Recruiting
Phase 1
Phase 2

Venetoclax with Chemotherapy

Sponsor:

M.D. Anderson Cancer Center

Code:

NCT03214562

Conditions

High Risk Myelodysplastic Syndrome

Recurrent Acute Myeloid Leukemia

Refractory Acute Myeloid Leukemia

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Cytarabine

Filgrastim

Fludarabine

Idarubicin

Pegfilgrastim

Study Details

Brief summary:

This phase Ib/II trial studies the best dose and side effects of venetoclax and how well it works when given with combination chemotherapy in treating patients with newly diagnosed acute myeloid leukemia or acute myeloid leukemia that has come back or does not respond to treatment. Venetoclax may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as fludarabine, cytarabine, filgrastim and idarubicin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving venetoclax together with combination chemotherapy may work better in treating patients with acute myeloid leukemia.

Conditions

High Risk Myelodysplastic Syndrome

Recurrent Acute Myeloid Leukemia

Refractory Acute Myeloid Leukemia

Study ID

NCT03214562

Start date

Sep 26, 2017

Status verified date

Mar, 2026

Completion date

Sep 30, 2027

Anticipated

Primary completion date

Sep 30, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Diagnosis of AML by World Health Organization (WHO) criteria. Patients with high risk myelodysplastic syndrome (MDS) as defined by the presence of >= 10% blasts are also eligible at the discretion of the principal investigator
  • Patients older than 65 who are deemed fit to receive intensive chemotherapy by the treating physician will be eligible after discussion with the principal investigator (PI).
  • Eastern Cooperative Oncology Group (ECOG) performance status of =< 2
  • Creatinine clearance >= 30 mL/min based on the Cockcroft-Gault equation
  • Total bilirubin < 1.5 x upper limit of normal (ULN) unless increase is due to Gilbert's disease or leukemic involvement
  • Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) < 3 x ULN unless considered due to leukemic involvement
  • Ability to understand and provide signed informed consent
  • Male subjects must agree to refrain from unprotected sex and sperm donation from initial study drug administration until 90 days after the last dose of study drug
  • Only patients who are relapsed, refractory, or intolerant of standard AML therapy will be eligible for Part 1 (minimum of 1 prior line of AML-directed therapy)

Exclusion Criteria:

  • Patients with t(15;17) karyotypic abnormality or acute promyelocytic leukemia (French-American-British \[FAB\] class M3-AML)
  • Patients having received any prior BCL2 inhibitor therapy
  • Subject has known active central nervous system (CNS) involvement with AML
  • Patients with New York Heart Association (NYHA) class III or IV congestive heart failure or left ventricular ejection fraction (LVEF) < 40% by echocardiogram or multi-gated acquisition (MUGA) scan
  • Patients with a history of myocardial infarction within the last 6 months or unstable / uncontrolled angina pectoris or history of severe and/or uncontrolled ventricular arrhythmias
  • Patients with known infection with human immunodeficiency virus (HIV) or active hepatitis B or C
  • Patients with known dysphagia, short-gut syndrome, or other conditions that would affect the ingestion or gastrointestinal absorption of drugs administered orally
  • Subject has any other significant medical or psychiatric history that in the opinion of the investigator would adversely affect participation in this study
  • Subject has a white blood cell count > 25 x 10{9}/L. (Note: hydroxyurea is permitted to meet this criterion)
  • Nursing women, women of childbearing potential (WOCBP) with positive urine pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception (a) appropriate method(s) of contraception include oral or injectable hormonal birth control, intrauterine device (IUD), and double barrier methods (for example a condom in combination with a spermicide)

Study Design

Enrollment

116 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment (venetoclax, FLAG-IDA)

See detailed description.

Interventions

Cytarabine

Given IV

Filgrastim

Given SC

Fludarabine

Given IV

Idarubicin

Given IV

Pegfilgrastim

Given SC

Venetoclax

Given PO

Primary outcome measure

  • Overall response rate (ORR) [ Time Frame: Up to 6 years ]
  • CR/CRi rate [ Time Frame: Up to 6 years ]
  • Hematologic response [ Time Frame: Up to 6 years ]
  • Duration of response [ Time Frame: From the date of initial response, assessed up to 6 years ]
  • Event-free survival [ Time Frame: From the date of treatment initiation, assessed up to 6 years ]
  • Overall survival [ Time Frame: Up to 6 years ]
  • Anti-tumor activity [ Time Frame: Up to 6 years ]
  • Pharmacodynamic markers [ Time Frame: Up to 6 years ]
  • Drug exposure levels [ Time Frame: Up to 6 years ]
  • Overall incidence and severity of all adverse events [ Time Frame: Up to 6 years ]

Central Contacts and Locations

Central contacts

DiNardo, MD

(713) 794-1141cdinardo@mdanderson.org

Locations

M D Anderson Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Courtney DiNardo

713-794-1141cdinardo@mdanderson.org

Principal Investigator:

Courtney DiNardo

More Information

Sponsor

M.D. Anderson Cancer Center

Last update posted

Mar 5, 2026

Last verified

Mar, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by M.D. Anderson Cancer Center on 2026-03-05.