Status:

TERMINATED

Omacetaxine and Venetoclax for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Harboring Mutant RUNX1

Lead Sponsor:

M.D. Anderson Cancer Center

Conditions:

Hematopoietic and Lymphoid Cell Neoplasm

Recurrent Acute Biphenotypic Leukemia

Eligibility:

All Genders

18+ years

Phase:

PHASE1

PHASE2

Brief Summary

This phase Ib/II trial best dose, possible benefits and/or side effects of omacetaxine and venetoclax in treating patients with acute myeloid leukemia or myelodysplastic syndrome that has come back (r...

Detailed Description

PRIMARY OBJECTIVES: I. To determine the safety and tolerability and recommended phase 2 dose (RP2D) of omacetaxine in combination with venetoclax for patients with relapsed/refractory acute myeloid l...

Eligibility Criteria

Inclusion

  • Patients with a diagnosis of relapsed or refractory acute myeloid leukemia (AML) (or biphenotypic or bilineage leukemia including a myeloid component) or myelodysplastic syndrome
  • For myelodysplastic syndrome (MDS) patients, patients must have no response, progression, or relapse following at least 4 cycles of azacytidine or decitabine; and/or intolerance defined as grade \>= 3 drug-related toxicity precluding continued therapy
  • Age \>= 18 years
  • Subjects must have documented RUNX1 gene mutation
  • Eastern Cooperative Oncology Group (ECOG) performance status =\< 2
  • Creatinine \< 2 unless related to the disease
  • Direct bilirubin \< 2x upper limit of normal (ULN) unless increase is due to Gilbert's disease or leukemic involvement
  • Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \< 3x ULN unless considered due to leukemic involvement
  • In the absence of rapidly proliferative disease, the interval from prior treatment to time of initiation will be at least 7 days for cytotoxic or non-cytotoxic (i.e. immunotherapy) agents. Oral hydroxyurea and/or cytarabine (up to 2 g/m\^2) for patients with rapidly proliferative disease is allowed before the start of study therapy, as needed, for clinical benefit and after discussion with the principal investigator (PI)
  • 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
  • Willing and able to provide informed consent

Exclusion

  • Patients with t(15;17) karyotypic abnormality or acute promyelocytic leukemia (French-American-British \[FAB\] class M3-AML)
  • Patients with any concurrent uncontrolled clinically significant medical condition including active infection or psychiatric illness, which could place the patient at unacceptable risk of study treatment
  • Patients with active graft-versus-host-disease (GVHD) status post stem cell transplant (patients without active GVHD on chronic suppressive immunosuppression and/or phototherapy for chronic skin GVHD are permitted after discussion with the PI)
  • Patients with any severe gastrointestinal or metabolic condition which could interfere with the absorption of oral study medications
  • Known active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection or known human immunodeficiency virus (HIV) infection
  • 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
  • Appropriate highly effective 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)

Key Trial Info

Start Date :

December 17 2021

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

September 16 2024

Estimated Enrollment :

24 Patients enrolled

Trial Details

Trial ID

NCT04874194

Start Date

December 17 2021

End Date

September 16 2024

Last Update

November 3 2025

Active Locations (1)

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Page 1 of 1 (1 locations)

1

M D Anderson Cancer Center

Houston, Texas, United States, 77030