Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies (NCT05476770)
A Phase I Study of Tagraxofusp With or Without Chemotherapy in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Children's Hospital Los Angeles — Los Angeles, California
Age range: 1 Year to 21 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (31 Sites)
Showing the first 25 of 31 sites.
- Children's Hospital Los Angeles — Los Angeles, California
- Children's Hospital Orange County — Orange, California
- UCSF School of Medicine — San Francisco, California
- Children's Hospital Colorado — Denver, Colorado
- Children's National Medical Center — Washington D.C., District of Columbia
- University of Miami — Miami, Florida
- Children's Healthcare of Atlanta, Emory University — Atlanta, Georgia
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois
- Riley Hospital for Children — Indianapolis, Indiana
- Johns Hopkins University — Baltimore, Maryland
- National Cancer Institute, Pediatric Oncology Branch — Bethesda, Maryland
- Dana-Farber Cancer Institute — Boston, Massachusetts
- C.S. Mott Children's Hospital — Ann Arbor, Michigan
- Children's Hospital and Clinics of Minnesota — Minneapolis, Minnesota
- Children's Hospital New York-Presbyterian — New York, New York
- Memorial Sloan Kettering Cancer Center — New York, New York
- Carolina-Levine Children's Hospital — Charlotte, North Carolina
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio
- Rainbow Babies — Cleveland, Ohio
- Nationwide Children's Hospital — Columbus, Ohio
- Oregon Health & Science University — Portland, Oregon
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- St. Jude Children's Research Hospital — Memphis, Tennessee
- University of Texas, Southwestern — Dallas, Texas
- Cook Children's Hospital — Fort Worth, Texas
📞 Contact the Study Coordinator
Phone: (323) 361-5429
Email: bbrookhart@chla.usc.edu
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Hematologic Malignancy
- A Positive Psychology Based Intervention (PATH-C) for Caregivers of HSCT Survivors — Boston, Massachusetts
- Tocilizumab for the Prevention of Graft Failure and GVHD in Haplo-Cord Transplantation — New York, New York
- Novel 3D Hematological Malignancy Organoid to Study Disease Biology and Chemosensitivity — Winston-Salem, North Carolina
- Patient Fitness and Body Composition During Hematopoietic Stem Cell Transplantation — Seattle, Washington
- Critical Care Outcomes of Hematologic Oncology Patients — Calgary, Alberta
- Expressive Writing Program Among Young Adults With Blood Cancer — Washington D.C., District of Columbia
Explore by condition: All Hematologic Malignancy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.