Luspatercept for Myelodysplastic Syndrome
(MAXILUS Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a treatment called Luspatercept to evaluate its effectiveness for people with low-risk Myelodysplastic Syndrome (MDS) who require regular red blood cell transfusions. Researchers aim to determine if Luspatercept can reduce the need for these transfusions and assess its safety at the highest approved dose. The trial includes two groups: one for those who have not tried certain anemia treatments and another for those whose previous treatments were ineffective. This trial may suit individuals with MDS who regularly need blood transfusions and have not undergone a stem cell transplant or have uncontrolled high blood pressure. As a Phase 3 trial, this treatment is in the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking therapy.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that Luspatercept is likely to be safe for humans?
Research has shown that Luspatercept is generally well-tolerated by patients with certain types of anemia. It serves as an effective alternative to blood transfusions for individuals with lower-risk myelodysplastic syndromes (MDS), a type of blood disorder. Some patients have reported side effects, but most were mild.
In a large study involving 242 patients, only about 2.1% experienced serious side effects, indicating relatively low risks. Other studies have demonstrated that Luspatercept can be used safely over a long period in patients with low-risk MDS.
Overall, Luspatercept has been used safely in conditions similar to the one under study. However, like any treatment, discussing potential risks with a healthcare provider is important.12345Why do researchers think this study treatment might be promising for Myelodysplastic Syndrome?
Luspatercept is unique because it targets the underlying problem in myelodysplastic syndrome by enhancing red blood cell production. Most treatments for this condition, like erythropoiesis-stimulating agents (ESAs), aim to stimulate red blood cell creation but often face resistance or relapse over time. Luspatercept, however, works by a different mechanism, targeting late-stage red blood cell maturation, which could provide a more effective solution for patients who have not responded well to traditional ESAs. Researchers are excited about its potential to improve anemia in these patients, offering a new hope for those with limited treatment options.
What evidence suggests that Luspatercept could be an effective treatment for Myelodysplastic Syndrome?
Research shows that luspatercept can reduce the need for blood transfusions in people with myelodysplastic syndrome (MDS). One study found that it made patients less dependent on transfusions, requiring fewer to manage their anemia. In this trial, participants will be divided into two cohorts: those who are erythropoiesis-stimulating agents (ESA) naïve and those who are ESA relapsed or refractory. Patients with lower-risk MDS have achieved better results with luspatercept compared to standard treatments. This treatment has significantly improved anemia, a common issue for those with MDS. Overall, luspatercept shows promise in easing patients' lives by reducing their need for transfusions.16789
Who Is on the Research Team?
Bristol-Myers Squibb
Principal Investigator
Bristol-Myers Squibb
Are You a Good Fit for This Trial?
This trial is for people with low-risk Myelodysplastic Syndrome (MDS) who need regular red blood cell transfusions. They should be relatively active and able to care for themselves (ECOG score of 0-2). People can't join if they have certain types of anemia, acute myeloid leukemia (AML), uncontrolled high blood pressure, or a history of stem cell transplant.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive Luspatercept at the maximum approved dose for low-risk Myelodysplastic Syndrome
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Luspatercept
Luspatercept is already approved in United States, European Union for the following indications:
- Anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions
- Anemia in adults with transfusion-dependent anemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts
- Anemia in adults with transfusion-dependent beta thalassemia
- Anemia in adults with transfusion-dependent anemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts
Find a Clinic Near You
Who Is Running the Clinical Trial?
Bristol-Myers Squibb
Lead Sponsor
Christopher Boerner
Bristol-Myers Squibb
Chief Executive Officer since 2023
PhD in Business Administration from the Haas School of Business, University of California, Berkeley; BA in Economics and History from Washington University in St. Louis
Deepak L. Bhatt
Bristol-Myers Squibb
Chief Medical Officer since 2024
MD from Yale University; MSc in Clinical Epidemiology from the University of Pennsylvania