Recurrent Acute Myeloid LeukemiaPhase 2NCT05799079
Decitabine and Cedazuridine in Combination With Venetoclax for the Treatment of Patients Who Have Relapsed Acute Myeloid Leukemia After Donor Stem Cell Transplant
A Phase 2 study testing Venetoclax, Decitabine and Cedazuridine in people with Recurrent Acute Myeloid Leukemia, run by Sanjay Mohan at 1 site in United States.
Trial summary
This phase II trial tests how well decitabine and cedazuridine (DEC-C) works in combination with venetoclax in treating acute myeloid leukemia (AML) in patients whose AML has come back after a period of improvement (relapse) after a donor stem cell transplant. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Recurrent Acute Myeloid Leukemia
- What is being tested?
- Venetoclax, Decitabine and Cedazuridine
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Apr 2023to 20 Sep 2024
- Phase
- Phase 2
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age >= 18 years at the time of signing the Informed Consent Form (ICF); must voluntarily sign an ICF and meet all study requirements
- History of morphologically confirmed AML (per World Health Organization [WHO] diagnostic criteria) with evidence of disease recurrence (>= 5% blasts consistent with prior disease) that occurs after allogeneic hematopoietic cell transplantation (HCT). Patients transplanted for another indication (e.g., myelodysplastic syndrome/chronic myelomonocytic leukemia [MDS/CMML]) who relapse with AML are eligible to enroll
- White blood cells (WBC) must be less than 25,000/ul for at least three days prior to cycle 1, day 1 (C1D1) (hydroxyurea allowed)
- A bone marrow biopsy must be performed and tissue collected for entrance to the trial
- Eastern Cooperative Oncology Group Performance Status of 0 - 2
- Alanine transaminase (ALT) serum glutamic pyruvic transaminase (SGPT) and/or aspartate aminotransferase (AST) serum glutamic-oxaloacetic transaminase (SGOT) less than or equal to 3x upper limit of normal (ULN)
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- Total bilirubin < 1.5 x ULN
- Patients with Gilbert's syndrome (hereditary indirect hyperbilirubinemia) must have a total bilirubin of < 3 x ULN
- Calculated creatinine clearance >= 30 ml/min (per the Cockroft-Gault formula)
- Willingness to abide by all study requirements, including contraception, maintenance of a pill diary, and acceptance of recommended supportive care medications
Who cannot join
- Prior relapse or progression while receiving venetoclax or other commercially available or investigational BCL-2 inhibitor
- Anticancer therapy, including investigational agents =< 2 weeks or =< 5 half-lives of the drug, whichever is shorter, prior to C1D1. (Use of hydroxyurea is permitted)
- Inadequate recovery from toxicity attributed to prior anti-cancer therapy to =< Grade 1 (National Cancer Institute Common Terminology Criteria for Adverse Events [NCI CTCAE] version [v]5.0), excluding alopecia or fatigue
- History of allogeneic HCT, or other cellular therapy product, within 3 months of signing consent
- Clinically active acute or chronic graft versus host disease (GVHD). Patients must be off calcineurin inhibitors for at least 4 weeks to be eligible
- Radiation therapy or major surgery within 3 weeks of signing consent
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- Active, uncontrolled infection. Patients with infection under active treatment and controlled with antibiotics are eligible. Prophylaxis is acceptable
- Inability to tolerate oral medication, presence of poorly controlled gastrointestinal disease, or dysfunction that could affect study drug absorption
- Active documented central nervous system leukemia
- Concurrent treatment with a non-permitted concomitant medication
- Other malignancy IF currently being treated or likely to be treated in next 6 months except for basal or squamous cell carcinoma of the skin or cervical carcinoma in situ
- Pregnancy or breastfeeding females
- Known chronic alcohol or drug abuse
- Clinically significant cardiovascular disease with major event or cardiac intervention within the past 6 months (e.g. percutaneous intervention, coronary artery bypass graft, documented cardiac heart failure) as determined by the investigator
- Any other condition deemed by the investigator to make the patient a poor candidate for clinical trial and/or treatment with investigational agents
Trial locations and contacts
1 site in 1 country.
United States1 site
Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee, 37232
About this study
Official title: Phase 2 Study of Decitabine and Cedazuridine in Combination With Venetoclax for AML Relapse After Allogeneic Hematopoietic Cell Transplantation
This phase II trial tests how well decitabine and cedazuridine (DEC-C) works in combination with venetoclax in treating acute myeloid leukemia (AML) in patients whose AML has come back after a period of improvement (relapse) after a donor stem cell transplant. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Giving DEC-C in combination with venetoclax may kill more cancer cells in patients with relapsed AML.
Read the detailed description
PRIMARY OBJECTIVE:
- To assess the effect of DEC-C/venetoclax on the investigator-assessed composite complete remission (CR) rate (CR/complete remission with partial hematologic recovery [CRh]/complete remission with incomplete hematologic recovery [CRi]).
SECONDARY OBJECTIVES:
- To assess the rate of partial response (PR) and morphologic leukemia free state (MLFS) following treatment with DEC-C/venetoclax. II. To assess the relapse free survival of patients treated with DEC-C/venetoclax.
III. To assess overall survival of patients treated with DEC-C/venetoclax. IV. To assess the safety and tolerability of DEC-C/venetoclax in the post-hematopoietic cell transplant (HCT) setting.
- To assess the rates of measurable residual disease negativity in patients achieving a CR.
OUTLINE:
Patients receive venetoclax orally (PO) daily for 28 days in a 28-day cycle. Patients receive DEC-C PO daily on days 1-5 of a 28-day cycle. Patients undergo bone marrow biopsy and aspiration and blood sample collection throughout the study.
- Conditions
- Recurrent Acute Myeloid Leukemia
- Being tested
- Venetoclax, Decitabine, Cedazuridine, Bone Marrow Aspiration and Biopsy, Biospecimen Collection
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VenetoclaxDrug
Given by mouth
DecitabineDrug
Given by mouth
CedazuridineDrug
Given by mouth
Bone Marrow Aspiration and BiopsyProcedure or surgery
Undergo bone marrow biopsy
Biospecimen CollectionProcedure or surgery
Undergo blood sample collection
| Study group | Role | What they receive |
|---|---|---|
| Treatment (Venetoclax, DEC-C) | Experimental | Patients receive venetoclax PO daily for 28 days in a 28-day cycle. Patients receive DEC-C PO daily on days 1-5 of a 28-day cycle. Patients undergo bone marrow biopsy and aspiration and blood sample collection throughout the study. Receives: Drug: Venetoclax; Drug: Decitabine; Drug: Cedazuridine; Procedure: Bone Marrow Aspiration and Biopsy; Procedure: Biospecimen Collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants That Achieved a Complete Response to Therapy
Measured: 9 months
Secondary outcomes (6)
Number of Participants That Achieved a Partial Response to Therapy
Measured: 9 months
Rate of Morphologic Leukemia Free State (MLFS) Following Treatment With DEC-C/Venetoclax
Measured: Up to 24 months post-treatment.
Rate of Relapse Free Survival
Relapse free survival was estimated by the Kaplan-Meier Method
Measured: Up to 24 months post-treatment.
Rate of Overall Survival
Overall survival was estimated using the Kaplan-Meier method.
Measured: Up to 24 months post-treatment.
Number of Treatment-related Adverse Events
List of adverse events that were attributed as related to study treatment is reported. All occurrences of a particular adverse event is reported.
Measured: The one patient that was enrolled on the study was on treatment for 3 months and adverse event data were followed during treatment and 35 days after completing treatment.
Rate of Measurable Residual Disease Negativity in Patients Achieving a CR
Measured: Up to 24 months post-treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2023
- Study start13 Apr 2023
- Primary completion20 Sep 2024
- Study completion20 Sep 2024
Why it stopped: Study was terminated early due to lack of accrual and loss of funding.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Recurrent Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanjay Mohan
- Sponsor type
- University, hospital or other
- Collaborators
- National Comprehensive Cancer Network, Taiho Oncology, Inc.
- Responsible party
- Sponsor-investigator, Sanjay Mohan, Vanderbilt-Ingram Cancer Center
- Investigators
- Sanjay Mohan, MD, Principal investigator, Vanderbilt University/Ingram Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05799079
- Sponsor's protocol ID
- VICCHEM2163
- Status last verified
- Sep 2025
- First submitted
- 22 Mar 2023
- Last updated
- 9 Jan 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided