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Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies
NCT05476770 · View on ClinicalTrials.gov ↗
Study 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.
Conditions Studied
Interventions
- DRUG Fludarabine
- DRUG Dexamethasone
- DRUG Vincristine
- DRUG Cytarabine
- DRUG Tagraxofusp
Study Locations (20)
California
- Children's Hospital Los Angeles — Los Angeles
- Children's Hospital Orange County — Orange
- UCSF School of Medicine — San Francisco
Ohio
- Cincinnati Children's Hospital Medical Center — Cincinnati
- Rainbow Babies — Cleveland
- Nationwide Children's Hospital — Columbus
Maryland
- Johns Hopkins University — Baltimore
- National Cancer Institute, Pediatric Oncology Branch — Bethesda
New York
- Children's Hospital New York-Presbyterian — New York
- Memorial Sloan Kettering Cancer Center — New York
Colorado
- Children's Hospital Colorado — Denver
District of Columbia
- Children's National Medical Center — Washington D.C.
Florida
- University of Miami — Miami
Georgia
- Children's Healthcare of Atlanta, Emory University — Atlanta
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 54 participants |
| Start Date | 2022-11-11 |
| Est. Completion | 2027-11-11 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05476770
The ClinicalTrials.gov registry entry for NCT05476770 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 54 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Therapeutic Advances in Childhood Leukemia Consortium, which has 5 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 10 conditions, with Hodgkin Lymphoma appearing as the primary indexed condition, and to 5 interventions — of which Fludarabine is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05476770 reports 20 study locations spanning 14 distinct geographic areas — top geographies include California, Ohio, Maryland. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05476770 about?
NCT05476770 is a clinical study titled "Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies". 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 patien...
What is the current status of trial NCT05476770?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 54 participants. The study started on 2022-11-11. Estimated completion is 2027-11-11.
What conditions does trial NCT05476770 study?
This clinical trial studies the following conditions: Hodgkin Lymphoma, Hematologic Malignancy, Lymphoma, B-Cell, Mixed Phenotype Acute Leukemia, AML. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05476770?
The interventions under investigation include: Fludarabine (DRUG), Dexamethasone (DRUG), Vincristine (DRUG), Cytarabine (DRUG), Tagraxofusp (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05476770?
This trial is sponsored by Therapeutic Advances in Childhood Leukemia Consortium, which has 5 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05476770 being conducted?
This trial has 20 study locations across California, Colorado, District of Columbia, Florida, Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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