In short
Currently, there is 1 ongoing clinical trial for bone marrow disorder, specifically focusing on lower-risk myelodysplastic syndromes with ring sideroblasts. This trial is investigating luspatercept as a treatment option for patients who require regular red blood cell transfusions and have not responded well to previous therapies. The trial is being conducted across multiple European countries including Spain, Austria, and Germany.
Clinical trial locations
- Austria
- Germany
- Spain
Study on the Effectiveness and Safety of Luspatercept for Patients with Lower-Risk Myelodysplastic Syndromes Requiring Red Blood Cell Transfusions
This trial is investigating luspatercept as a treatment for patients with lower-risk myelodysplastic syndrome with ring sideroblasts, a condition where the bone marrow produces poorly formed blood cells leading to anemia. The study aims to determine whether luspatercept can help patients become independent from red blood cell transfusions over a 24-week period.
Main inclusion criteria:
- Patients must be at least 18 years old and able to follow the study schedule
- Must have a confirmed diagnosis of myelodysplastic syndrome with specific characteristics, including the presence of ring sideroblasts in the bone marrow
- Must require regular red blood cell transfusions with specific requirements for the amount and timing
- Must have previously tried treatments with erythropoiesis-stimulating agents and either not responded, could not tolerate them, or were ineligible for such treatments
- Must have an ECOG performance score of 0, 1, or 2, which measures general health and ability to perform daily activities
- Women of childbearing potential must have negative pregnancy tests and agree to use effective birth control during and after the study
Main exclusion criteria:
- Patients whose anemia is not due to very low-, low-, or intermediate-risk myelodysplastic syndrome with ring sideroblasts
- Patients who do not require red blood cell transfusions
- Patients who have not tried erythropoiesis-stimulating agents or who are still responding to such treatments
Focus and goal:
The primary focus of this trial is to evaluate whether luspatercept can help patients achieve independence from red blood cell transfusions over 24 weeks. The study will monitor participants for changes in their transfusion needs, hemoglobin levels (the protein in red blood cells that carries oxygen), and any side effects they may experience. Additionally, researchers will assess overall survival and quality of life improvements.
Investigational drug:
Luspatercept, also known as ACE-536, is administered as an injection under the skin, typically in the upper arm, thigh, or abdomen. This medication is classified as an erythroid maturation agent, meaning it helps mature red blood cells by binding to certain proteins in the body. The goal is to increase red blood cell production and reduce or eliminate the need for regular transfusions in patients with this specific type of bone marrow disorder.
Summary
This single ongoing clinical trial for bone marrow disorder focuses specifically on a subset of patients with lower-risk myelodysplastic syndromes with ring sideroblasts who require regular red blood cell transfusions. The trial is being conducted across three European countries: Spain, Austria, and Germany, providing access to patients in multiple locations. The investigation centers on luspatercept, a relatively novel therapeutic approach that works by helping the body produce more healthy red blood cells rather than simply managing symptoms through transfusions. This trial represents an important opportunity for patients who have exhausted other treatment options or who have not responded to standard erythropoiesis-stimulating agents.
