Study on the Effectiveness and Safety of Luspatercept for Patients with Lower-Risk Myelodysplastic Syndromes Requiring Red Blood Cell Transfusions

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What is this study about?

This clinical trial is focused on studying a condition known as myelodysplastic syndrome (MDS), specifically a type called lower-risk MDS with ring sideroblasts (MDS-RS). MDS is a group of disorders caused by poorly formed or dysfunctional blood cells. The study is investigating the effectiveness and safety of a treatment called luspatercept, which is also known by its code name ACE-536. Luspatercept is a medication that is given as an injection under the skin and is designed to help improve anemia, a condition where the body does not have enough healthy red blood cells.

The purpose of this study is to see if luspatercept can help patients with lower-risk MDS-RS who need regular red blood cell transfusions. These patients have not responded well to other treatments that stimulate the production of red blood cells. The study will follow participants over a period of 24 weeks to see if they can achieve independence from red blood cell transfusions. This means that the patients would not need transfusions to maintain healthy red blood cell levels during this time.

Participants in the study will receive luspatercept injections and will be monitored for any changes in their need for red blood cell transfusions, as well as any side effects they may experience. The study will also look at other outcomes, such as changes in hemoglobin levels, which is a protein in red blood cells that carries oxygen, and overall survival. The goal is to determine if luspatercept can provide a safe and effective treatment option for patients with lower-risk MDS-RS.

1 joining the study

Upon joining the study, the patient will be required to sign an informed consent form. This form confirms the patient’s understanding of the study and willingness to participate.

The patient must be at least 18 years old and able to follow the study schedule and requirements.

2 initial assessment

The patient will undergo an initial assessment to confirm eligibility. This includes verifying a diagnosis of myelodysplastic syndrome (MDS) with specific characteristics, such as the presence of ring sideroblasts in the bone marrow.

The patient must have a history of anemia requiring red blood cell (RBC) transfusions and must have been unresponsive, intolerant, or ineligible for previous treatments with erythropoiesis-stimulating agents (ESA).

3 treatment initiation

The patient will begin treatment with luspatercept, administered as a subcutaneous injection. The specific dosage will be determined by the healthcare provider based on the patient’s needs.

The treatment aims to reduce the need for RBC transfusions and improve anemia symptoms.

4 treatment schedule

The patient will receive luspatercept injections at regular intervals, as determined by the healthcare provider. The frequency and duration of the treatment will be specified in the study protocol.

The patient will be monitored for any side effects or adverse reactions to the treatment.

5 ongoing assessments

Throughout the study, the patient will undergo regular assessments to evaluate the effectiveness of the treatment. This includes monitoring the patient’s hemoglobin levels and the need for RBC transfusions.

The patient will also be assessed for any changes in symptoms and overall health status.

6 end of treatment

At the end of the treatment period, the patient will have a final assessment to determine the overall impact of the treatment on their condition.

The patient will be informed about the results and any further steps or follow-up care that may be necessary.

Who Can Join the Study?

Must be 18 years or older.
Must be willing and able to follow the study schedule and requirements.
Must understand and sign the consent form before any study procedures.
Must have a confirmed diagnosis of a specific type of blood disorder called MDS with certain characteristics in the bone marrow and blood.
Must have tried certain treatments for anemia and either not responded, stopped responding, or been unable to tolerate them.
If previously treated with specific medications, they must be stopped at least 4 weeks before starting the study treatment.
Must need regular blood transfusions, with specific requirements for the amount and timing of these transfusions.
Must have an ECOG score of 0, 1, or 2, which is a measure of general health and ability to perform daily activities.
If a woman can become pregnant, she must have two negative pregnancy tests before starting the study and agree to use effective birth control during and after the study.
Men must agree to use a condom during sexual contact with a pregnant woman or a woman who can become pregnant while in the study and for a period after the study ends.

Who Cannot Join the Study?

Patients who do not have anemia due to very low-, low-, or intermediate-risk MDS with ring sideroblasts (RS) as defined by the International Prognostic Scoring System-Revised (IPSS-R).
Patients who do not require red blood cell (RBC) transfusions.
Patients who are not refractory, intolerant, or ineligible to prior erythropoiesis-stimulating agents (ESA) treatment. Refractory means that the treatment no longer works for the patient. Intolerant means that the patient cannot tolerate the side effects of the treatment. Ineligible means that the patient cannot receive the treatment for other reasons.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Medizinische Universitaet Innsbruck	Innsbruck	Austria
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Medical University Of Vienna	Vienna	Austria
Hospital Universitario De Salamanca	Salamanca	Spain
Universitaet Leipzig	Leipzig	Germany
Hospital Universitario De Navarra	Pamplona	Spain

Other Sites

Site Name	City	Country
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)	Munich	Germany
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Ivhfelhc Cttkxa Deoqswxrtnrsygztx	L'hospitalet De Llobregat	Spain
Hnqebwdp Vwhf dflcidmr	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	02.08.2021
Germany	Not recruiting	02.08.2021
Spain	Not recruiting	02.08.2021

Trial locations

Investigated drugs:

Luspatercept

Luspatercept is a medication used in this clinical trial to help patients with a specific type of blood disorder called myelodysplastic syndromes (MDS) that have a ring sideroblastic phenotype. This condition often leads to anemia, which means the body doesn’t have enough healthy red blood cells. Luspatercept works by helping the body produce more red blood cells, which can reduce or eliminate the need for blood transfusions. The goal of using luspatercept in this study is to see if it can help patients become independent of red blood cell transfusions, improving their quality of life.

Myelodysplastic Syndromes with Ring Sideroblasts (MDS-RS) – Myelodysplastic Syndromes with Ring Sideroblasts are a group of disorders caused by poorly formed or dysfunctional blood cells. In MDS-RS, the bone marrow produces ring sideroblasts, which are abnormal red blood cell precursors with iron-loaded mitochondria. This condition leads to anemia, as the body does not produce enough healthy red blood cells. Over time, patients may require regular red blood cell transfusions due to the persistent anemia. The disease can progress slowly, with symptoms like fatigue and weakness becoming more pronounced as the anemia worsens. The risk of progression to more severe forms of blood disorders varies among individuals.

Trial ID:

2024-515069-33-00

Protocol code:

LUSPLUS

NCT ID:

NCT05181592

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Effectiveness and Safety of Luspatercept for Patients with Lower-Risk Myelodysplastic Syndromes Requiring Red Blood Cell Transfusions

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?