Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies
Status:
Not yet recruiting
Trial end date:
2027-08-01
Target enrollment:
Participant gender:
Summary
Tagraxofusp is a protein-drug conjugate consisting of a diphtheria toxin redirected to target
CD123 has been approved for treatment in pediatric and adult patients with blastic
plasmacytoid dendritic cell neoplasm (BPDCN). This trial aims to examine the safety of this
novel agent in pediatric patients with relapsed/refractory hematologic malignancies.
The mechanism by which tagraxofusp kills cells is distinct from that of conventional
chemotherapy. Tagraxofusp directly targets CD123 that is present on tumor cells, but is
expressed at lower or levels or absent on normal hematopoietic stem cells. Tagraxofusp also
utilizes a payload that is not cell cycle dependent, making it effective against both highly
proliferative tumor cells and also quiescent tumor cells.
The rationale for clinical development of tagraxofusp for pediatric patients with hematologic
malignancies is based on the ubiquitous and high expression of CD123 on many of these
diseases, as well as the highly potent preclinical activity and robust clinical
responsiveness in adults observed to date.
This trial includes two parts: a monotherapy phase and a combination chemotherapy phase. This
design will provide further monotherapy safety data and confirm the FDA approved pediatric
dose, as well as provide safety data when combined with chemotherapy.
The goal of this study is to improve survival rates in children and young adults with
relapsed hematological malignancies, determine the recommended phase 2 dose (RP2D) of
tagraxofusp given alone and in combination with chemotherapy, as well as to describe the
toxicities, pharmacokinetics, and pharmacodynamic properties of tagraxofusp in pediatric
patients.
About 54 children and young adults will participate in this study. Patients with Down
syndrome will be included in part 1 of the study.
Phase:
Phase 1
Details
Lead Sponsor:
Therapeutic Advances in Childhood Leukemia Consortium