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Acute Myeloid LeukemiaPhase 1NCT07737769

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

A Phase 1 study testing Allogeneic Hematopoietic Stem Cell Transplantation, Arsenic Trioxide and Biospecimen Collection in people with Acute Myeloid Leukemia and Myelodysplastic Syndrome, run by University of Michigan Rogel Cancer Center at 1 site in United States.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
Search all trials

Trial 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). Read more about this study

Who is it for?
Ages 21 years and older
What is being tested?
Allogeneic Hematopoietic Stem Cell Transplantation, Arsenic Trioxide and Biospecimen Collection
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
1 siteUnited States
Study dates
Nov 2026to Nov 2029
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 21 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)
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  • 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

Who cannot join

  • 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
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  • 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

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Tracey Churay contact

1-800-865-1125

tchuray@med.umich.edu

United States1 site

  • University of Michigan Rogel Cancer Center

    Ann Arbor, Michigan, 48109

    Tracey Churay, 800-865-1125, tchuray@med.umich.edu

    John M. Magenau

Other trials in United States

About this study

Official title: A Phase I Non-Randomized Study of TP53 Reactivation With Arsenic Trioxide in Allogeneic Transplantation for Acute Myeloid Leukemia and Myelodysplastic Syndrome

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.

Being tested
Allogeneic Hematopoietic Stem Cell Transplantation, Arsenic Trioxide, Biospecimen Collection, Bone Marrow Biopsy, Busulfan
Medical subject headings
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes

What participants receive

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

  • Allogeneic Hematopoietic Stem Cell TransplantationProcedure or surgery

    Also called Allogeneic, Allogeneic Hematopoietic Cell Transplantation, Allogeneic Stem Cell Transplantation, HSC

    Given IV

  • Arsenic TrioxideDrug

    Also called Arsenic (III) Oxide, Arsenic Sesquioxide, Arsenous Acid, Arsenous Acid Anhydride

    Given IV

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection

    Undergo blood sample collection

  • Bone Marrow BiopsyProcedure or surgery

    Also called Biopsy of Bone Marrow, Biopsy, Bone Marrow

    Undergo bone marrow biopsy

  • BusulfanDrug

    Also called 1, 4-Bis[methanesulfonoxy]butane, BUS, Busilvex, Bussulfam

    Given IV

  • FludarabineDrug

    Also called Fluradosa

    Given fludarabine

Study groups in NCT07737769
Study groupRoleWhat they receive
Treatment (arsenic trioxide)ExperimentalPatients 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.
Receives: Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Drug: Arsenic Trioxide; Procedure: Biospecimen Collection; Procedure: Bone Marrow Biopsy; Drug: Busulfan; Drug: Fludarabine

What is being measured

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

  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.

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

Secondary outcomes (8)

  1. Progression free survival (PFS)

    Malignancy relapse is defined as: evidence of persistent morphological or cytogenetic findings of acute leukemia or myelodysplastic syndrome consistent with pretransplant features occurring after primary neutrophil engraftment or day 35, whichever occurs sooner. Progression-free survival (PFS) is defined as the duration of time from HCT (Day 0) to time of death or progression of primary malignancy detected by bone marrow assessment or other clinical monitoring. Will be estimated using Kaplan-Meier methods, with specific focus on estimates and 95% confidence intervals for PFS at 6- and 12-months. If no loss to follow-up is observed, then PFS will be estimated using observed proportions.

    Measured: At 6 and 12 months post HCT

  2. Absolute neutrophil recovery

    Engraftment for neutrophils is defined as the first of three consecutive days in which the absolute neutrophil count is > 500/uL.

    Measured: From day 0 to day 35 post HCT

  3. Incidence of severe cardiac arrythmia

    Defined as grade 3 or greater cardiac arrythmia by CTCAE v 5.0.

    Measured: From baseline to day 35 post HCT

  4. Incidence of hepatic sinusoidal obstruction syndrome/veno-occlusive disease

    Any Grade 3 or greater SOS/VOD by CTCAE v5.0 by day 35 post HCT will be considered for meeting this endpoint.

    Measured: From baseline to day 35 post HCT

  5. PFS

    Defined as whether a subject is alive and free of relapse. Will be estimated using Kaplan-Meier methods, with specific focus on estimates and 95% confidence intervals for PFS at 6- and 12-months. If no loss to follow-up is observed, then PFS will be estimated using observed proportions.

    Measured: At 12 months post HCT

  6. Incidence of non-relapse mortality (NRM)

    An event for NRM is death without prior evidence of relapse/progression of the primary disease, where relapse/progression is treated as a competing risk. Will be estimated using the methods of Fine and Gray in order to treat death as a competing event, rather than a censoring event.

    Measured: At 6 and 12 months post HCT

  7. Incidence of acute graft versus host disease

    Will be estimated using the methods of Fine and Gray in order to treat death as a competing event, rather than a censoring event.

    Measured: At 6 months post HCT

  8. Incidence and severity of chronic graft versus host disease

    Will be estimated using the methods of Fine and Gray in order to treat death as a competing event, rather than a censoring event.

    Measured: At 6 and 12 months post HCT

Timeline

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

  1. First posted30 Jul 2026
  2. Study startNov 2026 (estimated)
  3. Primary completionNov 2028 (estimated)
  4. Study completionNov 2029 (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
20 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07737769
Sponsor's protocol ID
UMCC 2025.124
Other IDs
NCI-2026-05320, HUM00287897
Status last verified
Jul 2026
First submitted
27 Jul 2026
Last updated
30 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: PI can be contacted for questions and specific data requests.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Allogeneic Hematopoietic Stem Cell Transplantation, Arsenic Trioxide and Biospecimen Collection in people with Acute Myeloid Leukemia and Myelodysplastic Syndrome, run by University of Michigan Rogel Cancer Center at 1 site 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 21 inclusion and 11 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: opening soon, as of the sponsor's last update on 30 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 1 site 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?

Contact Tracey Churay (1-800-865-1125, tchuray@med.umich.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of dose limiting toxicities (time frame: From baseline to day 35 post hematopoietic stem cell transplant (HCT)). 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 NCT07737769.

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 30 Jul 2026.

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