Recruiting
Phase 1

Arsenic Trioxide

Sponsor:

University of Michigan Rogel Cancer Center

Code:

NCT07737769

Conditions

Acute Myeloid Leukemia

Myelodysplastic Syndrome

Eligibility Criteria

Sex: All

Age: 21+

Healthy Volunteers: Not accepted

Interventions

Allogeneic Hematopoietic Stem Cell Transplantation

Arsenic Trioxide

Biospecimen Collection

Bone Marrow Biopsy

Busulfan

Study Details

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

Study ID

NCT07737769

Start date

Oct 26, 2026

Status verified date

Nov, 2026

Completion date

Nov, 2029

Anticipated

Primary completion date

Nov, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 21+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • AML or MDS patients eligible to receive first allogeneic HCT. Eligibility inclusion for HCT is defined as per University of Michigan bone marrow transplant (BMT) standard of care criteria
  • 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
  • Availability of a peripheral blood stem cell (PBSC) product
  • Ability to understand and the willingness to sign a written informed consent
  • Willingness to agree to use adequate contraception methods (subjects of reproductive potential only):

  • Females of reproductive potential must agree to use effective contraception (e.g., hormonal contraception, intrauterine device, tubal occlusion, vasectomized partner, abstinence) during treatment with ATO and for 6 months after the final dose.
  • Males with female partners of reproductive potential must agree to use effective contraception during treatment with ATO and for 3 months after the final dose

Exclusion Criteria:

  • 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
  • History of severe cardiac conduction abnormalities (complete heart block, Left Bundle Branch Block, Torsades de Pointes, or other ventricular arrythmias). History of any cardiac arrhythmia (e.g. rate controlled atrial fibrillation) is not an exclusion
  • 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

Study Design

Enrollment

20 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment (arsenic trioxide)

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.

Interventions

Allogeneic Hematopoietic Stem Cell Transplantation

Given IV

Arsenic Trioxide

Given IV

Biospecimen Collection

Undergo blood sample collection

Bone Marrow Biopsy

Undergo bone marrow biopsy

Busulfan

Given IV

Fludarabine

Given fludarabine

Primary outcome measure

  • Incidence of dose limiting toxicities [ Time Frame: From baseline to day 35 post hematopoietic stem cell transplant (HCT) ]

Central Contacts and Locations

Central contacts

Tracey Churay

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

Locations

University of Michigan Rogel Cancer Center

Recruiting

Ann Arbor, Michigan, United States, 48109

Contacts

Tracey Churay

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

Principal Investigator:

John M. Magenau

More Information

Sponsor

University of Michigan Rogel Cancer Center

Last update posted

Nov 12, 2026

Last verified

Nov, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-11-13. This information was provided to ClinicalTrials.gov by University of Michigan Rogel Cancer Center on 2026-11-12. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.