Assessment of Effectiveness and Safety of Luspatercept in Patients Suffering From Lower-risk Myelodysplastic Syndrome.
This study is testing a new medicine called Luspatercept for people with a specific type of blood disorder called 'lower-risk Myelodysplastic Syndromes' (MDS) who have ring sideroblasts. This condition can cause anaemia, meaning your body doesn't have enough healthy red blood cells. The study aims to understand how effective and safe Luspatercept is for these patients, especially if they need regular blood transfusions or haven't responded well to other common treatments. It will involve about 70 patients across several European countries, and scientists will closely monitor their health and response to the medication. This is a Phase 3 study, meaning it's one of the final stages of testing before a medicine might become widely available.
At a glance
What is this study about?
This research study is about a new treatment called Luspatercept for a condition called Myelodysplastic Syndromes (MDS). MDS is a group of blood disorders where the bone marrow (the spongy tissue inside your bones) doesn't make enough healthy blood cells. This particular study is focusing on a specific type of lower-risk MDS where patients often have anaemia, meaning they have too few red blood cells, and a feature called 'ring sideroblasts'.
The main goal of the study is to find out if Luspatercept can help these patients. Researchers want to see how well it works at improving their blood cell counts and reducing their need for blood transfusions. They also want to carefully check for any side effects and understand how safe the medicine is. This is important because current treatments don't work for everyone, and new options are always needed.
This is a 'Phase 3' study, which means the medicine has already been through earlier tests and shown some promise. Now, it's being given to a larger group of patients to get more clear information on its effects. The study also aims to gather more details about patients who haven't responded to other treatments like Erythropoiesis-Stimulating Agents (ESAs) or other common MDS medications.
Key takeaways
- Tests a new medicine, Luspatercept, for a type of MDS.
- Aimed at patients with anaemia and 'ring sideroblasts'.
- Focuses on those who haven't responded to other treatments.
- It's a advanced-stage study (Phase 3) for safety and effectiveness.
- Participation involves screening, treatment, and follow-up periods.
Who may be eligible?
To be considered for this study, you need to be at least 18 years old. You must have been diagnosed with a specific type of Myelodysplastic Syndrome (MDS) that doctors consider 'low-risk' or 'intermediate-risk', and have at least 15% 'ring sideroblasts' in your bone marrow (or 5% if you have a specific gene change called SF3B1).
Additionally, your current blood counts need to be within certain limits, for example, your bone marrow should have less than 5% immature blood cells (blasts), and your white blood cell count in your blood should not be too high. A key part of qualifying is that you've either not responded well to, couldn't tolerate, or weren't suitable for common treatments like ESAs (medicines that help your body make red blood cells).
If you have previously taken ESAs or other specific MDS treatments, there are rules about how long you must have stopped them before you can join this study. The medical team will check all these details carefully to see if this study is the right fit for you.
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?
- Have you been diagnosed with 'lower-risk' MDS with 'ring sideroblasts'?
- Have other common treatments for anaemia (like ESAs) not worked for you, or were you unable to tolerate them?
- Are you willing to participate in regular doctor visits and assessments?
What does participation involve?
If you decide to take part, the study will involve several stages. First, there's a 'screening period' where doctors will do tests to make sure you meet all the requirements. If you do, you'll then move into the 'treatment period'. This is split into a main part, where you'll receive the Luspatercept medicine, followed by an 'extension phase' where you might continue to receive it. After you finish the treatment, there will be a 'post-treatment follow-up period' where the doctors will continue to monitor your health. The total duration of your involvement could vary depending on how you respond to the treatment and the extension phase.
Potential risks and benefits
Locations (13)
- Medizinische Universität InnsbruckVerified postcodeInnsbruck, Austria
- Medizinische Universität WienVerified postcodeVienna, Austria
- Universitätsklinikum LeipzigVerified postcodeLeipzig, Germany
- Klinikum rechts der IsarVerified postcodeMünchen, Germany
- Institut Català d' Oncologia de BadalonaVerified postcodeBadalona, Spain
- Hospital Vall d´HebronVerified postcodeBarcelona, Spain
- Hospital General Universitario Gregorio MarañónVerified postcodeMadrid, Spain
- Hospital Universitario Central de AsturiasVerified postcodeOviedo, Spain
- University Hospital of SalamancaVerified postcodeSalamanca, Spain
- Hospital Universitario y Politecnico La Fe de ValenciaVerified postcodeValencia, Spain
- Universitätsspital BaselVerified postcodeBasel, Switzerland
- Clinica di Ematologia Istituto oncologico della Svizzera ItalianaVerified postcodeBellinzona, Switzerland
Common questions
What is Myelodysplastic Syndrome (MDS)?
MDS is a type of blood disorder where your bone marrow doesn't produce enough healthy blood cells, leading to problems like anaemia.
What is Luspatercept?
Luspatercept is an experimental medicine being tested to see if it can help improve red blood cell production in people with certain blood conditions like MDS.
What does 'lower-risk' MDS mean?
This means your MDS is not currently considered aggressive, but it can still cause symptoms like anaemia and may need treatment.
Will I get regular blood transfusions if I join?
The study aims to see if Luspatercept can reduce your need for blood transfusions, but you may still need them depending on your individual response and blood counts.
Where is this study taking place?
This study is happening in several countries including Germany, France, Austria, and Switzerland.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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