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

About the trial

Sponsor

Celgene (with Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA)

Tested

ACE-536 (drug)

Dates

Started 2019-11-07; completion 2035-08-31

Results posted

Not yet

This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia. The study will be conducted in 2 parts for both transfusion-dependent (TD) and non-transfusion-dependent (NTD) β-thalassemia participants: TD Part A will be in adolescent participants aged 12 to \<18 years with two dose escalation cohorts, followed by a dose expansion cohorts. NTD Part A will be conducted in the same age group participants as TD Part A with dose confirmation and expansion cohorts. After Part A TD participants have completed at least one year of treatment, all available safety data from Part A adolescent participants will be evaluated befo…

From ClinicalTrials.gov, last updated 2026-07-10. Full record, eligibility and contacts (opens in a new tab)

Industry funding

E.r. Squibb & Sons, L.l.c. reported $13,150 in research payments for this study in 2025 (2 payments).

Products named: Reblozyl

Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.