Targeted drug aims to stop blood cancer return after transplant
NCT ID NCT05233618
First seen Jul 16, 2026 · Last updated Jul 17, 2026 · Updated 1 time
Summary
This phase 1 trial tests whether the drug tagraxofusp can help prevent relapse in people with certain blood cancers (acute myeloid leukemia, myelodysplastic syndrome, myelofibrosis, or chronic myelomonocytic leukemia) who have recently received a stem cell transplant. Participants receive up to about 9 cycles of tagraxofusp, which targets cancer cells that carry a protein called CD123. The study focuses on safety and how much of the drug patients can tolerate.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- tagraxofusp (also known as Elzonris), a drug that targets CD123-positive cells
- What this could lead to
- If successful, this approach could help prevent relapse after stem cell transplant for certain blood cancers, potentially improving long-term survival.
- What could go wrong
- This is an early phase 1 trial with a small number of participants, so safety and effectiveness are not yet proven. Side effects, including serious ones, are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 44 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jul 2022
- Expected to finish
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Dec 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 75 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. The patient is ≥18 years old and ≤ 75 years old. 2. The patient has a life expectancy of \>6 months. 3. The patient has an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0-2. 4. The patient has adequate baseline organ function, including cardiac, renal, and hepatic function within 28 days of start of therapy: * Left ventricular ejection fraction (LVEF) ≥ 50% as measured by multigated acquisition scan (MUGA) or 2-dimensional (2-D) echocardiogram (ECHO) and no clinically significant abnormalities on a 12-lead electrocardiogram (ECG) * Serum Creatinine ≤ 1.5 mg/dL * Bilirubin ≤1.5 mg/dL * Aspartate transaminase (AST) and alanine transaminase (ALT) ≤2.5 times the upper limit of normal (ULN) * Absolute neutrophil count (ANC) ≥0.5 × 10⁹/L * Platelets ≥ 80,000/mm\^3 * Serum albumin ≥3.2 (note that albumin infusions are not permitted in order to enable eligibility) 5. Patient meets the 2016 WHO diagnostic criteria for MF, is CD 123+, and has an IPSS/DIPSS/DIPSS-plus intermediate-1 with anemia (Hb \< 10g/dl), splenomegaly (\> 12 cm), leukocytosis (WBC \> 25K) intermediate-2 or high-risk disease pre transplant. Or Patient has a 2016 WHO-defined diagnosis of CMML (persistent monocytosis ≥1 × 10⁹/L for at least 3 months, with other causes excluded, and monocytes ≥10% of WBC in peripheral blood, no criteria and no previous history of CML, ET, PV, and acute promyelocytic leukemia) pre transplant and is CD123+ Or Patient has 2016 WHO-defined CMML-1 (2-4% blasts in peripheral blood and/or 5-9% blasts in bone marrow) and CMML-2 (5-19% blasts in peripheral blood and/or 10-19% blasts in bone marrow, and/or presence of Auer rods) pre transplant and is CD 123+ Or Patient has CD 123+ AML in morphologic remission pre transplant Or Patient has Intermediate or high risk MDS by IPSS-R or moderate or high risk by IPSS-M pre transplant and has had no morphologic progression of disease post-transplant. Receipt of first allogeneic stem cell transplant (related, unrelated, haploidentical or cord blood) 60-120 days prior to study registration 6. Patient is in morphologic remission according to bone marrow biopsy completed within 30 days prior to planned start of study treatment 7. Provision of signed and dated informed consent form 8. Stated willingness to comply with all study procedures and availability for the duration of the study 9. For females and males of reproductive potential: agreement to use adequate contraception for at least one month prior to screening, during study participation and for an additional one week after the end of study drug administration. Other (non-study) medications may require participants to use adequate contraception for longer. 10. For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner. Other (non-study) medications may require participants to use adequate contraception for longer. 11. Agreement to adhere to Lifestyle Considerations throughout study duration Exclusion Criteria: 1. Treatment with any disease-related therapy, including radiation therapy or investigational agent, within 14 days of study entry 2. Previous treatment with tagraxofusp or known hypersensitivity to any components of the drug product 3. Active malignancy and/or cancer history (excluding myeloproliferative disorders and concomitant myeloid malignancies as specified in the inclusion criteria) that can confound the assessment of the study endpoints. Patients with a past cancer history (within 2 years of entry) and/or ongoing active malignancy or substantial potential for recurrence must be discussed with the Sponsor before study entry. Patients with the following neoplastic diagnoses are eligible: non-melanoma skin cancer, carcinoma in situ (including superficial bladder cancer), cervical intraepithelial neoplasia, or organ-confined prostate cancer with no evidence of progressive disease. 4. Known active or suspected disease involvement of the central nervous system (CNS) 5. Receiving \> 10 mg prednisone daily for GVHD 6. Overall Grade 2 or greater acute GVHD (per Magic criteria) at time of registration 7. Pregnant or breast feeding 8. Requirement of supplemental oxygen 9. Clinically significant cardiovascular disease (e.g., uncontrolled or any New York Heart Association Class 3 or 4 congestive heart failure, uncontrolled angina, history of myocardial infarction or stroke within 6 months of study entry, uncontrolled hypertension or clinically significant arrhythmias not controlled by medication) 10. Uncontrolled, clinically significant pulmonary disease (e.g., chronic obstructive pulmonary disease, pulmonary hypertension) that in the opinion of the Investigator would put the patient at significant risk for pulmonary complications during the study 11. Uncontrolled intercurrent illness including, but not limited to, uncontrolled infection, disseminated intravascular coagulation, or psychiatric illness/social situations that would limit compliance with study requirements 12. Known positive status for human immunodeficiency virus or active or chronic Hepatitis B or Hepatitis C 13. Receiving treatment for known or suspected fungal infection (prophylaxis is acceptable) 14. Known positive SARS-COV-2 test within 3 weeks of study entry. Exception: Tests that reflect past, resolved infection where the patient is determined to NOT be infectious, according to an infectious disease specialist, do not exclude the patient from participation. 15. Pedal edema ≥ grade 2
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Thomas Jefferson University
Philadelphia, Pennsylvania, 19107, United States
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University of Virginia
Charlottesville, Virginia, 22901, United States
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Other studies related to the condition(s) this trial covers.
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