What is luspatercept studying for anemia in alpha-thalassemia?

Luspatercept is being studied to evaluate its effect on anemia in people with alpha-thalassemia hemoglobin H disease. Researchers want to see if luspatercept combined with supportive care reduces the need for blood transfusions and increases hemoglobin levels compared to placebo. The study is sponsored by Bristol-Myers Squibb.

Who may be eligible for this anemia clinical trial?

People aged 12 years and older with a diagnosis of alpha-thalassemia hemoglobin H disease may be eligible. Adults must meet specific transfusion and health status criteria, and adolescents need certain transfusion histories and performance scores. Both males and females can participate. Please review eligibility criteria for more details.

Is there any cost to participate in this anemia clinical trial?

Study-related care, including the study drug or procedure and all trial visits, is provided at no cost. Personal travel expenses may vary and are not guaranteed to be covered. Participants should plan accordingly.

Actively Recruiting

Phase 2

Age: 12Years +

All Genders

ID05664737

Phase 2 Study of Luspatercept for Treating Anemia in Adults and Adolescents with Alpha-Thalassemia Hemoglobin H Disease Evaluating Effectiveness and Safety Compared to Placebo

Led by Bristol-Myers Squibb · Updated on 2026-08-10

189

Participants Needed

Research Sites

369 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the efficacy and safety of luspatercept combined with best supportive care compared to placebo with best supportive care in adults with alpha-thalassemia hemoglobin H disease who experience anemia. The study also assesses the safety and drug levels of luspatercept in adolescents with this condition. This phase 2 trial aims to understand how luspatercept affects anemia and transfusion needs in this population. Participants are randomly assigned to one of four groups based on their transfusion dependence status adults who receive luspatercept plus best supportive care, adults who receive placebo plus best supportive care, adolescents who are transfusion dependent receiving luspatercept plus best supportive care, or adolescents who are non-transfusion dependent receiving luspatercept plus best supportive care. The treatments are given on specified days, and participants are monitored for up to 108 weeks, including assessments of drug levels, transfusion burden, hemoglobin changes, and adverse events. Throughout the study, participants undergo regular evaluations including blood tests to measure hemoglobin and transfusion needs, monitoring for side effects, and quality of life assessments. Researchers also track long-term safety outcomes and pharmacokinetics of the drug. The main outcomes include reductions in red blood cell transfusions over 12 to 48 weeks and increases in hemoglobin levels without transfusions. Participants may be followed for up to five years to monitor safety and treatment effects.

CONDITIONS

Brief Title

A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia

Who Can Participate

Age: 12Years +

All Genders

Eligibility Criteria

You may qualify if you...

Adults aged 18 years or older with documented alpha-thalassemia hemoglobin H disease and transfusion dependence defined by 6 or more RBC units in the past 24 weeks
Adults with ECOG performance status of 0 or 1
Adolescents aged 12 to under 18 years with documented alpha-thalassemia hemoglobin H disease
Transfusion-dependent adolescents with 4 or more RBC events in past 24 weeks and no transfusion-free period longer than 56 days during that time, with at least 2 years of regular transfusions
Non-transfusion-dependent adolescents with fewer than 4 RBC events in past 24 weeks and transfusion-free for at least 8 weeks prior, with mean baseline hemoglobin of 10 g/dL or less based on 2 measurements
Adolescents with Karnofsky (age ≥16) or Lansky (age <16) performance status score of 50 or higher at screening

You will not qualify if you...

Diagnosis of alpha-thalassemia trait, Hb Bart hydrops, ATRx alpha-thalassemia, hemoglobin S/beta-thalassemia, myelodysplasia subtype anemia, or HbE homozygous beta gene mutation
Anemia due to nutritional deficiency, chronic disease, autoimmune hemolytic anemia, or other hemolytic anemias
Hemolytic episodes unrelated to alpha-thalassemia within 8 weeks prior to randomization
For EU only: bleeding disorders with frequent bleeding episodes
History of deep vein thrombosis, stroke, or other thromboembolic events requiring intervention within 24 weeks prior to randomization
Uncontrolled hypertension (controlled hypertension allowed if grade 1 or less)
Women who are pregnant, planning pregnancy during the study, or breastfeeding
Previous hematopoietic stem cell transplant or gene therapy (except candidates with 12 months waiting period)
Use of hydroxyurea treatment within 12 weeks for non-transfusion-dependent or 24 weeks for transfusion-dependent participants
Extramedullary hematopoiesis complications requiring treatment during screening
Any medical or psychiatric condition that poses unacceptable risk or affects data interpretation as judged by the investigator

Research Team

BMS Clinical Trials Contact Center www.BMSClinicalTrials.com

First line of the email MUST contain NCT # and Site #.

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