Not yet recruitingPHASE2INTERVENTIONAL

Evaluation of Treosulfan Versus Melphalan Conditioning Followed by PTCy in Patients With AML and MDS Undergoing Allogeneic Transplantation

This study compares two chemotherapy drugs, Treosulfan and Melphalan, which are used to prepare patients with certain blood cancers (Acute Myeloid Leukaemia and Myelodysplastic Syndrome) for a stem cell transplant. Both drugs are given alongside another drug called Fludarabine. The main goal is to find out which combination is more effective at treating the cancer and also easier for patients to tolerate. Researchers will carefully check how long patients live, if their cancer comes back, how well the new stem cells settle in (engraftment), and if they develop a common complication called Graft versus Host Disease (GvHD). This information will help doctors choose the best treatment for future patients.

At a glance

Status

Not yet recruiting

Phase

PHASE2

Sponsor

Technische Universität Dresden

Enrolment target

220

Start

01 Sep 2025

Estimated completion

01 Dec 2028

AML - Acute Myeloid Leukemia MDS (Myelodysplastic Syndrome)

What is this study about?

This research study is for people who have specific types of blood cancer called Acute Myeloid Leukaemia (AML) or Myelodysplastic Syndrome (MDS) and are planning to have a stem cell transplant. Before a stem cell transplant, patients usually receive strong chemotherapy to clear out their existing bone marrow cells. This makes space for the new, healthy stem cells. This process is called 'conditioning'.

In this study, we are comparing two different chemotherapy mixtures for this conditioning step. One mixture uses a drug called Treosulfan, and the other uses a drug called Melphalan. Both mixtures also include another drug called Fludarabine. We want to see which of these two combinations works better at getting rid of the cancer cells and preparing the body for the new stem cells, while also being as gentle as possible on the patient.

We will be looking at several important things. We want to understand if one treatment helps people live longer, if it prevents their cancer from coming back, and if the new stem cells successfully start working in the body. We'll also be watching for a potential side effect called Graft versus Host Disease (GvHD), where the donor's cells might see the patient's body as foreign. By carefully comparing these two treatments, we hope to improve the standard care for people needing a stem cell transplant for AML or MDS.

Key takeaways

This study compares two chemotherapy drugs for stem cell transplants in AML/MDS.
It aims to find out which treatment is safer and more effective.
Researchers will track survival, cancer return, and potential side effects.
Participation involves receiving one of two tested chemotherapy combinations.
Eligible patients must be adults with AML/MDS needing a transplant and a suitable donor.

Who may be eligible?

To join this study, you must be an adult aged 18 or over and scheduled for a stem cell transplant within the next three weeks for AML or MDS. Your doctor will need to confirm your specific type of AML or MDS meets the study's requirements. The study focuses on patients who might have a higher risk of side effects from very strong chemotherapy, for example, if you are aged 50 or over, or have certain health conditions that your doctor will check. You also need to have a suitable stem cell donor, which could be a family member or an unrelated donor.

There are also some reasons why you might not be able to join. For example, if you have a specific type of AML called acute promyelocytic leukaemia, or if you've already had a stem cell transplant in the last year and it didn't work. You also can't have received Melphalan or Treosulfan specifically in the last year. If you have severe problems with your liver, or need extra oxygen to breathe regularly, you might also not be able to take part.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have AML or MDS and are scheduled for a stem cell transplant soon?
Do you have a suitable stem cell donor?
Are you generally well enough for a stem cell transplant, without severe heart or liver problems?
Have you NOT had Treosulfan or Melphalan as a treatment in the last year?
Are you NOT dependent on oxygen for breathing?

Answer every question to see your result.

What does participation involve?

As part of this study, you will receive one of the two chemotherapy conditioning treatments (Treosulfan or Melphalan, plus Fludarabine) before your stem cell transplant. You will also receive a standard medication called PTCy to help prevent Graft versus Host Disease. During your treatment and afterwards, the study team will closely monitor your health through regular blood tests, check-ups, and scans. They will be looking at how well the new stem cells are settling in, if your cancer remains in remission, and if you experience any side effects. The exact number of visits and the full length of follow-up will be discussed by the study team, but generally, monitoring continues for some time after the transplant to ensure your long-term health and to track the study's outcomes.

Potential risks and benefits

Taking part in this study may offer a potential benefit by receiving one of two established chemotherapy treatments under close medical supervision, which could lead to a better outcome for your condition. However, as with any medical treatment, there are potential risks, including side effects from chemotherapy and the stem cell transplant itself, such as infection, fatigue, and Graft versus Host Disease. The study team will explain these risks in detail. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (4)

Medizinische Fakultät der TU Dresden, Medizinische Klinik und Poliklinik I
Verified postcode
Dresden, Germany
Universitätsmedizin Halle (Saale)
Verified postcode
Halle, Germany
Universitätsklinikum Schleswig-Holstein, Campus Kiel
Verified postcode
Kiel, Germany
Universitätsklinikum Münster, Medizinische Klinik A, KMT-Zentrum
Verified postcode
Münster, Germany

Common questions

What is 'conditioning' before a stem cell transplant?

Conditioning is the chemotherapy given to you before your stem cell transplant. It helps to clear out your existing bone marrow and prepare your body for the new, healthy stem cells.

What is Graft versus Host Disease (GvHD)?

GvHD is a complication where the new stem cells from the donor see your body as 'foreign' and attack it. Medications are given to try and prevent this.

What does 'engraftment' mean?

Engraftment means that the new, healthy stem cells have successfully settled in your body and started producing new blood cells. This is a very important step after a transplant.

Can I choose which treatment I receive?

No, if you join this study, you will be randomly assigned to receive either the Treosulfan or Melphalan treatment. This is like flipping a coin and helps ensure the study results are fair.

What if my cancer comes back?

The study aims to measure how well the treatments prevent cancer relapse. Your medical team will continue to provide care and discuss treatment options if your cancer does return.

How to find out more

Prof. Friedrich Stölzel, MD

etal-5@ukdd.de +49 431 500-22701 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.