Actively Recruiting

Phase 2
Age: 18Years +
All Genders
NCT03683433

Enasidenib and Azacitidine in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia and IDH2 Gene Mutation

Led by M.D. Anderson Cancer Center · Updated on 2026-03-05

50

Participants Needed

1

Research Sites

469 weeks

Total Duration

On this page

Sponsors

M

M.D. Anderson Cancer Center

Lead Sponsor

N

National Cancer Institute (NCI)

Collaborating Sponsor

AI-Summary

What this Trial Is About

This phase II trial studies how well enasidenib and azacitidine work in treating patients with IDH2 gene mutation and acute myeloid leukemia that has come back (recurrent) or does not respond to treatment (refractory). Enasidenib and azacitidine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

CONDITIONS

Official Title

Enasidenib and Azacitidine in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia and IDH2 Gene Mutation

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients with AML or biphenotypic or bilineage leukemia (including a myeloid component) who have failed prior therapy
  • Patients with isolated extramedullary AML are eligible
  • Elderly patients over 60 years with newly diagnosed AML not eligible for intensive chemotherapy are eligible
  • AML patients with prior myelodysplastic syndrome or chronic myelomonocytic leukemia are eligible at AML diagnosis
  • Documented IDH2 gene mutation
  • ECOG performance status of 0, 1, or 2
  • Adequate kidney function with creatinine less than 2 unless related to disease
  • Total bilirubin less than twice the upper limit of normal unless due to Gilbert's disease or leukemic involvement
  • AST and/or ALT less than 3 times the upper limit of normal unless due to leukemic involvement
  • Written informed consent provided
  • Oral hydroxyurea and/or cytarabine (up to 2 g/m2) allowed before study therapy if needed
  • CNS prophylaxis or continuation of therapy for controlled CNS disease allowed
  • Females must be surgically or biologically sterile, postmenopausal for at least 12 months, or have a negative pregnancy test within 72 hours before treatment
  • Women of childbearing potential must agree to use contraception during and for 3 months after treatment
  • Males must be surgically or biologically sterile or agree to use contraception during and for 3 months after treatment
Not Eligible

You will not qualify if you...

  • Patients with t(15;17) karyotypic abnormality or acute promyelocytic leukemia (FAB class M3-AML)
  • Active and uncontrolled infections or other comorbidities such as uncontrolled hypertension, congestive heart failure NYHA class III/IV, or significant arrhythmias
  • Any medical, psychological, or social condition that may interfere with study participation or patient safety
  • Pregnant or breastfeeding

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

M D Anderson Cancer Center

Houston, Texas, United States, 77030

Actively Recruiting

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Research Team

C

Courtney DiNardo

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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