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MyelofibrosisPhase 1NCT05233618

Study of Tagraxofusp for Post-Transplant Maintenance for Patients With CD 123+ AML, MDS, MF and CMML (HSCT 002)

A Phase 1 study testing Tagraxofusp in people with Myelofibrosis and Chronic Myelomonocytic Leukemia, run by Karen Ballen, MD at 2 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

In this study, tagraxofusp (Tag) is given to patients with CD 123+ myelofibrosis (MF), chronic myelomonocytic leukemia (CMML), and acute myeloid leukemia (AML) after allogeneic stem cell transplant (HCT) to help prevent relapse. Read more about this study

Who is it for?
Ages 18 to 75 years
What is being tested?
Tagraxofusp
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
44 participants
Where
2 sitesUnited States
Study dates
13 Jul 2022to 1 Dec 2026
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • The patient is ≥18 years old and ≤ 75 years old.
  • The patient has a life expectancy of >6 months.
  • The patient has an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0-2.
  • 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
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  • 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)
  • 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
  • Patient is in morphologic remission according to bone marrow biopsy completed within 30 days prior to planned start of study treatment
  • Provision of signed and dated informed consent form
  • Stated willingness to comply with all study procedures and availability for the duration of the study
  • 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.
  • 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.
  • Agreement to adhere to Lifestyle Considerations throughout study duration

Who cannot join

  • Treatment with any disease-related therapy, including radiation therapy or investigational agent, within 14 days of study entry
  • Previous treatment with tagraxofusp or known hypersensitivity to any components of the drug product
  • 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.
  • Known active or suspected disease involvement of the central nervous system (CNS)
  • Receiving > 10 mg prednisone daily for GVHD
  • Overall Grade 2 or greater acute GVHD (per Magic criteria) at time of registration
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  • Pregnant or breast feeding
  • Requirement of supplemental oxygen
  • 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)
  • 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
  • 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
  • Known positive status for human immunodeficiency virus or active or chronic Hepatitis B or Hepatitis C
  • Receiving treatment for known or suspected fungal infection (prophylaxis is acceptable)
  • 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.
  • Pedal edema ≥ grade 2

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • University of Virginia

    Charlottesville, Virginia, 22901

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107

Other trials in United States

About this study

Official title: Phase I Study of Tagraxofusp for Post-Transplant Maintenance for Patients With CD 123+ Acute Myeloid Leukemia, Myelodysplastic Syndrome, Myelofibrosis and Chronic Myelomonocytic Leukemia

In this study, tagraxofusp (Tag) is given to patients with CD 123+ myelofibrosis (MF), chronic myelomonocytic leukemia (CMML), and acute myeloid leukemia (AML) after allogeneic stem cell transplant (HCT) to help prevent relapse. Patients will receive up to about 9 cycles of treatment with Tag and have a bone marrow biopsy after cycle 4 and about 1 year after HCT.

Read the detailed description

Relapsed disease is the primary cause of treatment failure after hematopoietic cell transplant (HCT). In this study, patients are given increasing levels of tagraxofusp (Tag) to evaluate the safety of each dose. Participants will start treatment with Tag starting between 60 and 120 days following HCT. Tag will be given by IV over about 15 minutes on days 1 through 3 of cycles 1-4 of treatment (28 day cycles) and then on days 1 and 2 of subsequent cycles, for up to 9 cycles or until disease progression or if you have a bad side effect. In the first cycle, Tag will be given while participants are inpatient. In all other cycles, Tag will be given outpatient and participants will be observed for 4 hours following each infusion. After about 4 cycles of treatment and again about 1 year after HCT, participants will have a bone marrow biopsy and also take a questionnaire about their quality of life. During the study, participants will have their blood checked regularly to monitor for side effects.

Being tested
Tagraxofusp
Keywords
Hematopoietic Stem Cell Transplant
Medical subject headings
Primary Myelofibrosis, Leukemia, Myelomonocytic, Chronic, Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TagraxofuspDrug

    Also called SL-701, Elzonris

    inpatient on days 1-3 of cycles 1-4 and days 1-2 of additional cycles

Study groups in NCT05233618
Study groupRoleWhat they receive
Tagraxofusp (escalating doses)ExperimentalIV tagraxofusp on days 1-3 of cycles 1-4 and days 1-2 of additional cycles for up to 9 cycles (some participants could receive more if considered in their best interest)
Receives: Drug: Tagraxofusp

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence and severity of grade ≥ 3 adverse events

    Severity is based upon CTCAE v5 criteria.

    Measured: Through about 30 days following last infusion of tagraxofusp

  2. Dose limiting toxicities

    Frequencies of certain, more severe, types of side effects from the study drug

    Measured: During cycles 1 and 2 (each cycle is 28 days) of study treatment for each participant

  3. Percent of planned tagraxofusp dose received

    During cycles 1-4 only

    Measured: Through cycle 4 (each cycle is 28 days) of study treatment for each participant

Secondary outcomes (2)

  1. Time from HCT to relapse and death or last contact.

    Duration

    Measured: Participants will be followed through 2 years after date of HCT

  2. Frequency and severity of acute GVHD grades II-IV and chronic GVHD

    Mild, moderate or severe per Magic criteria

    Measured: Participants will be followed through 2 years after date of HCT

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Feb 2022
  2. Study start13 Jul 2022
  3. Primary completion1 Oct 2026 (estimated)
  4. Study completion1 Dec 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
44 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05233618
Sponsor's protocol ID
HSR210434
Status last verified
Jul 2026
First submitted
19 Jan 2022
Last updated
16 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Tagraxofusp in people with Myelofibrosis and Chronic Myelomonocytic Leukemia, run by Karen Ballen, MD at 2 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 27 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 16 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence and severity of grade ≥ 3 adverse events (time frame: Through about 30 days following last infusion of tagraxofusp). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05233618.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2026.

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