Actively Recruiting
Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies
Led by Therapeutic Advances in Childhood Leukemia Consortium · Updated on 2024-12-06
54
Participants Needed
31
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must be between 1 and 21 years old at enrollment.
- Diagnosis of relapsed or refractory hematologic malignancy expressing CD123 confirmed by local testing.
- Disease status: second or greater relapse or refractory after two or more chemotherapy cycles for monotherapy; first or greater relapse or refractory after two or more chemotherapy cycles for combination therapy.
- For leukemia, >5% blasts in bone marrow or 1%-5% with stable or rising disease on serial tests.
- For lymphoma, histologically confirmed relapse with measurable disease; CNS disease allowed only in combination phase.
- Karnofsky > 50% for patients >16 years; Lansky > 50% for patients ≤16 years.
- Full recovery from acute toxic effects of all prior therapies as specified.
- Adequate bone marrow, renal, liver, cardiac, and pulmonary function as defined.
- Female patients of childbearing potential must have a negative pregnancy test within 2 weeks prior.
- Agreement to use effective contraception during study and for 12 weeks after last dose.
- Patients with Down syndrome are eligible for monotherapy phase only.
You will not qualify if you...
- CNS disease not allowed in monotherapy phase; isolated CNS disease excluded from both phases.
- Patients on unstable or increasing corticosteroid doses within 7 days prior to enrollment.
- Current use of other investigational drugs.
- Use of other anti-cancer agents, radiation, or immunotherapy during study except allowed prior therapies.
- Use of anti-GVHD or organ rejection medications within 4 weeks prior to enrollment.
- Active infections requiring intravenous treatment or recent positive blood/fungal cultures.
- Known allergy to any study drugs.
- Significant concurrent illness, psychiatric disorder, or social issues impacting safety or compliance.
- Presence of DNA fragility syndromes such as Fanconi anemia or Bloom syndrome.
AI-Screening
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Trial Site Locations
Total: 31 locations
1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
2
Children's Hospital Orange County
Orange, California, United States, 92868
Not Yet Recruiting
3
UCSF School of Medicine
San Francisco, California, United States, 94143-0106
Actively Recruiting
4
Children's Hospital Colorado
Denver, Colorado, United States, 80045
Actively Recruiting
5
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
6
University of Miami
Miami, Florida, United States, 33136
Not Yet Recruiting
7
Children's Healthcare of Atlanta, Emory University
Atlanta, Georgia, United States, 30322
Not Yet Recruiting
8
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
9
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
Actively Recruiting
10
Johns Hopkins University
Baltimore, Maryland, United States, 21231
Not Yet Recruiting
11
National Cancer Institute, Pediatric Oncology Branch
Bethesda, Maryland, United States, 20892
Not Yet Recruiting
12
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Not Yet Recruiting
13
C.S. Mott Children's Hospital
Ann Arbor, Michigan, United States, 48109-0914
Actively Recruiting
14
Children's Hospital and Clinics of Minnesota
Minneapolis, Minnesota, United States, 55404
Not Yet Recruiting
15
Children's Hospital New York-Presbyterian
New York, New York, United States, 10032
Not Yet Recruiting
16
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
Actively Recruiting
17
Carolina-Levine Children's Hospital
Charlotte, North Carolina, United States, 28204
Not Yet Recruiting
18
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
19
Rainbow Babies
Cleveland, Ohio, United States, 44106
Not Yet Recruiting
20
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Not Yet Recruiting
21
Oregon Health & Science University
Portland, Oregon, United States, 97239
Not Yet Recruiting
22
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Not Yet Recruiting
23
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Not Yet Recruiting
24
University of Texas, Southwestern
Dallas, Texas, United States, 75235
Actively Recruiting
25
Cook Children's Hospital
Fort Worth, Texas, United States, 76104
Actively Recruiting
26
Texas Children's Hospital/Baylor College of Medicine
Houston, Texas, United States, 77030
Actively Recruiting
27
Primary Children's Hospital
Salt Lake City, Utah, United States, 84113
Actively Recruiting
28
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Not Yet Recruiting
29
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
30
Children's Hospital at Westmead
Westmead, New South Wales, Australia
Actively Recruiting
31
Sydney Children's Hospital
Sydney, Australia
Not Yet Recruiting
Research Team
B
Benjamin N Brookhart
CONTACT
E
Ellynore Florendo
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
4
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