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AzTrials

Allogeneic Stem Cell TransplantationPhase 2NCT06297629

A Phase I/II Trial to Assess the Efficacy and Toxicity of ASTX727 (Oral Decitabine/Cedazuridine) for the Treatment of Hematological Neoplasms After Allogeneic Stem Cell Transplantation

A Phase 2 study testing ASTX727 and Donor Lymphocyte Infusion in people with Allogeneic Stem Cell Transplantation, run by M.D. Anderson Cancer Center at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

To learn if ASTX727 given alone or in combination with donor lymphocyte infusion (DLI) can help to control certain types of hematological neoplasms (blood-based cancers) after a stem cell transplant. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Allogeneic Stem Cell Transplantation
What is being tested?
ASTX727 and Donor Lymphocyte Infusion
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
1 siteUnited States
Study dates
1 Jul 2024to 11 Jul 2024
Phase
Phase 2
Are there results?
None posted in the registry

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

  • Diagnosis of AML and MDS according to World Health Organization (WHO) classification that underwent first or second allogeneic HSCT with either peripheral blood or bone marrow as the source of the hematopoietic stem cells.
  • Age 18 to 75 years old.
  • High risk patients defined per cohorts as below:
  • Cohort #1: AML and MDS patients in morphological remission with persistence or reappearance of MRD by flow cytometry or molecular after allogeneic stem cell transplantation who are beyond day 100 after allogeneic stem cell transplantation.
  • When MRD is detected by flow cytometry, disease level at or above the sensitivity level of the test will be required.
  • MRD level at or above 0.1%.
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  • When MRD is detected by molecular testing, disease level at or above the sensitivity level of the test will be required.
  • The limit of detection is 0.01%
  • Cohort #2: High risk AML and MDS patients who are in complete remission morphologically with no evidence of minimal residual disease by flow cytometry or cytogenetic or molecular testing within 100 days after allogeneic stem cell transplantation.
  • MDS patients:
  • Moderate-high, high or very high-risk groups by International Prognostic Scoring System-Molecular (IPSS-M) classification if classification is available (58).
  • TP53 with biallelic mutations(49).
  • Therapy-related MDS(50).
  • Presence of mutation ASXL1, SRSF2, DNMT3A, RUNX1, U2AF1, TP53, EZH2, STAG2, CBL, NRAS, BCOR ((49, 51-54))
  • Bone marrow blast count 10% or higher prior to allogeneic stem cell transplantation.
  • AML patients
  • Cytogenetics and molecular features consistent with adverse risk group by 2017 European LeukemiaNet classification for AML(55)
  • Presence of MRD by multi-color flow cytometry or cytogenetics or molecular studies by the last bone marrow evaluation prior to HSCT.
  • Primary induction failure defined as absence of complete remission after two different lines of anti-leukemia therapy following diagnosis.
  • Presence of active disease defined as bone marrow blast count >5% but <= 20% at the time of HSCT.
  • Patients transplanted beyond first CR.
  • Therapy-related AML.
  • Overexpression of EVI1 (52)
  • All patients in cohort #2:
  • Post-transplant bone marrow consistent with complete remission with no evidence of minimal residual disease by flow-cytometry or cytogenetics or molecular testing.
  • Adequate engraftment within 7 days prior to starting study drug: Absolute neutrophil count (ANC) >/= 1.0 x 109/L without daily use of myeloid growth factor (G-CSF) for at least 7 days; and,platelet >/= 30 x 109/L without platelet transfusion within 1 week.
  • ECOG performance status of 0, 1, or 2.
  • Creatinine clearance greater or equal than 40 cc/min as defined by the Cockcroft-Gault Equation*.
  • Males (mL/min) :(140-age) IBW (kg) / 72(serum creatinine(mg/dl)) Females (mL/min):0.85(140-age) IBW (kg) / 72(serum creatinine(mg/dl)).
  • Serum bilirubin </= 1.5 x upper limit of normal (ULN). Aspartate transaminase (AST) or alanine transaminase (ALT) </= 2.5 x ULN. Alkaline phosphatase </= 2.5 x UL.
  • No active bleeding.
  • No clinical evidence of life-threatening infection. Capable of understanding the investigational nature, potential risks and benefits of the study, and able to provide valid informed consent.
  • Negative serum or urine pregnancy test for women with reproductive potential. The only subjects who will be exempt from this criterion are postmenopausal women (defined as women who have been amenorrheic for > 12 months) or subjects who have been surgically sterilized or otherwise proven sterile.
  • Females of childbearing potential must refrain from becoming pregnant and commit to either apply highly effective method of birth control (two reliable methods of birth control) or continue abstinence from heterosexual intercourse during study period and for at least 6 months after last dose of ASTX727. Male subjects who are sexually active with a women of childbearing potential (WOCBP) and who have not had vasectomies must be willing to use a barrier method of contraception and refrain from sperm donation from initial study drug until 3 months after last dose of study drug.

Who cannot join

  • Use of any anti-leukemic agents after MRD is documented (note that the use of these anti-leukemic agents given as post-transplant maintenance therapy is allowed in this study, e.g., subcutaneous or oral 5-Azacytidine or FLT3 inhibitors for maintenance, for cohort #1 patients. However, all those agents will be discontinued once the patient enrolls into the current trial for cohort #1.
  • Use of any of the following after transplantation and prior to starting study therapy for cohort #2. Anti-leukemic agents given as post-transplant maintenance therapy (e.g., subcutaneous or oral 5-Azacytidine or FLT3 inhibitors for maintenance).
  • Overall grade II-IV acute GVHD. However, upon complete resolution of acute GVHD-related symptoms, patients are eligible for enrollment if they are on prednisone 0.5 mg/kg daily dose or lower, tacrolimus, sirolimus and ruxolitinib.
  • Chronic GvHD, moderate or severe by NIH criteria.
  • Active uncontrolled systemic fungal, bacterial or viral infection. However, patients receiving anti-microbial agents including antibiotics, antiviral and antifungal therapies are allowed if hemodynamically stable.
  • Symptomatic or uncontrolled arrhythmias.
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  • Significant active cardiac disease within the previous 6 months, including:
  • New York Hear Association (NYHA) class III or IV congestive heart failure;
  • Unstable angina or angina requiring surgical or medical intervention, and/or; Myocardial infarction.
  • Known active viral infection with Human Immunodeficiency Virus (HIV), Hepatitis B Virus (HBV) or Hepatitis C Virus (HCV).
  • Patients with known active hepatitis B virus (HBV) infection will be excluded because of potential effects on immune function and/or drug interactions. However, if a patient has HBV history with an undetectable HBV load by polymerase chain reaction (PCR), no liver-related.
  • complications, and is on definitive HBV therapy that is not contraindicated on this study, then he/she would be eligible for study.
  • Patients with known active hepatitis C virus (HCV) infection will be excluded because of potential effects on immune function and/or drug interactions. However, if a patient with a history of HCV infection has received definitive therapy (and is now HCV viral load negative), or if a patient has a reactive HCV antibody test but has an undetectable viral load by PCR, then he/she would be eligible.
  • Patients with known active HIV infection will be excluded out of concern for the drug-drug interaction with venetoclax and highly active antiretroviral therapy (HAART).
  • Prior history of solid tumors other than AML and MDS, unless the subject has been free of the disease for >/= 1 year. However, subjects with the following history/concurrent conditions are allowed:
  • Basal or squamous cell carcinoma of the skin;
  • Carcinoma in situ of the cervix;
  • Carcinoma in situ of the breast;
  • Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, node, metastasis [TNM] clinical staging system).

Trial locations and contacts

1 site in 1 country.

United States1 site

  • MD Anderson Cancer Center

    Houston, Texas, 77030

Other trials in United States

About this study

To learn if ASTX727 given alone or in combination with donor lymphocyte infusion (DLI) can help to control certain types of hematological neoplasms (blood-based cancers) after a stem cell transplant.

Read the detailed description

Primary Objectives

  • To determine the safety and toxicity of ASTX727 with or without donor lymphocyte infusion (type, frequency, severity of adverse events [AEs] and relationship of AEs to ASTX727) in the study population (cohort #1 and cohort #2).
  • To estimate the complete response (CR) rate of ASTX727 with or without donor lymphocyte infusion (DLI) for the eradication of minimal residual disease (MRD) in participants with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), after hematopoietic stem cell transplantation (cohort #1).
  • Estimate relapse-free survival (RFS) with the use of ASTX727 as maintenance therapy in participants with high-risk AML and MDS after study entry following hematopoietic stem cell transplantation (cohort #2).

Secondary Objectives

  • Participants with creatinine clearance 40-60 cc/min at enrollment will be observed and reported for safety and toxicity separately.
  • To determine the overall response and response duration in cohort #1, in participants with MRD detected in the post-transplant setting.
  • Duration of remission after study entry following allogeneic stem cell transplant for cohort #2.
  • To determine incidence of acute and chronic graft versus host disease (GvHD)
  • To evaluate overall survival (OS).

Exploratory Objective

  • To investigate possible relationships between protein and gene expression signatures/mutation profile and DNA methylation in predicting relapse-free survival time to the ASTX727.
  • To gain insight into clinically relevant genomic factors influencing transplant outcomes, especially into the mechanism of relapse after transplant.
  • To characterize the composition of different T-cell subsets, and to determine how those correlate with each other and with transplant outcomes.
Being tested
ASTX727, Donor Lymphocyte Infusion

What participants receive

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

  • ASTX727Drug

    Also called Inqovi

    Given by PO

  • Donor Lymphocyte InfusionDrug

    Also called DLI

    Given by Infusion

Study groups in NCT06297629
Study groupRoleWhat they receive
Group 1ExperimentalIf participants test positive for minimal residual disease (MRD), participants will be enrolled in Group 1. MRD refers to small numbers of cancer cells that remain in the body during or after treatment. Participants in this group will receive ASTX727 and DLI. If participants are enrolled in Group 1, the participant will take ASTX727 on Days 1-4 of each cycle.
Receives: Drug: ASTX727; Drug: Donor Lymphocyte Infusion
Group 2ExperimentalIf participants do not test positive for MRD, you will be enrolled in Group 2. Participants in this group will only receive ASTX727. If participants are enrolled in Group 2, the participant will take ASTX727 on Days 1-3 of each cycle.
Receives: Drug: ASTX727

What is being measured

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

  1. Safety and adverse events (AEs)

    Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0

    Measured: Through study completion; an average of 1 year

Timeline

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

  1. First posted7 Mar 2024
  2. Study start1 Jul 2024
  3. Primary completion11 Jul 2024
  4. Study completion11 Jul 2024

Why it stopped: Abandoned

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 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06297629
Sponsor's protocol ID
2021-0636
Other IDs
NCI-2024-01689
Status last verified
May 2026
First submitted
21 Feb 2024
Last updated
15 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ASTX727 and Donor Lymphocyte Infusion in people with Allogeneic Stem Cell Transplantation, run by M.D. Anderson 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 34 inclusion and 19 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: withdrawn, as of the sponsor's last update on 15 May 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

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?

Safety and adverse events (AEs) (time frame: Through study completion; an average of 1 year). 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 NCT06297629.

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 15 May 2026.

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