Luspatercept for Thalassemia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests luspatercept, a treatment designed to help children and teens with β-thalassemia, a blood disorder that reduces hemoglobin, the protein responsible for carrying oxygen in the blood. The study will assess the safety of luspatercept and its movement through the body in children who either require regular blood transfusions or do not. It includes different groups and doses to identify the most effective treatment. Children diagnosed with β-thalassemia or Hemoglobin E/β-thalassemia who need frequent blood transfusions or have low hemoglobin levels may be suitable candidates for this trial. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants.
Will I have to stop taking my current medications?
The trial protocol does not specify if you must stop taking your current medications. However, certain medications like hydroxyurea, erythropoiesis-stimulating agents, and some herbs or supplements must be stopped 12 to 24 weeks before joining, depending on your transfusion status.
Is there any evidence suggesting that luspatercept is likely to be safe for pediatric participants with β-thalassemia?
Research has shown that luspatercept is generally safe for patients with beta thalassemia. In one study with adults, about 3.6% of participants taking luspatercept experienced serious side effects, including blood clots in the veins.
Luspatercept can help increase hemoglobin levels in patients who don't require regular blood transfusions. For those who do need transfusions, it can reduce the amount needed. While there are some risks, luspatercept could be a useful treatment for managing beta thalassemia.
This information is based on studies with adults. The current trial focuses on children and teenagers, so results might differ. Always consult a doctor or healthcare provider to understand what these findings mean for individual cases.12345Why do researchers think this study treatment might be promising for thalassemia?
Luspatercept is unique because it targets anemia in thalassemia by enhancing the maturation of red blood cells, a different mechanism from the standard treatments like blood transfusions and iron chelation therapy. Most treatments for thalassemia focus on managing symptoms, but luspatercept works at a cellular level to potentially reduce the need for transfusions. This novel approach not only aims to lessen the burden of frequent transfusions but also minimizes the risk of iron overload, a common complication in current treatments. Researchers are excited because luspatercept offers a promising new avenue for improving quality of life for those with thalassemia.
What evidence suggests that luspatercept might be an effective treatment for β-thalassemia?
Research has shown that luspatercept helps treat beta-thalassemia by reducing the need for red blood cell transfusions. One study found that almost 95% of patients taking luspatercept experienced a significant increase in hemoglobin levels, compared to only 22% in the placebo group. Long-term evidence indicates that luspatercept remains effective over time, reducing the need for transfusions. This treatment has demonstrated lasting benefits, making it a promising option for managing beta-thalassemia. Participants in this trial will receive different dosages of luspatercept, as the study examines its effectiveness across various age groups and dosing regimens.678910
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 children and adolescents aged 6 to less than 18 with β-thalassemia who need regular blood transfusions. They should have a performance status score ≥50, be on transfusion treatment for at least 2 years, and agree to contraception if applicable. Excluded are those with recent major surgery, certain other health conditions or treatments, or prior exposure to similar drugs.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive luspatercept with dose escalation and expansion cohorts for both transfusion-dependent and non-transfusion-dependent β-thalassemia participants
Long-term Treatment
Participants benefiting from the study treatment may continue luspatercept treatment for up to 5 years
Posttreatment Follow-up
Participants are monitored for safety and effectiveness after discontinuing 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?
Celgene
Lead Sponsor
Jay Backstrom
Celgene
Chief Medical Officer since 2016
MD
Mark Alles
Celgene
Chief Executive Officer since 2016
Bachelor's degree from Lock Haven University of Pennsylvania
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Industry Sponsor
Acceleron Pharma Inc. (a wholly owned subsidiary of Merck Sharp and Dohme, a subsidiary of Merck & Co., Inc.)
Industry Sponsor