A Phase 2a Study to Evaluate the Safety and Pharmacokinetics of Luspatercept (ACE-536) in Pediatric Participants with Beta (β) Thalassemia.

Conditions

Beta (β) Thalassemia

Brief summary

To determine the recommended dose (RD) for TD and NTD β-thalassemia participants, To evaluate the pharmacokinetics (PK) for TD and NTD β-thalassemia participants

Detailed description

• The safety for TD and NTD β-thalassemia participants including: Type,frequency, seriousness, and severity of AEs and relationship to luspatercept (per NCI CTCAE version 5.0), • The immunogenicity in TD andNTD β-thalassemiaparticipants, • The mean change in RBC transfusion burden in TDβ-thalassemia participants, • The mean change in hemoglobin levels in NTD β-thalassemia participants, • The mean change in mean daily dose of iron chelation therapy (ICT) in TD and NTD β-thalassemia participants, • The mean change in serum ferritin in TD andNTD β-thalassemia participants

Vip Viprakasit, Jianpei Fang, Khaled M. Musallam, Yeşim Aydınok, Thomas D. Coates et al. (11 authors)

Blood2025-11-03

10.1182/blood-2025-1135

Interventions

DRUGReblozyl 25 mg powder for solution for injection

DRUGReblozyl 75 mg powder for solution for injection

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
To determine the recommended dose (RD) for TD and NTD β-thalassemia participants, To evaluate the pharmacokinetics (PK) for TD and NTD β-thalassemia participants	—

Secondary

Measure	Time frame
• The safety for TD and NTD β-thalassemia participants including: Type,frequency, seriousness, and severity of AEs and relationship to luspatercept (per NCI CTCAE version 5.0), • The immunogenicity in TD andNTD β-thalassemiaparticipants, • The mean change in RBC transfusion burden in TDβ-thalassemia participants, • The mean change in hemoglobin levels in NTD β-thalassemia participants, • The mean change in mean daily dose of iron chelation therapy (ICT) in TD and NTD β-thalassemia participants, • The mean change in serum ferritin in TD andNTD β-thalassemia participants	—

Measure

Time frame

• The safety for TD and NTD β-thalassemia participants including: Type,frequency, seriousness, and severity of AEs and relationship to luspatercept (per NCI CTCAE version 5.0), • The immunogenicity in TD andNTD β-thalassemiaparticipants, • The mean change in RBC transfusion burden in TDβ-thalassemia participants, • The mean change in hemoglobin levels in NTD β-thalassemia participants, • The mean change in mean daily dose of iron chelation therapy (ICT) in TD and NTD β-thalassemia participants, • The mean change in serum ferritin in TD andNTD β-thalassemia participants

—

Countries

Germany, Greece, Italy

Outcome results

None listed