Study on Eltrombopag for Treating Low and Intermediate Risk Myelodysplastic Syndromes in Patients with Thrombocytopenia

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What is this study about?

This clinical trial is focused on studying a condition known as myelodysplastic syndromes (MDS), which affects the blood and bone marrow, leading to low blood cell counts. The study is investigating the use of a medication called eltrombopag, also known by its code name SB 497115, to treat a specific problem associated with MDS called thrombocytopenia. Thrombocytopenia is a condition where there are fewer platelets in the blood than normal, which can lead to increased bleeding and bruising.

The purpose of the study is to evaluate how effective eltrombopag is compared to a placebo in improving platelet counts and to assess its safety and tolerability. The study is divided into two phases. In the first phase, the focus is on understanding how many patients achieve a complete or partial response to the treatment and monitoring any side effects. The second phase aims to look at how long the improvement in platelet counts lasts and to continue assessing the long-term safety of the treatment.

Participants in the study will take eltrombopag in the form of film-coated tablets, which are taken orally. The study will monitor the participants over a period of time to see how their platelet levels respond to the treatment and to ensure that the medication is safe to use. The study will also look at other factors, such as the frequency of platelet transfusions needed and any changes in the quality of life of the participants. The ultimate goal is to find a safe and effective treatment option for patients with low or intermediate-risk MDS who are experiencing thrombocytopenia.

1 initial treatment phase

The treatment involves taking eltrombopag, which is available in two forms: Revolade 50 mg and Revolade 25 mg film-coated tablets. The medication is taken orally.

The dosage and frequency of administration will be determined by the healthcare provider based on individual needs and response to the treatment.

2 monitoring and evaluation

Regular monitoring of platelet counts and overall health is conducted to assess the response to the treatment.

The primary goal is to achieve a complete response or a significant improvement in platelet levels.

3 safety and tolerability assessment

The safety and tolerability of the treatment are evaluated by monitoring any adverse events or side effects experienced during the trial.

This includes both common and serious adverse events, with a focus on maintaining patient safety.

4 long-term follow-up

The duration of the platelet response is observed over a longer period to ensure sustained benefits from the treatment.

Long-term safety and tolerability are also assessed to ensure the treatment remains suitable for continued use.

5 completion of trial

The trial is expected to conclude by October 31, 2026, with all data collected and analyzed to determine the overall effectiveness and safety of the treatment.

Who Can Join the Study?

Participants must be adults, meaning they are 18 years of age or older.
Participants should have low or intermediate-1 IPSS risk MDS and their condition should be stable. MDS stands for myelodysplastic syndromes, which are a group of disorders caused by poorly formed or dysfunctional blood cells.
Participants must use an acceptable method of contraception. Women who can have children must use a highly effective method of contraception starting 2 weeks before the study, during the study, and for 28 days after the study ends.
Participants must have a platelet count of less than 30 Gi/L within the 4 weeks before joining the study. Platelets are cells in the blood that help with clotting.
Participants must not be eligible for or have not responded to other treatments like azacitidine or lenalidomide, and they must not be eligible for intensive chemotherapy or stem cell transplantation.
Participants must have platelet count and transfusion data available for the 8 weeks before joining the study.
Participants must have had a bone marrow examination in the 2 months before joining the study. This includes looking at the cells and genes in the bone marrow.
Participants can use certain medications like erythropoiesis-stimulating agents (ESAs) for anemia or granulocyte colony-stimulating factor (G-CSF) for low white blood cells and infections, as long as they follow accepted standards.
Participants must have an ECOG Performance Status of 0-3, which is a scale that measures how well they can perform daily activities.
Participants must be able to understand and follow the study requirements and instructions.
Participants must have adequate organ function. This means their liver and kidney functions should be within certain limits, and their albumin levels should not be too low.

Who Cannot Join the Study?

Patients who have a different medical condition than the one being studied, which is myelodysplastic syndromes. This is a group of disorders caused by poorly formed or dysfunctional blood cells.
Patients who are not within the specified age range for the study. The study is open to certain age groups only.
Patients who are not part of the specified clinical trial groups. The study is designed for specific groups of patients.
Patients who are not male or female, as the study includes both genders.
Patients who are considered part of a vulnerable population. This term refers to groups of people who may be at a higher risk of harm or exploitation, such as children, pregnant women, or those with certain disabilities.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Azienda Ospedaliero Universitaria Careggi	Florence	Italy

Other Sites

Site Name	City	Country
Istituto Tumori Bari Giovanni Paolo II	Bari	Italy
Grande Ospedale Metropolitano Bianchi Melacrino Morelli	Reggio Calabria	Italy
ASST Grande Ospedale Metropolitano Niguarda	Milan	Italy
IRCCS Ospedale Policlinico San Martino	Genoa	Italy
Azienda Ospedaliero-Universitaria Maggiore Della Carita	Novara	Italy
Ospedale S. Eugenio, ASL Roma 2	Rome	Italy
Iivwkl Ixlymnww Fdhutimwehcxr Owgdbkbxlju	Rome	Italy
Uvomyveynl Doirq Smyiu Dy Rbuy Ln Sirzfggu	Rome	Italy

Trial status

Country	Status	Recruitment Start
Italy	Recruiting	11.06.2011

Trial locations

Investigated drugs:

Eltrombopag

Eltrombopag is a medication used in this clinical trial to treat thrombocytopenia, which is a condition characterized by low platelet counts. It is being tested for its effectiveness in patients with low and intermediate risk myelodysplastic syndromes. The trial aims to see how well Eltrombopag can improve platelet counts and assess its safety and tolerability in patients.

Myelodysplastic syndromes – These are a group of disorders caused by poorly formed or dysfunctional blood cells. They occur when something goes wrong in the bone marrow, where blood cells are produced. Over time, the bone marrow becomes less effective at producing healthy blood cells, leading to symptoms like fatigue, infections, and easy bruising or bleeding. The progression of the disease can vary, with some individuals experiencing mild symptoms for years, while others may have a more rapid decline in blood cell production. As the condition advances, it can lead to severe anemia, increased risk of bleeding, and susceptibility to infections. In some cases, myelodysplastic syndromes can progress to acute myeloid leukemia, a more aggressive form of blood cancer.

Trial ID:

2024-520133-72-00

Protocol code:

EQoL-MDS

NCT ID:

NCT02912208

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on Eltrombopag for Treating Low and Intermediate Risk Myelodysplastic Syndromes in Patients with Thrombocytopenia

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?