OncoMatch/Clinical Trials/NCT05476770
Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies
Is NCT05476770 recruiting? Yes, currently enrolling (May 2026). This Phase 1 trial studies multiple treatments for hematologic malignancy.
Treatment: Tagraxofusp · Fludarabine · Cytarabine · Dexamethasone · Vincristine · Azacitidine · Methotrexate · Cytarabine IT · Hydrocortisone — 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.
Check if I qualifyExtracted eligibility criteria
Cancer type
Acute Myeloid Leukemia
Acute Lymphoblastic Leukemia
Non-Hodgkin Lymphoma
Multiple Myeloma
Myelodysplastic Syndrome
Chronic Lymphocytic Leukemia
Hodgkin Lymphoma
Biomarker criteria
Required: IL3RA overexpression (surface expression as defined by local institution)
Tumor cells must demonstrate surface expression of CD123 at the time of enrollment by flow cytometry or immunohistochemistry, as defined by the local institution.
Prior therapy
Cannot have received: CD123-targeted therapy (tagraxofusp)
Exception: Patients that have received other non-tagraxofusp CD123 targeting agents are eligible.
Patients that have previously received tagraxofusp are not eligible.
Lab requirements
Blood counts
Platelet count must be ≥20,000/mm3 to initiate therapy (may receive platelet transfusions); not refractory to red blood cell or platelet transfusions
Kidney function
Creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73m2 OR normal serum creatinine for age/gender
Liver function
Total bilirubin ≤ 1.5 x institutional ULN for age; SGPT (ALT) and SGOT (AST) < 3x institutional ULN; serum albumin ≥3.2 g/dL (albumin infusion independent)
Cardiac function
Shortening fraction of ≥27% by echocardiogram, or ejection fraction of ≥50% by gated radionuclide study/echocardiogram
Adequate Bone Marrow Function... Adequate Renal Function... Adequate Liver Function... Adequate Cardiac Function...
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial 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
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