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Enasidenib and Azacitidine for AML with IDH2 Mutation

This study is looking at how well two drugs, enasidenib and azacitidine, work together for people with acute myeloid leukemia (AML) that has an IDH2 gene mutation. This is for AML that has come back or hasn't responded to previous treatments. It also includes older patients (over 60) with newly diagnosed AML who can't have strong chemotherapy. These drugs may help stop cancer cells from growing. The main goal is to see how many patients respond to this treatment. The study plans to enroll about 50 participants.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 50 participants.
What's involved
You would receive azacitidine by injection or IV and enasidenib mesylate by mouth daily. Treatment cycles repeat every 4-6 weeks.
Compensation
Not stated in the trial record.
Follow-up
After treatment, you would have follow-up appointments at 30 days and then every 3-6 months for up to 5 years.

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NCT03683433

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

Recruiting
PHASE2Ages 18+InterventionalTreatment
M.D. Anderson Cancer Center
~50 participants
Updated 2026-03-05 on ClinicalTrials.gov
What's tested:AzacitidineEnasidenib Mesylate

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Overall response rate (ORR)
Measured over Up to 5 years

Conditions

Acute Bilineal Leukemia
Acute Biphenotypic Leukemia
Chronic Myelomonocytic Leukemia
IDH2 Gene Mutation
Myelodysplastic Syndrome
Recurrent Acute Myeloid Leukemia
Refractory Acute Myeloid Leukemia

Explore related trials

Acute Bilineal LeukemiaAcute Biphenotypic LeukemiaAcute Myeloid LeukemiaChronic Myelomonocytic LeukemiaMyelodysplastic SyndromeIDH2 Gene

Where it's being run

1 sites across 1 states
Texas1

Study leadership

  • Courtney DiNardo · PRINCIPAL_INVESTIGATOR · M.D. Anderson Cancer Center

Who to contact

Courtney DiNardo
[email protected]
713-794-1141
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Patients with AML or biphenotypic or bilineage leukemia (including a myeloid component) who have failed prior therapy. Patients with isolated extramedullary AML are eligible. The World Health Organization (WHO) classification will be used for AML
Elderly (\> 60 years old) patients with newly diagnosed AML not eligible for intensive chemotherapy are also eligible
AML patients with prior history of myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) regardless of prior therapy received, are eligible at the time of diagnosis of AML
Subjects must have documented IDH2 gene mutation
Eastern Cooperative Oncology Group (ECOG) performance status =\< 3
Adequate renal function including creatinine \< 2 unless related to the disease
Total bilirubin \< 2 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
Provision of written informed consent
Oral hydroxyurea and/or cytarabine (up to 2 g/m2) 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). Concurrent therapy for central nervous system (CNS) prophylaxis or continuation of therapy for controlled CNS disease is permitted
Females must be surgically or biologically sterile or postmenopausal (amenorrheic for at least 12 months) or if of childbearing potential, must have a negative serum or urine pregnancy test within 72 hours before the start of the treatment
Women of childbearing potential must agree to use an adequate method of contraception during the study and until 3 months after the last treatment. Males must be surgically or biologically sterile or agree to use an adequate method of contraception during the study until 3 months after the last treatment

Exclusion

Patients with t(15;17) karyotypic abnormality or acute promyelocytic leukemia (French-American-British \[FAB\] class M3-AML)
Active and uncontrolled comorbidities including active uncontrolled infection, uncontrolled hypertension despite adequate medical therapy, active and uncontrolled congestive heart failure New York Heart Association (NYHA) class III/IV, clinically significant and uncontrolled arrhythmia as judged by the treating physician
Any other medical, psychological, or social condition that may interfere with study participation or compliance, or compromise patient safety in the opinion of the investigator
Pregnant or breastfeeding

What this trial measures

  • Overall response rate (ORR)Up to 5 years

    The study will estimate the ORR for the combination treatment, along with the Bayesian 95% credible interval. ORR will be defined as the proportion of patients who had CR (complete remission), CRh (complete remission with incomplete hematologic recovery), CRi (complete remission with incomplete count recovery), PR (partial response), and/or marrow clearance of blasts (MLFS).