Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

GFM-EPO-PRETAR: A randomized trial testing early versus late onset of EPO alfa treatment in lower risk MDS with non RBC transfusion dependent anemia and without del 5q

This study is for adults with a type of blood condition called Myelodysplastic Syndromes (MDS). Specifically, it's for those with a lower risk form of MDS who don't currently need regular blood transfusions for their anaemia. The main goal is to find out if starting EPO alfa, a common treatment, early on is better than waiting to start it until blood transfusions become necessary. Researchers will compare how long it takes for people to become dependent on blood transfusions in both groups. This research hopes to improve how we manage anaemia in people with lower-risk MDS, potentially helping them avoid or delay the need for transfusions, which can impact their quality of life.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Groupe Francophone Des Myelodysplasies

Enrolment target

124

Start

24 Sep 2024

Adult subject (18 years of age older) with low or intermediate-1 IPSS risk MDS with transfusions independent anemia

What is this study about?

You might have heard of Myelodysplastic Syndromes, or MDS. It's a group of conditions where your bone marrow, which is like the factory for your blood cells, doesn't produce enough healthy blood cells. One common problem in MDS is anaemia, where you don't have enough red blood cells to carry oxygen around your body, making you feel tired and weak.

This study is looking at a medicine called EPO alfa. This medicine helps your body make more red blood cells. Currently, doctors sometimes wait until a person with MDS starts needing blood transfusions often before they begin giving EPO alfa. This study wants to find out if starting EPO alfa treatment earlier, even before someone needs transfusions, might be better.

The idea is to see if starting EPO alfa earlier can delay or even prevent people from needing regular blood transfusions. Blood transfusions can be life-saving, but they also mean regular hospital visits and can have their own set of challenges. By understanding the best time to start this treatment, doctors hope to improve the lives of people with lower-risk MDS by helping them feel better and potentially reducing their need for transfusions.

Key takeaways

Study is comparing early vs. late start of EPO alfa for lower-risk MDS.
Aims to see if early treatment delays need for blood transfusions.
Participants must have lower-risk MDS and not currently need regular transfusions.
EPO alfa helps your body make more red blood cells.
You will be closely monitored if you choose to participate.

Who may be eligible?

This study is for adults who are 18 years old or older. You need to have a specific type of MDS, which doctors call 'lower-risk' or 'intermediate-1 risk' MDS. This is decided by a special scoring system called IPSS.

More importantly, you should also have anaemia (low red blood cells) but not currently be dependent on regular blood transfusions. This means you aren't regularly needing to receive donor blood to manage your anaemia.

Finally, your MDS should not involve a specific genetic change called 'del 5q'. Your doctor will be able to tell you if you meet all these specific requirements 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 lower-risk or intermediate-1 risk MDS?
Do you have anaemia but are not currently reliant on regular blood transfusions?
Has your doctor confirmed you do not have 'del 5q' in your MDS?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be randomly assigned to one of two groups, like flipping a coin. One group will start receiving the EPO alfa medicine right away. The other group will delay starting the medicine until they reach a point where blood transfusions become necessary.

Both groups will be closely monitored by doctors and nurses. This will involve regular visits to the clinic for check-ups, blood tests, and to assess how you are feeling and if the treatment is working. The EPO alfa medicine is given as an injection, usually under the skin. The study will track how long it takes for a person to start needing regular blood transfusions and also look at how well the treatment helps your body make red blood cells, how it affects your quality of life, and your overall health. The total duration of participation in the trial will depend on your individual response and clinical needs.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A potential benefit of this study is that starting EPO alfa earlier might help delay or reduce your need for blood transfusions, potentially improving your energy levels and overall quality of life. However, like all medicines, EPO alfa can have side effects, and your doctor will discuss these with you. There's also a chance the treatment might not work for you, or that waiting to start treatment might mean you become transfusion-dependent sooner. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
France

Common questions

What is EPO alfa and how is it given?

EPO alfa is a medicine that helps your body produce more red blood cells. It is given as an injection, usually under the skin, often in a pre-filled syringe.

What does 'lower-risk MDS' mean?

Lower-risk MDS means your condition is generally less aggressive and may progress more slowly compared to higher-risk types. Your doctor uses a special scoring system to determine your risk level.

What does 'transfusion independent' mean?

It means you are not currently needing regular blood transfusions to manage your anaemia. You might occasionally have a transfusion, but not on a routine basis.

Will I know which group I'm in?

No, this is a 'randomized' study, meaning you will be assigned to a group by chance. This helps ensure the study results are fair and unbiased.

What happens if my condition changes during the study?

Your health will be closely monitored, and your doctors will adjust your treatment as needed, always prioritising your well-being. You can also withdraw at any time.