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

Launched by M.D. ANDERSON CANCER CENTER · Sep 21, 2018

Keywords

ClinConnect Summary

This clinical trial is studying the effectiveness of two medications, enasidenib and azacitidine, in treating patients with a type of blood cancer called acute myeloid leukemia (AML) who have a specific genetic change known as the IDH2 gene mutation. The trial focuses on patients whose cancer has either come back after treatment or has not responded to previous therapies. By blocking certain enzymes, these medications may help slow down or stop cancer cells from growing.

To be eligible for this trial, patients must be over 60 years old and have been diagnosed with AML or related conditions, like biphenotypic or bilineage leukemia, that have not improved with other treatments. Patients with a documented IDH2 gene mutation are particularly needed. Participants can expect to receive the study medications and will be closely monitored for their health and response to the treatment. It's also important for potential participants to be in reasonably good health overall, as certain medical conditions might prevent them from joining. If you're considering this trial, you’ll need to provide informed consent and may have to meet specific health requirements before starting.

Gender

ALL

Eligibility criteria

• 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