Phase 2a Evaluation of Luspatercept Safety and Pharmacokinetics in Children and Adolescents With Beta-Thalassemia

What this Trial Is About

Researchers are evaluating the safety and how the body processes the drug luspatercept in children and adolescents aged 6 to under 18 years with beta-thalassemia, a blood disorder. This Phase 2a study includes both transfusion-dependent and non-transfusion-dependent participants. The study aims to find the recommended dose and assess pharmacokinetics over up to one year, with additional measurements of red blood cell transfusion burden, hemoglobin levels, iron therapy use, and safety events. The study is divided into two parts. Part A involves adolescents aged 12 to under 18 years with dose escalation and expansion cohorts for both transfusion-dependent and non-transfusion-dependent groups. After one year of treatment in Part A transfusion-dependent participants, Part B will enroll younger children aged 6 to under 12 years for further dose escalation cohorts. Luspatercept is given at specified doses, and participants benefiting from treatment may continue for up to five years in a long-term treatment period. Those who stop treatment will be followed up for at least five years or until study end. Participants will attend scheduled visits for treatment administration and assessments, including blood tests, monitoring of transfusions, hemoglobin levels, iron therapy, and adverse events. Pregnancy testing and reproductive education are required for participants of childbearing potential. The study monitors pharmacokinetics and safety throughout treatment, with follow-up continuing after treatment ends. Total participation can last up to 5 years or more depending on treatment continuation or follow-up needs.

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia