Using enasidenib and azacitidine to treat patients with recurrent or refractory acute myeloid leukemia and IDH2 gene mutation

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase II trial investigates the effectiveness of enasidenib mesylate in combination with azacitidine for patients suffering from recurrent or refractory acute myeloid leukemia (AML) with an IDH2 gene mutation. The study aims to measure the overall response rate, duration of response, event-free survival, and overall survival among participants. Patients will receive azacitidine either subcutaneously or intravenously for the first week of each treatment cycle, while enasidenib will be administered orally daily. The trial also explores the safety of the combination therapy and the potential for achieving minimal residual disease negativity.

Why it matters

Who can join: the full rules

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

Where it's running

• M D Anderson Cancer Center
  Houston, Texas, United States Recruiting

Study contacts

• Principal investigator: Courtney DiNardo — M.D. Anderson Cancer Center
• Study coordinator: Courtney DiNardo
• Email: cdinardo@mdanderson.org
• Phone: 713-794-1141

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.