Ongoing Clinical Trials for Myelodysplastic Syndrome Transformation
There are currently 2 clinical trials exploring new approaches to prevent or treat conditions related to myelodysplastic syndrome transformation. These studies are testing different medications including metformin, S227928, and venetoclax, and are being conducted across several European countries including Denmark, Finland, France, and Germany.
Clinical trial locations
- Denmark
- Finland
- France
- Germany
Study on Metformin for Preventing Leukemia in Patients with Clonal Cytopenia and Low-Risk Myelodysplastic Syndromes
This clinical trial is taking place in Denmark and explores an innovative approach to preventing leukemia in people with early blood disorders. The study focuses on using metformin, a medication commonly prescribed for diabetes, in a completely different way: to potentially slow or stop the progression of clonal cytopenia of undetermined significance and low-risk myelodysplastic syndromes into leukemia.
Main goal: The primary purpose is to assess whether metformin is safe and practical to use in patients with these specific blood conditions. Researchers will monitor participants for up to 52 weeks to understand how the medication might control disease progression and prevent the development of leukemia.
Who can participate: To join this study, you must be at least 18 years old and diagnosed with either very low- or low-risk myelodysplastic syndromes or clonal cytopenia of undetermined significance. Women participating must be postmenopausal, meaning over 45 years old without menstruation for at least 12 months. You must be able to swallow pills, provide written consent, and be willing to follow all study requirements.
Who cannot participate: The trial excludes patients outside the specified age range and those considered part of vulnerable populations, such as children, pregnant women, or individuals unable to provide informed consent.
Investigational drug: Metformin hydrochloride is taken as an oral coated tablet. While typically used for managing blood sugar in diabetes, this study investigates whether it can activate certain cellular pathways that might slow down abnormal blood cell growth and prevent progression to leukemia.
Study of S227928 Alone and with Venetoclax for Patients with Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia
This international clinical trial is being conducted in Finland, France, and Germany. It investigates a new experimental treatment for patients whose blood cancers have returned or not responded to previous therapies. The study tests S227928, a novel antibody-drug conjugate that specifically targets cancer cells, both alone and in combination with venetoclax.
Main goal: The trial has two phases. Phase 1 focuses on finding the safest and most tolerable dose of S227928, testing it both as a single treatment and combined with venetoclax. Phase 2 evaluates how effectively the combination therapy fights the cancer. Researchers want to understand both the safety profile and the potential benefits of this new treatment approach.
Who can participate: You must be at least 18 years old with a confirmed diagnosis of acute myeloid leukemia, myelodysplastic syndrome/AML, or chronic myelomonocytic leukemia that has relapsed or not responded to treatment. Your performance status should indicate reasonable ability to carry out daily activities. You need adequate kidney and liver function, and your white blood cell count should be below a certain threshold. Most importantly, you should have received at least one previous standard treatment but are not a candidate for further standard therapy.
Who cannot participate: Patients without the specific diagnoses mentioned above, those outside the required age range, or individuals who don’t meet the health criteria cannot join the study.
Investigational drugs: S227928 is an antibody-drug conjugate administered through intravenous infusion. It works by targeting the CD74 protein on cancer cells and delivering a toxic agent directly to them. Venetoclax is taken as oral tablets and works by blocking the BCL-2 protein, which helps cancer cells survive. By inhibiting this protein, venetoclax promotes cancer cell death. The study tests whether using these medications together provides better results than S227928 alone.
Summary
These two clinical trials represent different approaches to addressing blood disorders related to myelodysplastic syndrome transformation. The Danish study takes a preventive approach, exploring whether a readily available diabetes medication might stop early-stage blood disorders from progressing to leukemia. In contrast, the multi-country European trial tests cutting-edge targeted therapies for patients with more advanced disease who have exhausted standard treatment options.
Geographically, these trials show research activity across Northern and Central Europe, with the more complex combination therapy trial spanning multiple countries. The studies reflect the spectrum of treatment needs, from early intervention to advanced disease management. Notably, one trial repurposes an existing medication, while the other explores novel experimental compounds, demonstrating the diverse strategies being pursued to improve outcomes for patients with these challenging blood disorders.
