Clinical trial for Hematologic Malignancy
Official title Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies
ClinicalTrials.gov ID: NCT05476770
What this study is testing
What is Tagraxofusp?
Tagraxofusp is an investigational medicine, given as an IV infusion (through a vein), being studied as a potential treatment for hematologic malignancy.
Also referred to as Elzonris.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study looks at how safe tagraxofusp is and what dose works best when given alone or with chemotherapy in pediatric patients with relapsed or refractory blood cancers.
- Who it's for
- Children and young adults with relapsed or refractory hematologic malignancies, including those with Down syndrome in the first part of the study.
- What's involved
- Participants receive tagraxofusp by itself and later may receive it together with standard chemotherapy, along with regular medical visits, lab tests, and monitoring for side effects.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 21
You may be able to join if
- Patients must be ≥ 1 and ≤21 years of age at the time of study enrollment. Diagnosis
- Relapsed and/or refractory hematologic malignancy (including, but not limited to, acute lymphoblastic leukemia, acute myeloid leukemia...
- Tumor cells must demonstrate surface expression of CD123 at the time of enrollment by flow cytometry or immunohistochemistry, as defined by the local...
- Second or greater relapse; or
- Refractory after 2 or more chemotherapy cycles; or
You likely can't join if
- Hydroxyurea: Hydroxyurea can be initiated and/or continued for up to 24 hours prior to the start of protocol therapy.
- "Maintenance-style" therapy: therapy including vincristine (dosed a maximum of one-time weekly), oral 6-mercaptopurine, oral methotrexate (dosed a...
- Hematopoietic stem cell transplant: Patients who have experienced their relapse after a HSCT are eligible, provided they have no evidence of acute or...
- Hematopoietic growth factors: It must have been at least 7 days since the completion of therapy with granulocyte colony stimulating factor (GCSF) or...
- Biologic (anti-neoplastic agent): At least 7 days after the last dose of a biologic agent.
- Monoclonal antibodies: Maximum of 3 half-lives of the antibody or 21 days (whichever is shorter) must have elapsed after the last dose of monoclonal...
See the full eligibility criteria
Who can join
- Patients must be ≥ 1 and ≤21 years of age at the time of study enrollment. Diagnosis
- Relapsed and/or refractory hematologic malignancy (including, but not limited to, acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, mixed phenotype acute leukemia, acute undifferentiated...
- Tumor cells must demonstrate surface expression of CD123 at the time of enrollment by flow cytometry or immunohistochemistry, as defined by the local institution. Disease Status: Monotherapy, Part 1
- Second or greater relapse; or
- Refractory after 2 or more chemotherapy cycles; or
- First relapse after primary chemotherapy-refractory disease; or
- BPDCN in first relapse or refractory after 1 or more chemotherapy cycles Combination therapy, Part 2
- First or greater relapse; or
- Refractory after 2 or more chemotherapy cycles; or
- \>5% blasts in the bone marrow aspirate or biopsy by morphology or flow cytometry
- Histologic verification of relapse
- Measurable disease documented by radiographic criteria or bone marrow
- Patients in Part 1 may have sites of non-CNS extramedullary disease, but no CNS disease. Patients in Part 2 may have CNS disease and/or other non-CNS extramedullary disease.
- Patients with Down syndrome are eligible to participate in Part 1 only. Performance Level
- Karnofsky \> 50% for patients \> 16 years of age and Lansky \> 50% for patients ≤ 16 years of age (See Appendix I for Performance Scales).
- Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy, defined as resolution of all such toxicities to ≤ Grade 2 or lower per the inclusion/
What rules you out
- Hydroxyurea: Hydroxyurea can be initiated and/or continued for up to 24 hours prior to the start of protocol therapy.
- "Maintenance-style" therapy: therapy including vincristine (dosed a maximum of one-time weekly), oral 6-mercaptopurine, oral methotrexate (dosed a maximum of one-time weekly), intrathecal therapy (dosed a maximum of...
- Hematopoietic stem cell transplant: Patients who have experienced their relapse after a HSCT are eligible, provided they have no evidence of acute or chronic Graft-versus-Host Disease (GVHD) and are at least 100 days...
- Hematopoietic growth factors: It must have been at least 7 days since the completion of therapy with granulocyte colony stimulating factor (GCSF) or other growth factors at the time of enrollment.
- Biologic (anti-neoplastic agent): At least 7 days after the last dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the...
- Monoclonal antibodies: Maximum of 3 half-lives of the antibody or 21 days (whichever is shorter) must have elapsed after the last dose of monoclonal antibody.
- Immunotherapy: At least 30 days from last infusion of chimeric antigen receptor T cell (CART) therapy or tumor vaccine.
- ≥ 84 days must have passed, from the end of therapy, if patient received prior total body irradiation (TBI).
- ≥ 42 days must have passed, from the end of therapy, if patient received craniospinal irradiation (CSI).
- ≥ 14 days must have passed after whole brain radiotherapy or stereotactic radiation therapy.
- No washout period is required for: i. Extramedullary site other than CNS that is a maximum 10 x 10 cm total radiation non-CNS field. If the field is \> 10 x 10 cm, a 14-day washout period is required. ii.
- Patients should not be known to be refractory to red blood cell or platelet transfusions.
- 1 to \< 2 years old - Male: 0.6, Female: 0.6
- 2 to \< 6 years old - Male:0.8, Female: 0.8
- 6 to \< 10 years old - Male: 1, Female: 1
- 10 to \< 13 years old - Male: 1.2, Female: 1.2
- 13 to \< 16 years old - Male: 1.5, Female: 1.4
- Total bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x institutional upper limit of normal for age
- SGPT (ALT) and SGOT (AST) must be less than 3x institutional upper limit of normal.
- Shortening fraction of ≥27% by echocardiogram, or
- Pulse oximetry \> 94% on room air (\> 90% if at high altitude)
- No evidence of dyspnea at rest and no exercise intolerance. Reproductive Function
- Female patients of childbearing potential must have a negative urine or serum pregnancy test confirmed within 2 weeks prior to enrollment.
- Female patients with infants must agree not to breastfeed their infants while on this study.
- Patients with CNS disease are not eligible for Part 1.
- Patients with isolated CNS disease are not eligible for Part 1 or Part 2.
- Patients with isolated non-CNS disease are eligible for Part 1 and Part 2. Concomitant Medications
- Corticosteroids - Patients receiving corticosteroids for disease control who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible.
- Investigational Drugs - Patients who are currently receiving another investigational drug are not eligible.
- Anti-cancer Agents - Patients who are currently receiving or may receive while on therapy, other anti-cancer agents, radiation therapy or immunotherapy are not eligible [with the exceptions being laid out in the...
- Positive blood culture within 48 hours of study enrollment;
- Fever above 38.2 within 48 hours of study enrollment with clinical signs of infection. Fever that is determined to be due to tumor burden is allowed if patients have documented negative blood cultures for at least 48...
- A positive fungal culture within 30 days of study enrollment.
- Active fungal, viral, bacterial, or protozoal infection requiring IV treatment. Chronic prophylaxis therapy to prevent infections is allowed.
- Patients will be excluded if they have a known allergy to any of the drugs used in the study.
- Patients will be excluded if they have significant concurrent disease, illness, psychiatric disorder or social issue that would compromise patient safety or compliance with the protocol treatment or procedures...
- Patients with DNA fragility syndromes (such as Fanconi anemia, Bloom syndrome) are excluded.
The study team makes the final eligibility decision.
Where it is enrolling
- Los Angeles, California, United States
- San Francisco, California, United States
- Denver, Colorado, United States
- Washington D.C., District of Columbia, United States
- Chicago, Illinois, United States
- Indianapolis, Indiana, United States
- Ann Arbor, Michigan, United States
- New York, New York, United States
- Cincinnati, Ohio, United States
- Dallas, Texas, United States
- Fort Worth, Texas, United States
- Houston, Texas, United States
- Salt Lake City, Utah, United States
- Milwaukee, Wisconsin, United States
- Westmead, New South Wales, Australia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 21 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Los Angeles, California, United States; San Francisco, California, United States; Denver, Colorado, United States; Washington D.C., District of Columbia, United States; Chicago, Illinois, United States; Indianapolis, Indiana, United States and 9 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.