Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants Who Require Regular RBC Transfusions Due to Beta (β)-Thalassemia.

• Status: Recruiting
• Trial end date: 2033-09-30

Summary

This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants who require regular red blood cell transfusions due to β-thalassemia.

The study will be conducted in 2 parts: Part A will be in adolescent participants aged 12 to <18 years with two dose escalation cohorts of 6 participants each, followed by a dose expansion cohort of 30 participants. Part B will begin after a review of the safety in participants completing at least one year of treatment in Part A and will be in participants aged 6 to <12 with two dose escalation cohorts of 6 participants each.

Upon completion of the Treatment Period, participants of any cohort who are benefiting from the study treatment, will be offered the opportunity to continue luspatercept treatment in the Long-term Treatment Period for up to 5 years from their first dose (Cycle 1 Day 1).

Participants who discontinue study treatment any time will continue in the Posttreatment Follow-up Period for at least 5 years from their first dose of luspatercept (Cycle 1 Day 1), or 3 years from their last dose, whichever occurs later, or until they withdraw consent/assent, are lost to follow-up, or the End of Trial, whichever occurs first.

• Phase: Phase 2

Details

• Lead Sponsor: Celgene
• Collaborators: Acceleron Pharma Inc.; Acceleron Pharma, Inc.
• Treatments: Immunoglobulin Fc Fragments; Luspatercept