A Randomized, Open-Label, Phase 1-2 Study of ASTX727 Low Dose (ASTX727 LD) Extended Schedule in Subjects with Lower Risk (IPSS Low or Intermediate-1) Myelodysplastic Syndromes (MDS)
This research trial is looking at a new medicine, ASTX727, for people diagnosed with myelodysplastic syndromes (MDS) that are considered lower risk. MDS is a condition where the bone marrow, the spongy tissue inside bones, doesn't make enough healthy blood cells. The study has a couple of main goals. First, it wants to find out how safe ASTX727 is and if it causes any serious side effects. Second, it will check if the drug helps improve blood cell numbers, which is called a hematologic response. Researchers are also comparing ASTX727 with another treatment called Decitabine, which is similar. This study involves adults and is done in phases to carefully assess the new treatment.
At a glance
What is this study about?
This study is investigating a new medication called ASTX727 for adults who have a specific type of blood disorder called myelodysplastic syndromes (MDS). MDS means that your body's bone marrow isn't producing enough healthy blood cells, which can lead to problems like anaemia (low red blood cells), frequent infections (due to low white blood cells), or easy bruising and bleeding (due to low platelets). The 'lower risk' part means that your condition is not considered to be as aggressive as some other forms of MDS.
The main purpose of this research is to see how safe the new drug, ASTX727, is and to understand any side effects it might cause. Researchers will also be looking closely at whether the treatment helps improve your blood counts, such as increasing healthy red blood cells, white blood cells, or platelets. This improvement in blood counts is known as a 'hematologic response'. The study is designed to carefully observe how people react to the medication.
This trial is being conducted in two parts, or 'phases'. The first phase is mainly about safety, trying to find the right dose while checking for any serious side effects. Once a safe dose is identified, the second phase will focus more on how well the drug works to improve blood counts. Comparing it to Decitabine, another similar medication, helps researchers understand the potential benefits and how it stacks up against existing treatments.
Key takeaways
- This study is testing a new drug, ASTX727, for lower-risk myelodysplastic syndromes (MDS).
- It aims to check the safety of the drug and if it improves blood counts.
- Participation involves regular clinic visits, tests, and medical monitoring.
- You must be an adult with lower-risk MDS (IPSS Low or Intermediate-1) to be considered.
- You can discuss this study with your doctor to see if it's right for you.
Who may be eligible?
To be considered for this study, you need to be an adult, 18 years old or older. The study is open to both men and women. You must have a diagnosis of lower-risk myelodysplastic syndromes (MDS).
The term 'lower risk' is determined by a scoring system called IPSS (International Prognostic Scoring System), which your doctor will be familiar with. This means your MDS is categorised as 'Low' or 'Intermediate-1'. Your doctor will be able to confirm if your specific diagnosis fits these criteria. This study is not for people with more aggressive types of MDS.
There will also be other specific medical checks and criteria that the research team will review to ensure the study is safe and appropriate for you. These can include things like your general health, how well your liver and kidneys are working, and if you are taking certain other medications. All these checks are in place to protect your safety during the trial.
- Are you 18 years old or older?
- Do you have a diagnosis of myelodysplastic syndromes (MDS)?
- Has your doctor told you your MDS is 'lower risk' (IPSS Low or Intermediate-1)?
- Are you able to attend regular clinic appointments and undergo medical tests?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, you would first go through a screening process to make sure you meet all the necessary health requirements. During the study, you would receive the study medication, ASTX727, possibly at different doses, or another treatment called Decitabine, depending on which group you are assigned to. This would involve regular visits to the clinic for medical check-ups, blood tests, and other assessments like bone marrow examinations. These tests are important for the research team to monitor your health, how well the treatment is working, and to identify any side effects. The exact number of visits, frequency of tests, and the total length of your participation would be explained in detail by the study team, as it can vary.
Potential risks and benefits
Locations (3)
- —Spain
- —Belgium
- —Germany
Common questions
What is MDS?
MDS, or myelodysplastic syndromes, is a condition where your bone marrow doesn't make enough healthy blood cells. This can lead to problems like anaemia.
What is ASTX727?
ASTX727 is a new medicine being tested in this study to see if it can help people with lower-risk MDS.
Why is this study being done?
This study aims to find out how safe ASTX727 is and if it helps improve blood counts in people with lower-risk MDS.
Will I know if I'm getting ASTX727 or another treatment?
This is an 'open-label' study, which means both you and your doctor will know which treatment you are receiving.
What does 'lower-risk MDS' mean?
It means your MDS is not considered to be as aggressive as some other forms, based on specific medical assessments.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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