Arsenic Trioxide to Prevent Relapse in Patients Undergoing Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia or Myelodysplastic Syndrome

NCT07737769 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 3 other identifiers: UMCC 2025.124NCI-2026-05320HUM00287897
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

This phase I trial tests the safety, side effects, best dose and how well arsenic trioxide works to prevent relapse in patients undergoing allogenic stem cell transplantation for acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Chemotherapy drugs, such as arsenic trioxide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving arsenic trioxide may be safe, tolerable and/or effective in preventing relapse in patients undergoing allogenic stem cell transplant for AML or MDS.

Conditions

Acute Myeloid Leukemia; Myelodysplastic Syndrome

Outcome Measures

Primary Outcomes (1)

• Incidence of dose limiting toxicities

  Assessed via the incidence and severity of adverse events as graded by Common Terminology Criteria for Adverse Events (CTCAE) version (v) 5.0.

  From baseline to day 35 post hematopoietic stem cell transplant (HCT)

Secondary Outcomes (8)

• Progression free survival (PFS)

  At 6 and 12 months post HCT

• Absolute neutrophil recovery

  From day 0 to day 35 post HCT

• Incidence of severe cardiac arrythmia

  From baseline to day 35 post HCT

• Incidence of hepatic sinusoidal obstruction syndrome/veno-occlusive disease

  From baseline to day 35 post HCT

• PFS

  At 12 months post HCT

Study Arms (1)

Treatment (arsenic trioxide)

EXPERIMENTAL

Patients receive arsenic trioxide IV on days -6 to -2 and days +1 to +5 in the absence of disease progression or unacceptable toxicity. Patients also receive standard of care fludarabine and busulfan on days -5 to -2 and allogenic hematopoietic stem cells IV on day 0. Patients undergo bone marrow biopsy, and blood sample collection throughout the study.

Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Drug: Arsenic Trioxide; Procedure: Biospecimen Collection; Procedure: Bone Marrow Biopsy; Drug: Busulfan; Drug: Fludarabine

Interventions

Allogeneic Hematopoietic Stem Cell Transplantation PROCEDURE

Given IV

Also known as: Allogeneic, Allogeneic Hematopoietic Cell Transplantation, Allogeneic Stem Cell Transplantation, HSC, HSCT, Stem Cell Transplantation, Allogeneic

Treatment (arsenic trioxide)

Arsenic Trioxide DRUG

Given IV

Also known as: Arsenic (III) Oxide, Arsenic Sesquioxide, Arsenous Acid, Arsenous Acid Anhydride, Arsenous Oxide, ATO, Trisenox, White Arsenic

Treatment (arsenic trioxide)

Biospecimen Collection PROCEDURE

Undergo blood sample collection

Also known as: Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection

Treatment (arsenic trioxide)

Bone Marrow Biopsy PROCEDURE

Undergo bone marrow biopsy

Also known as: Biopsy of Bone Marrow, Biopsy, Bone Marrow

Treatment (arsenic trioxide)

Busulfan DRUG

Given IV

Also known as: 1, 4-Bis[methanesulfonoxy]butane, BUS, Busilvex, Bussulfam, Busulfanum, Busulfex, Busulphan, CB 2041, CB-2041, Glyzophrol, GT 41, GT-41, Joacamine, Methanesulfonic Acid Tetramethylene Ester, Methanesulfonic acid, tetramethylene ester, Mielucin, Misulban, Misulfan, Mitosan, Myeleukon, Myeloleukon, Myelosan, Mylecytan, Myleran, Sulfabutin, Tetramethylene Bis(methanesulfonate), Tetramethylene bis[methanesulfonate], WR-19508

Treatment (arsenic trioxide)

Fludarabine DRUG

Given fludarabine

Also known as: Fluradosa

Treatment (arsenic trioxide)

Eligibility Criteria

• Age: 21 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Presence of high-risk AML/MDS with probable biallelic TP53 deletion or mutation status, defined by one of the following in prior blood or bone marrow assessments:
• TP53 loss by karyotype (chromosome 17 or 17p deletion) or single nucleotide polymorphism (SNP) (Cancer Microarray) AND ≥ one clonal TP53 mutation by next generation sequencing (NGS) (VAF \> 10%).
• ≥ one TP53 mutation by NGS testing (with VAF \> 40%)
• ≥ two distinct TP53 mutation(s) by NGS (VAF \>10%)
• Evidence that at least one TP53 allele harbors a structural TP53 missense mutation. In cases where the mutation type is unclassified, molecular pathology assessment is required to determine if one of the lesions is a missense mutation (as described in NGS report)
• Patients may be enrolled with either active disease or in morphological remission, provided the bone marrow blast count on the pre-HCT assessment does not exceed 30%
• Age \> 21 years at enrollment and receiving allogeneic HCT in the adult transplant program
• Karnofsky performance score (KPS) ≥ 70%
• Documentation of adequate pulmonary function by forced expiratory volume and 1 second (FEV1) ≥ 50% of predicted, forced vital capacity (FVC) ≥ 50% of predicted and DLCO (corrected for hemoglobin) ≥ 50% of predicted (must meet all criteria at time of enrollment based on standard of care pre HCT testing)
• Transthoracic echocardiogram with ejection fraction ≥ 50% (must meet all criteria at time of enrollment based on standard of care pre HCT testing)
• Estimated glomerular filtration rate ≥ 50% ml/min/1.73m\^2 by the Cockcroft-Gault equation. Glomerular filtration rate (GFR) should be corrected for body surface area (BSA) (must meet all criteria at time of enrollment based on standard of care pre HCT testing)
• Total bilirubin ≤ 2 x upper limit of normal (unless directly attributed to Gilbert's Syndrome) (must meet all criteria at time of enrollment based on standard of care pre HCT testing)
• Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≤ 5 x upper limit of normal (must meet all criteria at time of enrollment based on standard of care pre HCT testing)
• Electrocardiogram (ECG) with corrected QT (QTc) ≤ 450 msec (using the Framingham correction) (must meet all criteria at time of enrollment based on standard of care pre HCT testing)
• Availability of an 8 of 8 HLA matched unrelated donor with matching at HLA A, B, C and DRB1

You may not qualify if:

• Uncontrolled infections. Patients still under therapy for presumed or proven infection are eligible provided there is clear evidence (radiologic, clinical and/or culture) that the infection is well controlled
• Patients may NOT have evidence or symptoms of central nervous system (CNS) disease at the time of enrollment
• HIV or human t-lymphotropic virus (HTLV) 1 / HTLV 2 (seropositivity and/or polymerase chain reaction \[PCR\] positivity)
• Pregnant and nursing mothers are excluded
• Any physical, psychological or psychosocial condition that, in the opinion of the investigator, would pose unacceptable risk to the patient
• Other malignancy requiring systemic therapy or with life expectancy of \< 1 year
• Receipt of prior allogeneic HCT
• QTc \> 450 msec (using the Framingham correction)
• Patients under 40 years of age at time of evaluation will be screened for Shwachman-Diamond syndrome (SDS), which is a rare, inherited disorder characterized primarily by exocrine pancreatic insufficiency, bone marrow failure, and skeletal abnormalities. These patients commonly progress to myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Given potential for increased liver toxicities in this population, these patients will be excluded from the study. All patients under 40 years old will be screened through the University of Michigan Prevention Genetics Clinic prior to enrollment
• Patients with known hypersensitivity to arsenic

Sponsors & Collaborators

• University of Michigan Rogel Cancer Center: lead

Study Sites (1)

University of Michigan Rogel Cancer Center

Ann Arbor, Michigan, 48109, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes

Interventions

Stem Cell Transplantation; Arsenic Trioxide; arsenous acid; Specimen Handling; Biopsy; Busulfan; fludarabine

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative; Arsenicals; Inorganic Chemicals; Oxides; Oxygen Compounds; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Cytodiagnosis; Cytological Techniques; Diagnostic Techniques, Surgical; Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds

Study Officials

• John M Magenau

  University of Michigan Rogel Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Tracey Churay

CONTACT

1-800-865-1125; tchuray@med.umich.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 27, 2026
• First Posted: July 30, 2026
• Study Start (Estimated): November 1, 2026
• Primary Completion (Estimated): November 1, 2028
• Study Completion (Estimated): November 1, 2029
• Last Updated: July 30, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)