Luspatercept for Thalassemia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test the effectiveness of a new treatment called luspatercept (Reblozyl) for individuals with anemia linked to α-thalassemia hemoglobin H (HbH) disease. The researchers will assess whether luspatercept, combined with usual care, reduces anemia more effectively than a placebo. They seek participants diagnosed with this condition who require regular blood transfusions. The trial will also collect data on the drug's safety and how teenagers' bodies process it. Participants should have received regular blood transfusions for at least two years due to α-thalassemia. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but it does mention that participants should not have used hydroxyurea treatment within 12 to 24 weeks before joining, depending on their transfusion status. It's best to discuss your specific medications with the trial team.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that luspatercept has been tested in various studies for its safety and effectiveness. These studies found that luspatercept increased hemoglobin levels, which is important for reducing anemia. For individuals requiring regular blood transfusions, it also reduced the number of transfusions needed.
One study found that luspatercept was well-tolerated over a long period, with patients experiencing improvements in anemia. Another study focused on individuals who didn't need regular transfusions and showed that luspatercept led to lasting increases in hemoglobin levels for up to about four and a half years.
Although these studies did not report serious safety concerns, it is important to remember that this is a clinical trial. The goal is to learn more about the safety and effectiveness of luspatercept for thalassemia. Joining a trial means contributing to this important research while being closely monitored by healthcare professionals.12345Why do researchers think this study treatment might be promising for thalassemia?
Luspatercept is unique because it targets a key underlying issue in thalassemia: ineffective erythropoiesis, which is the production of red blood cells. Unlike traditional treatments like regular blood transfusions and iron chelation therapy that primarily manage symptoms, Luspatercept works by enhancing the maturation of red blood cells. This not only helps reduce the need for transfusions but also potentially improves patients' overall quality of life. Researchers are excited about Luspatercept because it offers a novel mechanism of action that directly addresses the root cause of the condition, providing hope for more sustainable management of thalassemia.
What evidence suggests that luspatercept could be an effective treatment for thalassemia?
Research has shown that luspatercept, which participants in this trial may receive, can significantly help with anemia in people with thalassemia. Studies have found that it leads to lasting increases in hemoglobin levels, reducing the need for blood transfusions. In patients who do not require regular blood transfusions, luspatercept treatment improved hemoglobin levels for up to about 4.6 years. This trial will compare luspatercept with a placebo to evaluate its effectiveness in managing anemia in thalassemia, potentially easing life for those with this condition.26789
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 adults with α-thalassemia hemoglobin H (HbH) disease, which may include those dependent on blood transfusions. Participants should have a stable health status (ECOG score of 0 or 1), and not be at risk of pregnancy or causing one. Exclusions include other types of anemia, bleeding disorders, recent unrelated hemolysis episodes, significant medical conditions that could affect study participation, prior gene therapy for α-thalassemia, recent use of certain medications like ESAs, and history of DVT or stroke.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive luspatercept or placebo plus best supportive care for anemia management
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term Follow-up
Participants are monitored for long-term safety and efficacy outcomes
What Are the Treatments Tested in This Trial?
Interventions
- Luspatercept
- Placebo
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