Condition: Hematologic Malignancy · AML · ALL · Sponsor: Therapeutic Advances in Childhood Leukemia Consortium
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)…
This description comes directly from the study's public registry record.
Benjamin N Brookhart · 323-361-5429 · bbrookhart@chla.usc.edu
Ellynore Florendo · 323-361-3022 · eflorendo@chla.usc.edu
Always discuss trial participation with your own doctor first.
| Children's Hospital Los Angeles | Los Angeles, California, United States | Recruiting |
| Children's Hospital Orange County | Orange, California, United States | Not Yet Recruiting |
| UCSF School of Medicine | San Francisco, California, United States | Recruiting |
| Children's Hospital Colorado | Denver, Colorado, United States | Recruiting |
| Children's National Medical Center | Washington D.C., District of Columbia, United States | Recruiting |
| University of Miami | Miami, Florida, United States | Not Yet Recruiting |
| Children's Healthcare of Atlanta, Emory University | Atlanta, Georgia, United States | Not Yet Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago, Illinois, United States | Recruiting |
| Riley Hospital for Children | Indianapolis, Indiana, United States | Recruiting |
| Johns Hopkins University | Baltimore, Maryland, United States | Not Yet Recruiting |
| National Cancer Institute, Pediatric Oncology Branch | Bethesda, Maryland, United States | Not Yet Recruiting |
| Dana-Farber Cancer Institute | Boston, Massachusetts, United States | Not Yet Recruiting |
+ 19 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT05476770