Study on Enasidenib and Azacitidine for Treating Recurrent or Resistant Acute Myeloid Leukemia with IDH2 Gene Mutation

Sponsor: M.D. Anderson Cancer Center

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT03683433

Provider View

This clinical trial is testing how well the combination of enasidenib and azacitidine works in patients with acute myeloid leukemia (AML) that has returned or is not responding to treatment, specifically in those with an IDH2 gene mutation.

Patient Parameters

Program Overview

This phase II study is exploring the effectiveness of enasidenib and azacitidine in treating patients with acute myeloid leukemia (AML) that has come back or is not responding to treatment. These drugs may help stop cancer cells from growing by blocking certain enzymes needed for their growth. The study aims to see how well patients respond to this treatment, how long the response lasts, and the overall safety of the drug combination.

Description

The main goal of this study is to see how well enasidenib and azacitidine work together in treating patients with relapsed or refractory acute myeloid leukemia (AML) by measuring the overall response rate. Secondary goals include assessing how long the response lasts, event-free survival, and overall survival. The study will also evaluate the safety of the drug combination.

Exploratory goals include checking for minimal residual disease (MRD) using IDH2 mutation analysis and flow cytometry, exploring the relationship between protein and gene expression and treatment response, and examining the occurrence of IDH-inhibitor related differentiation syndrome (IDH-DS).

Patients will receive azacitidine either under the skin or through an IV for 30 minutes on days 1-7, and enasidenib by mouth once daily starting on day 1. Treatment cycles repeat every 4-6 weeks unless the disease progresses or side effects become unacceptable. After completing the study treatment, patients will be followed up at 30 days and then every 3-6 months for up to 5 years.

Eligibility Criteria

Inclusion Criteria

  • Have acute myeloid leukemia (AML) or a similar type of leukemia that has not responded to previous treatments. Patients with AML that has spread outside the bone marrow are also eligible.
  • Be over 60 years old with newly diagnosed AML and not eligible for intensive chemotherapy.
  • Have AML with a history of myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML).
  • Have a documented IDH2 gene mutation.
  • Have a performance status score of 3 or less, indicating the ability to carry out self-care.
  • Have adequate kidney function with creatinine levels below 2, unless related to the disease.
  • Have total bilirubin levels below 2 times the upper limit of normal, unless due to Gilbert's disease or leukemia.
  • Have liver enzyme levels (AST/ALT) below 3 times the upper limit of normal, unless due to leukemia.
  • Provide written informed consent.
  • May use oral hydroxyurea or cytarabine for rapidly growing disease before starting the study, after discussing with the principal investigator.
  • Women must be surgically or biologically sterile, postmenopausal, or have a negative pregnancy test before starting treatment.
  • Women of childbearing potential must agree to use contraception during the study and for 3 months after the last treatment. Men must agree to use contraception during the study and for 3 months after the last treatment.

Exclusion Criteria

  • Have a specific type of leukemia known as acute promyelocytic leukemia (APL) with a t(15;17) genetic abnormality.
  • Have active and uncontrolled health issues, such as infections, high blood pressure, heart failure, or irregular heartbeats.
  • Have any medical, psychological, or social conditions that could interfere with the study or compromise safety.
  • Be pregnant or breastfeeding.

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