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

NCT05233618 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: HSR210434
• Study Type: interventional
• Target: 44
• Locations: 1 country
• Sites: 2

Brief 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. 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.

Conditions

Myelofibrosis; Chronic Myelomonocytic Leukemia; Acute Myeloid Leukemia

Keywords

Hematopoietic Stem Cell Transplant

Outcome Measures

Primary Outcomes (3)

• Incidence and severity of grade ≥ 3 adverse events

  Severity is based upon CTCAE v5 criteria.

  Through about 30 days following last infusion of tagraxofusp

• Dose limiting toxicities

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

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

• Percent of planned tagraxofusp dose received

  During cycles 1-4 only

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

Secondary Outcomes (2)

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

  Participants will be followed through 2 years after date of HCT

• Frequency and severity of acute GVHD grades II-IV and chronic GVHD

  Participants will be followed through 2 years after date of HCT

Study Arms (1)

Tagraxofusp (escalating doses)

EXPERIMENTAL

IV 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)

Drug: Tagraxofusp

Interventions

Tagraxofusp DRUG

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

Also known as: SL-701, Elzonris

Tagraxofusp (escalating doses)

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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.
• 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+
• 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+
• Patient has CD 123+ AML in morphologic remission pre transplant

You may not qualify if:

• 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
• 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
• 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

Sponsors & Collaborators

• Karen Ballen, MD: lead

Study Sites (2)

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

RECRUITING

University of Virginia

Charlottesville, Virginia, 22901, United States

RECRUITING

MeSH Terms

Conditions

Primary Myelofibrosis; Leukemia, Myelomonocytic, Chronic; Leukemia, Myeloid, Acute

Interventions

tagraxofusp

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myelodysplastic-Myeloproliferative Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Karen Ballen

  UVA

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Samantha Brooks

CONTACT

4349823365; SNB3GC@uvahealth.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief of the Hematology/Oncology Division and the Medical Director of the Stem Cell Transplant Program

Study Record Dates

• First Submitted: January 19, 2022
• First Posted: February 10, 2022
• Study Start: July 13, 2022
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): October 1, 2026
• Last Updated: May 28, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)