Research study for adults with anemia due to MDS is seeking participants

Research study for adults with anemia due to MDS is seeking participants

Myelodysplastic Syndrome (MDS) is a group of diverse bone marrow disorders in which the bone marrow does not produce enough healthy blood cells. Therefore, myelodysplastic syndrome is often referred to as a “bone marrow failure disorder”. In healthy people, the bone marrow functions as a factory that manufactures three kinds of blood cells: red blood cells, white blood cells, and platelets. Healthy bone marrow produces immature blood cells – called stem cells, that normally develop into red blood cells, white blood cells, and platelets. In myelodysplastic syndrome, these stem cells may not mature, or they may have a shortened life span, resulting in fewer than normal blood cells in circulation. Due to this, patients with myelodysplastic syndrome often suffer from anemia (low blood cell counts) and may require red blood cell transfusions.

If you or any of your loved ones suffer from anemia due to myelodysplastic syndrome, you may be eligible to participate in a research study.


Disease information

Myelodysplastic Syndrome (MDS) is a group of bone marrow disorders, where immature blood cells (stem cells) in the bone marrow do not mature the right way. It is a disease that is primarily affecting the elderly (older than 65), but younger people may also get the disease. The cause of the disease is often unknown. Anemia (low blood cell counts) is a hallmark feature of myelodysplastic syndrome and is responsible for some of the symptoms that patients experience, such as infections, spontaneous bleeding or easy bruising, fatigue, shortness of breath and pale skin.

Patient with myelodysplastic syndrome can be categorized into 1 of 5 risk groups depending on the severity of their disease (IPSS-R classification).

The different risk groups are:

  • Very low-risk
  • Low-risk
  • Intermediate-risk
  • High-risk
  • Very high-risk

The risk level describes the risk of disease progression to acute myeloid leukemia (AML), which is one of most the common types of blood and bone marrow cancer.

The current treatment for lower-risk myelodysplastic syndrome is:

  • Supportive treatment to enhance production of red blood cells (erythropoiesis stimulating agents - ESAs)
  • Blood transfusion (red blood cells transfusion and/or platelet transfusions)
  • Treatment of infection


Therapeutic background

People suffering from myelodysplastic syndrome often experience clinical symptoms such as anemia (a decrease in red blood cells and hemoglobin, which may cause fatigue and tiredness) and other abnormal laboratory results (e.g., low white blood cell and/or platelet counts). This often results in the need for blood transfusions, a higher risk for developing infections and an increased risk of developing acute myeloid leukemia (AML). Being dependent on blood transfusions is associated with iron overload, and with a negative impact on your quality of life, thus the need for other treatments.

If you are interested and qualify to participate in a research study, you will receive 1 out of the 2 types of medications, i.e., luspatercept or epoetin alfa. Luspatercept is the investigational medication to help anemia, while epoetin alfa is the current standard treatment. Luspatercept has recently been approved in some countries for the treatment of anemia due to MDS in adults who require regular blood transfusions and have not responded well to or cannot receive an erythropoiesis stimulating agent (ESA). However, its use in this study is investigational. The study is an open-label study, meaning the study participant, the study team and your doctor will know which medication you will receive. The study team will also provide you with information about study-related procedures.

Please note that by participating in a research study, regardless of which two treatments you receive, you may contribute to the clinical research and further understanding of the treatments for MDS. However, you have the right to withdraw from the research study at any time and the study team will follow you up as appropriate.


Who can participate?

You may participate in this research study if you meet all the following criteria:

  • Male or female
  • Age 18 or above
  • Have anemia due to bone marrow failure disorder (Myelodysplastic Syndrome (MDS))
  • Receive red blood cell transfusions




  • Concord
  • Adelaide
  • Malvern
  • Clayton
  • West Perth
  • Wodonga
  • Wollongong
  • Auchenflower
  • Melbourne
  • Newcastle
  • Randwick
  • Blacktown


  • Ottawa
  • Toronto
  • Hamilton
  • Montréal
  • Edmonton
  • Calgary


  • Whittier
  • Paducah
  • New Haven
  • Greenville
  • Tampa
  • Houston
  • Hackensack
  • San Diego
  • Saint Petersburg
  • Tallahassee
  • Bethesda
  • Newport News
  • Portland
  • Charlottesville

United Kingdom:

  • Manchester
  • Aberdeen
  • Bournemouth
  • London
  • Oxford
  • Boston



There will be no additional costs to you if you choose to participate in this research study. The investigational medication and any tests required by the study are provided by the study team, and you will receive reimbursement for travel expenses incurred by taking part in the research study until your participation is complete.

Please note that taxes may need to be paid on travel expense reimbursement; it might also need to be reported to authorities.



The research study assesses the safety and efficacy of an investigational medication in the treatment of anemia due to very low, low or intermediate risk of myelodysplastic syndromes (MDS). This means that the main purpose of this study is to see if people with your type of anemia due to MDS will experience a lesser need for regular blood transfusions. If you are interested and qualify to participate, the study team will provide you a detailed overview of the study procedures before you agree to participate. The study team will also take the time to review and discuss any questions you may have to ensure you are comfortable participating.


The following are typical examples of participation in a research study related to anemia:

Screening period

After receiving full verbal and written information about the research study and discussing the study procedures with the study team, and signing the informed consent form, you will enter the screening period. This period is conducted to confirm your eligibility into the research project based on safety and clinical assessments. For the screening session you will have to visit a site up to 35 days before you can receive a study medication and will perform some tests (e.g., physical examination, blood tests, urinalysis, bone marrow and pregnancy tests etc.). If you are eligible after screening you will enter the treatment period.


Treatment period

The treatment period usually consists of at least 24 weeks. After 24 weeks of treatment, your study doctor will determine if you can continue the study treatment by confirming you are receiving benefit from the study treatment and your MDS disease has not worsened. Additional assessments to check if you are benefiting from the study treatment will be performed every 24 weeks.

The dose frequency will depend on which of the two treatments you will be receiving. You will receive the medication either every week or every 3 weeks. The study team will provide you with more information about the treatment you will be receiving.

Your health will be closely monitored by the study team throughout your participation in the research study. You can withdraw your participation at any time without providing a reason.


Post treatment follow-up period

This period consists of 6 weeks of safety follow-up. After this period the study team will follow you either for 5 years from the date of the first dose or 3 years from the last dose of the study medication. During this period the visits take place telephonically from the site and will be conducted every 12 weeks for the first 3 years from the last given dose of the study medication, and every 6 months thereafter, if applicable.



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