomab in patients with B cell precursor acute lymphoblastic leukemia that has not responded to or come back after treatment given.

B cell precursor is a fast-growing type of leukemia, with too many B-cell blast cells (immature white blood cells) are found in the bone marrow and blood. It is the most common type of acute lymphoblastic leukemia.

Blinatumomab is a type of treatment called immunotherapy, which works with your immune system to help destroy cancer cells or stop them growing. AMG 404 is a type of treatment called monoclonal antibody. A monoclonal antibody is a type of antibody (protein made by the body as part of the immune response to a foreign substance) produced in a laboratory designed to bind specifically to cancer cells. When used in anti-cancer treatments, the monoclonal antibody delivers the treatment directly to cancer cells. The combination of AMG 404 is expected to work together to increase the treatment outcome of Blinatumomab. Blinatumomab is given in liquid form by injection into the vein over several days using a small pump that can be used at home, as well as in the hospital. AMG 404 in liquid form by injection into the vein.

The clinical trial has two parts, the first will find the best dose of AMG 404 given in combination with Blinatumomab and the second will test the combination in a larger group of patients to further test the safety and effectiveness of the treatment.

Who can participate

Patients who:

  • Have B cell precursor acute lymphoblastic leukemia
  • Have cancer that has not responded to or come back after treatment given

Clinical trials can have restrictive criteria of who can and can’t participate, talk to your doctor if you are interested in this clinical trial.

For full inclusion / exclusion criteria refer to the Australian Cancer Clinical Trial Registry.