Study on Luspatercept for Improving Brain Oxygenation in Patients with Non-Transfusion Dependent Thalassemia

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What is this study about?

This clinical trial is focused on studying a condition known as non-transfusion dependent beta-thalassemia, a type of blood disorder that affects the body’s ability to produce hemoglobin, the protein in red blood cells that carries oxygen. The treatment being tested in this study is called luspatercept, which is administered as a powder for solution for injection. Luspatercept is designed to help improve anemia, a condition where the body doesn’t have enough healthy red blood cells, in patients with this type of thalassemia.

The purpose of the study is to see if luspatercept can improve the way the brain uses oxygen in patients with non-transfusion dependent thalassemia. This will be assessed by measuring the cerebral metabolic rate of oxygen (CMRO2) using a special imaging technique called MRI. The study will also look at other factors, such as blood flow in the brain and heart function, over a period of 27 weeks. Participants will receive regular assessments, including blood tests and heart scans, to monitor their health and the effects of the treatment.

During the study, some participants will receive luspatercept, while others may receive a placebo. The study aims to determine if luspatercept can lead to an increase in hemoglobin levels by at least 1 gram per deciliter, which could potentially improve the symptoms of anemia in these patients. The trial is expected to run until early 2025, with recruitment starting in October 2023.

1 initial assessment

Upon joining the study, an initial assessment is conducted. This includes a review of your medical history and a physical examination.

Baseline measurements are taken, including a transthoracic echocardiography if not done within the past year, and general laboratory tests on blood samples to assess hemoglobin, leukocyte and platelet count, as well as kidney and liver function.

2 treatment initiation

The treatment involves the administration of luspatercept, known by the brand name Reblozyl, which is a powder for solution for injection.

The medication is given through an intravenous route. The specific dosage and frequency are determined by the study protocol and your healthcare provider.

3 ongoing monitoring

Throughout the study, regular monitoring is conducted every three weeks. This includes blood tests to check hemoglobin levels and other blood components.

MRI scans are performed to measure the cerebral metabolic rate of oxygen (CMRO2) using a technique called T2 relaxation under spin tagging (TRUST).

4 secondary assessments

Additional assessments include measuring cerebral blood flow (CBF) using time-encoded CASL and evaluating the tricuspid regurgitant velocity (TRV) through echocardiography.

These assessments are conducted at the start of the study and again 27 weeks later.

5 completion of study

The study is expected to conclude by January 1, 2025. At the end of the study, a final assessment is conducted to evaluate the overall effects of the treatment.

Who Can Join the Study?

Have a condition called NTDT beta-thalassemia, which means you have a type of thalassemia that doesn’t require regular blood transfusions.
Have a baseline Hb level of 10.0 or less. Hb stands for hemoglobin, which is a protein in your blood that carries oxygen. This level is based on the average Hb level 24 weeks before starting the study treatment.
Be 18 years of age or older.
Have received 5 or fewer RBC units in blood transfusions in the 24 weeks before joining the study. RBC stands for red blood cells.
If you are a female who can have children, you must use highly effective birth control methods from the start of the study to 30 days after the last dose of the study drug. If you are male, you must be willing to use barrier methods of birth control during the same period.
Have provided documented informed consent or assent. This means you have read and signed a form that explains the study and agree to participate. If you have a legal representative or guardian, they must also agree and sign.

Who Cannot Join the Study?

Patients who need regular blood transfusions cannot participate.
Patients with other serious health conditions that might interfere with the study cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who have participated in another clinical trial recently cannot participate.
Patients with allergies to the study medication cannot participate.
Patients who are unable to follow the study procedures cannot participate.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Alavsatbk Utk	Amsterdam	The Netherlands
Aqfvkqilu Ugo	Amsterdam	The Netherlands

Trial status

Country	Status	Recruitment Start
The Netherlands	Recruiting	01.10.2023

Trial locations

Investigated drugs:

Luspatercept

Luspatercept is a medication being studied to see if it can improve brain oxygen metabolism in patients with non-transfusion-dependent thalassemia. This condition often leads to anemia, and luspatercept is being tested to see if it can help increase hemoglobin levels, which may improve oxygen delivery to the brain.

Non-transfusion dependent beta-thalassemia – This is a genetic blood disorder characterized by reduced production of hemoglobin, the protein in red blood cells that carries oxygen. Unlike transfusion-dependent beta-thalassemia, individuals with this condition do not require regular blood transfusions. The disease progresses with symptoms such as mild to moderate anemia, fatigue, and sometimes an enlarged spleen. Over time, the body may try to compensate for the lack of hemoglobin by increasing the production of red blood cells, which can lead to bone deformities and other complications. The severity of symptoms can vary widely among individuals, and some may experience more pronounced effects than others.

Trial ID:

2023-504908-28-01

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on Luspatercept for Improving Brain Oxygenation in Patients with Non-Transfusion Dependent Thalassemia

What is this study about?

Who Can Join the Study?

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