RecruitingRare disease

Study on Luspatercept for Treating Anemia in Patients with Myelodysplastic Syndromes (MDS) with del5q, Resistant to Prior Treatments and Needing Blood Transfusions

Verified siteRegistered drugNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effectiveness and safety of a treatment for a specific type of anemia. The anemia being studied is due to Myelodysplastic Syndromes (MDS) with a particular genetic feature called del5q. This condition often requires patients to receive regular red blood cell transfusions. The treatment being tested in this study is called Luspatercept, which is administered as a solution for injection under the skin. Luspatercept is a type of protein designed to help reduce the need for blood transfusions in patients with this type of anemia.

The purpose of the study is to evaluate how well Luspatercept can help patients achieve a period of at least eight weeks without needing a blood transfusion within the first 24 weeks of treatment. The study will also look at the safety and tolerability of Luspatercept, as well as its effects on reducing the number of transfusions needed, increasing hemoglobin levels, and improving quality of life. Participants will receive either Luspatercept or a placebo, and their progress will be monitored over a period of time to assess these outcomes.

Participants in the study will receive regular injections and attend scheduled visits to monitor their health and response to the treatment. The study aims to provide valuable information on whether Luspatercept can be an effective treatment option for patients with anemia due to MDS with del5q, who are resistant or intolerant to previous treatments and require regular blood transfusions.

The research process

The trial runs in 3 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial treatment phase
The treatment involves the administration of luspatercept, which is provided as a powder for solution for injection. The available dosages are 75 mg and 25 mg.
The medication is administered through a subcutaneous injection. This means the injection is given under the skin.
The primary goal during this phase is to evaluate the effect of luspatercept on achieving red blood cell transfusion independence for 8 consecutive weeks within the first 24 weeks.
Step 2
Monitoring and assessment
Throughout the trial, regular monitoring is conducted to assess the safety and tolerability of luspatercept.
The study also evaluates the duration of red blood cell transfusion independence, reduction in transfusion needs, and any increase in hemoglobin levels.
Changes in quality of life scores, serum ferritin levels, and the use of iron chelation therapy are also monitored.
Step 3
Extended evaluation
The study continues to assess the long-term effects of luspatercept, including red blood cell transfusion independence at 48 weeks and at the end of the study.
The estimated end date for the trial is December 31, 2029.

Who can join the trial?

11 criteria

The person must be at least 18 years old when they sign the consent form.
The person must understand and willingly sign a consent form before any study-related activities are done.
The person must have a confirmed diagnosis of **MDS with del5q**. MDS stands for **Myelodysplastic Syndromes**, which are a group of disorders caused by poorly formed or dysfunctional blood cells. **Del5q** refers to a specific genetic abnormality.
The person must have a specific risk level of the disease, classified as very low, low, or intermediate, with less than 5% **blasts** in the bone marrow. Blasts are immature blood cells. The white blood cell count in the blood must be less than 13,000 per microliter.
The person must be unable to respond to, cannot tolerate, or is not eligible for previous **ESA** treatment. ESA stands for **Erythropoiesis-Stimulating Agents**, which are drugs that help the body make more red blood cells.
If the person was previously treated with ESAs or **G-CSF** (Granulocyte Colony-Stimulating Factor, a drug that helps the body make more white blood cells), both treatments must have been stopped at least 4 weeks before the screening date.
The person must be unable to respond to, cannot tolerate, or is not eligible for previous **lenalidomide** treatment. Lenalidomide is a medication used to treat certain blood disorders. The person must have stopped lenalidomide treatment at least 4 weeks before the screening date. They must also need regular **RBC transfusions** (red blood cell transfusions), with specific requirements for the amount and timing of these transfusions.
The person must have an **ECOG score** of 0, 1, or 2. The ECOG score is a scale used to assess how a disease affects a person's daily living abilities, with 0 being fully active and 5 being dead.
Females who can have children must have two negative pregnancy tests before starting the study therapy. They must agree to ongoing pregnancy testing during and after the study. If sexually active, they must use highly effective birth control starting 5 weeks before the study, during the study, and for 12 weeks after stopping the study therapy.
Male participants must agree to use a condom during sexual contact with a pregnant woman or a woman who can have children while in the study, during breaks in treatment, and for at least 12 weeks after stopping the study treatment, even if they have had a vasectomy.
The person must be willing and able to follow the study visit schedule and other study requirements.

Who cannot join the trial?

7 criteria

Patients with a different type of anemia than the one specified in the study.
Patients with a bone marrow blast count of 5% or higher. Bone marrow blasts are immature blood cells found in the bone marrow.
Patients who have not tried or are not resistant to treatments like ESA or lenalidomide. ESA stands for erythropoiesis-stimulating agents, which help the body make more red blood cells. Lenalidomide is a medication used to treat certain blood disorders.
Patients who do not require regular red blood cell transfusions.
Patients who are not within the specified age range for the study.
Patients who are not male or female, as the study includes both genders.
Patients who are part of a vulnerable population that the study does not include.

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Verified sites

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Verified
ASST Grande Ospedale Metropolitano Niguarda
Milan, Italy
Responsive
Azienda Ospedaliero Universitaria Careggi
Florence, Italy
Responsive
Universita Degli Studi Di Roma La Sapienza
Rome, Italy

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Investigated drugs

Luspatercept
is a medication being studied for its ability to treat anemia in patients with a specific type of blood disorder called Myelodysplastic Syndromes (MDS) with del5q. This condition often requires patients to receive regular red blood cell transfusions. The trial is investigating whether luspatercept can help reduce or eliminate the need for these transfusions over a certain period.
Lenalidomide
is a medication that some patients with Myelodysplastic Syndromes (MDS) may have used before participating in this trial. The study includes patients who are resistant, intolerant, or have not responded well to lenalidomide, and it aims to find an alternative treatment option for them.

Luspatercept

What is already known about the treatment

Luspatercept – Luspatercept is administered via subcutaneous injection and is currently being studied for its effectiveness in treating anemia associated with myelodysplastic syndromes (MDS), particularly in patients with a specific genetic deletion known as del5q. These patients are often resistant or intolerant to previous treatments and require regular red blood cell transfusions. Luspatercept works by binding to certain proteins in the body, which helps to increase the production of red blood cells. It is classified as an erythroid maturation agent, and its role in medical literature is focused on its potential to reduce the need for transfusions in affected individuals.

Investigated diseases

Myelodysplastic Syndromes with del(5q) – This is a type of blood disorder where the bone marrow does not produce enough healthy blood cells. It is characterized by a specific genetic abnormality known as deletion 5q, which affects the production of red blood cells. Patients with this condition often require regular red blood cell transfusions due to anemia. The disease progresses slowly and is classified based on risk levels, such as very low, low, or intermediate, according to the IPSS-R scoring system. The bone marrow typically shows less than 5% of immature cells, known as blasts. Individuals may become resistant or intolerant to certain treatments, such as lenalidomide, and may not respond to erythropoiesis-stimulating agents.