Decitabine and Cedazuridine in Combination With Venetoclax for the Treatment of Patients Who Have Relapsed Acute Myeloid Leukemia After Donor Stem Cell Transplant

NCT05799079 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: VICCHEM2163
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Recurrent Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• Number of Participants That Achieved a Complete Response to Therapy

  9 months

Secondary Outcomes (6)

• Number of Participants That Achieved a Partial Response to Therapy

  9 months

• Rate of Morphologic Leukemia Free State (MLFS) Following Treatment With DEC-C/Venetoclax

  Up to 24 months post-treatment.

• Rate of Relapse Free Survival

  Up to 24 months post-treatment.

• Rate of Overall Survival

  Up to 24 months post-treatment.

• Number of Treatment-related Adverse Events

  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.

Study Arms (1)

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.

Drug: Venetoclax; Drug: Decitabine; Drug: Cedazuridine; Procedure: Bone Marrow Aspiration and Biopsy; Procedure: Biospecimen Collection

Interventions

Venetoclax DRUG

Given by mouth

Treatment (Venetoclax, DEC-C)

Decitabine DRUG

Given by mouth

Treatment (Venetoclax, DEC-C)

Cedazuridine DRUG

Given by mouth

Treatment (Venetoclax, DEC-C)

Bone Marrow Aspiration and Biopsy PROCEDURE

Undergo bone marrow biopsy

Treatment (Venetoclax, DEC-C)

Biospecimen Collection PROCEDURE

Undergo blood sample collection

Treatment (Venetoclax, DEC-C)

Eligibility Criteria

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

You may qualify if:

• 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)
• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Sanjay Mohan: lead
• National Comprehensive Cancer Network: collaborator
• Taiho Oncology, Inc.: collaborator

Study Sites (1)

Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, 37232, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

venetoclax; Decitabine; cedazuridine; Biopsy

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Azacitidine; Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides; Cytodiagnosis; Cytological Techniques; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Specimen Handling; Diagnostic Techniques, Surgical; Surgical Procedures, Operative; Investigative Techniques

Results Point of Contact

• Title: Sanjay Mohan
• Organization: Vanderbilt-Ingram Cancer Center

sanjay.mohan@vumc.org

615-936-8422

Study Officials

• Sanjay Mohan, MD

  Vanderbilt University/Ingram Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor of Medicine

Study Record Dates

• First Submitted: March 22, 2023
• First Posted: April 5, 2023
• Study Start: April 13, 2023
• Primary Completion: September 20, 2024
• Study Completion: September 20, 2024
• Last Updated: January 9, 2026
• Results First Posted: January 9, 2026

Record last verified: 2025-09

Locations

US (1)