Recruiting
Phase 2

Enasidenib & Azacitidine

Sponsor:

M.D. Anderson Cancer Center

Code:

NCT03683433

Conditions

Acute Bilineal Leukemia

Acute Biphenotypic Leukemia

Chronic Myelomonocytic Leukemia

IDH2 Gene Mutation

Myelodysplastic Syndrome

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Azacitidine

Enasidenib Mesylate

Study Details

Brief summary:

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

Acute Bilineal Leukemia

Acute Biphenotypic Leukemia

Chronic Myelomonocytic Leukemia

IDH2 Gene Mutation

Myelodysplastic Syndrome

Study ID

NCT03683433

Start date

Sep 18, 2018

Status verified date

Mar, 2026

Completion date

Sep 20, 2027

Anticipated

Primary completion date

Sep 20, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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 Criteria:

  • 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

Study Design

Enrollment

50 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment (azacitidine, enasidenib mesylate)

Patients receive azacitidine SC or IV over 30 minutes on days 1-7 and enasidenib mesylate PO QD beginning on day 1. Cycles repeat every 4-6 weeks in the absence of disease progression or unacceptable toxicity.

Interventions

Azacitidine

Given SC or IV

Enasidenib Mesylate

Given PO

Primary outcome measure

  • Overall response rate (ORR) [ Time Frame: Up to 5 years ]

Central Contacts and Locations

Central contacts

Courtney DiNardo

713-794-1141cdinardo@mdanderson.org

Locations

M D Anderson Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Courtney DiNardo

713-794-1141

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 2026-09-10. This information was provided to ClinicalTrials.gov by M.D. Anderson Cancer Center on 2026-03-05.