A Study Evaluating Venetoclax in Combination With Azacitidine in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)
This research study is investigating a new treatment approach for people diagnosed with higher-risk myelodysplastic syndromes (MDS) who haven't received treatment for it yet. MDS is a condition where your bone marrow doesn't make enough healthy blood cells. The study will combine two medicines, venetoclax and azacitidine, which are thought to work together to target the disease. This is a very early stage study, called Phase 1, meaning it's one of the first times this specific combination is being given to people. The main goals are to check if the treatment is safe, understand how the body reacts to it, and see if there are any early signs that it could be helpful for patients. Participants will be closely monitored by a medical team.
At a glance
What is this study about?
You might be reading this because you or someone you know has been diagnosed with myelodysplastic syndromes (MDS), especially the higher-risk form, and you're looking into treatment options. MDS is a condition where your body's bone marrow, which is like the factory for making blood cells, doesn't work properly. This leads to a shortage of healthy red blood cells, white blood cells, and platelets. Higher-risk MDS means the condition might progress more quickly or affect your health more severely.
This particular study is a clinical trial, which means it's a carefully planned medical research study involving people. Its main aim is to test a new combination of two medicines: venetoclax and azacitidine. These medicines are already used to treat certain blood cancers, but this study wants to see how they work together specifically for higher-risk MDS in people who haven't had treatment before. The hope is that by combining them, they might be more effective at tackling the disease.
Because this is a very early stage, called a Phase 1 study, the main focus is on safety. Doctors will be looking very closely at whether the treatment is well-tolerated and what potential side effects might occur. They also want to understand the best dose of the medicines and how your body processes them. If this early study shows promising results, it could pave the way for bigger studies in the future, ultimately aiming to find better treatments for MDS patients.
Key takeaways
- This study is for adults with higher-risk MDS who haven't had previous treatment.
- It's testing a new combination of two medicines: venetoclax and azacitidine.
- This is an early-stage (Phase 1) study focused on safety and understanding the medicines.
- Participants will receive the study medications and have regular health checks.
- You can stop participating at any time if you wish.
- The goal is to find better future treatments for MDS.
Who may be eligible?
This study is looking for adult volunteers, aged 18 years or older, regardless of whether they are male or female. You would need to have been recently diagnosed with higher-risk myelodysplastic syndromes (MDS) and not have started any specific treatment for it yet.
The research team will need to check your general health, medical history, and specific details about your MDS to make sure this study is suitable for you. There will be certain medical conditions or treatments that might mean you can't join, as this is important for your safety and to make sure the study results are clear.
The medical team will carefully review all your information to determine if you meet all the necessary requirements to participate. They will discuss this in detail with you if you are interested.
- Are you 18 years old or older?
- Have you been diagnosed with higher-risk myelodysplastic syndromes (MDS)?
- Have you NOT received any prior treatment specifically for your MDS?
- Are you willing to attend regular clinic visits for treatment and monitoring?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to participate, the study team will first explain everything in detail and answer all your questions. If you agree, you'll sign an informed consent form. You'll then undergo a series of health checks, which may include blood tests, bone marrow biopsies, and other examinations to make sure you're suitable for the study.
The treatment will involve receiving the study medications, venetoclax and azacitidine. The azacitidine is usually given as an injection, and venetoclax is usually taken as a pill. You will have regular visits to the clinic for treatments, monitoring, and assessments. These visits will involve physical examinations, blood tests, and discussions about how you're feeling and any side effects you might be experiencing. The exact schedule of visits and treatments will be explained to you in detail.
The total duration of your participation will depend on how you respond to the treatment and the study plan. Even after treatment finishes, there will be follow-up appointments to monitor your health. The medical team will guide you through every step, ensuring you understand what is expected and providing support throughout your involvement in the study.
Potential risks and benefits
Locations (2)
- —Germany
- —France
Common questions
What is MDS?
MDS stands for Myelodysplastic Syndromes. It's a condition where your bone marrow doesn't make enough healthy blood cells, which can lead to low blood counts and other problems.
What does 'higher-risk' MDS mean?
Higher-risk MDS means your condition might progress more quickly or be more serious compared to lower-risk forms of MDS.
What is a Phase 1 study?
A Phase 1 study is the first stage of testing a new treatment in humans. Its main goal is to find out if the treatment is safe and what dose can be given safely, rather than how well it works.
Will I get the actual study medication?
Yes, in this type of study, all participants who meet the criteria will receive the new combination treatment of venetoclax and azacitidine. There isn't a placebo group in this early phase.
Can I leave the study if I change my mind?
Yes, you can withdraw from the study at any time, for any reason, without it affecting your current or future medical care. Your decision will be respected.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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