Enasidenib for IDH2-Mutated AML After Stem Cell Transplant
This study is looking at whether enasidenib (AG-221) can help prevent acute myeloid leukemia (AML) from coming back in patients who have a specific gene change (IDH2 mutation) after they've had a stem cell transplant. Enasidenib works by blocking certain enzymes that cancer cells need to grow. Researchers want to see how many side effects occur with enasidenib and how well it helps patients live longer without their leukemia returning. To join, you need to be at least 18 years old, have an IDH2 mutation, and have received a stem cell transplant. The study aims to enroll 35 participants.
- Study design
- This is an interventional study, meaning participants will receive a specific treatment. It is a Phase II trial, which typically involves a smaller number of participants to evaluate effectiveness and safety.
- What's involved
- Participants will take enasidenib by mouth once daily for 28 days, with this cycle repeating for up to 24 cycles. This continues as long as the treatment is working and side effects are manageable.
- Compensation
- Not stated in the trial record.
- Follow-up
- After treatment ends, participants will be followed for side effects for up to 30 days and periodically thereafter for up to 2 years.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT03728335
Enasidenib (AG-221) Maintenance Post Allogeneic HCT in Patients With IDH2 Mutation
Suspended
PHASE2Ages 18+InterventionalTreatmentCity of Hope Medical CenterInvestigator-initiated
~35 participants
Updated 2026-08-21 on ClinicalTrials.gov
What's tested:Enasidenib Mesylate
At a glance
Recruiting sites
0 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of adverse events (AEs)
Measured over Up to 30 days post treatment completion
Conditions
Where it's being run
2 sites across 2 statesCalifornia1
Florida1
Study leadership
- Amandeep Salhotra · PRINCIPAL_INVESTIGATOR · City of Hope Medical Center
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
Documented informed consent of the participant and/or legally authorized representative
Agreement to allow the use of archival tissue from diagnostic tumor biopsies
If unavailable, exceptions may be granted with study principal investigator (PI) approval
Eastern Cooperative Oncology Group (ECOG) =\< 2 or Karnofsky performance status (KPS) \>= 70
Recipients of allogeneic HCT - all stem cell sources including sibling, unrelated, mismatched related/unrelated, cord and haploidentical transplant patients will be included
Conditioning regimen: Investigator's choice based on center guidelines
GvHD prophylaxis: sirolimus + tacrolimus or tacrolimus + methotrexate or investigator choice
Patients must have acute myeloid leukemia (AML) with IDH2 mutation at diagnosis. Day 30 marrow post HCT should show evidence of morphologic remission with \< 5% bone marrow blasts. Patients with MRD either by flow cytometry or IDH2 mutation testing will be allowed
Patients with previous therapy with IDH2 inhibitors will be included
Absolute neutrophil count (ANC) \> 1000 (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated)
Hemoglobin \>= 9.5 gm% (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated)
Platelets \> 50,000/mm\^3 (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated)
Platelets \>= 20,000/mm\^3 (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated)
NOTE: Patients with lower counts can enroll if infection cytomegalovirus (CMV)/human herpesvirus 6 (HHV6) etc. is being treated actively
Total bilirubin =\< 1.5 X upper limit of normal (ULN) (unless has Gilbert's disease) (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated)
Total bilirubin \< 2.0 mg/dl-exception permitted in patients with Gilbert's Syndrome (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated)
Aspartate aminotransferases (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 2 x ULN, patients with abnormal liver function tests (LFTs) in the context of active GVHD will not be included (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated)
Creatinine clearance of \>= 40/min/1.73 m\^2 for participants with creatinine levels above institutional normal per 24 hour urine test or the Cockcroft-Gault formula (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated)
Corrected QT (QTc) =\< 480 ms
Note: To be performed within 28 days prior to day 1 of protocol therapy
Seronegative for human immunodeficiency virus (HIV) antigen/antibody (Ag/Ab) combo, hepatitis C virus (HCV), active hepatitis B virus (HBV) (surface antigen negative), and syphilis (rapid plasma reagin \[RPR\])
If positive, hepatitis C ribonucleic acid (RNA) quantitation must be performed
Women of childbearing potential (WOCBP): negative urine or serum pregnancy test
If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
Agreement by females and males of childbearing potential to use an effective method of birth control or abstain from heterosexual activity for the course of the study through at least 3 months after the last dose of protocol therapy
Childbearing potential defined as not being surgically sterilized (men and women) or have not been free from menses for \> 1 year (women only)
Exclusion
History of allergic reactions attributed to compounds of similar chemical or biologic composition to study agent
Active diarrhea considered clinically significant and may impair oral drug administration
Clinically significant uncontrolled illness
Active infection requiring antibiotics
Active infection. Patients with treated viral, bacterial or fungal infections that are controlled on therapy will be allowed to participate
Known history of immunodeficiency virus (HIV) or hepatitis B or hepatitis C infection
Diagnosis of Gilbert's disease
Other active malignancy. Participants with history of prior malignancy treated with curative intent who achieved complete response (CR) more than 2 years before study entry are eligible. This exclusion rule does not apply to non-melanoma skin tumors and in-situ cervical cancer
Females only: Pregnant or breastfeeding
Active grade II-IV acute GVHD and/or requiring systemic steroids with prednisone dose equivalent of \>= 0.25 mg/kg at end of 4 weeks
Any other condition that would, in the investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures
Prospective participants, who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics)
What this trial measures
- Incidence of adverse events (AEs)Up to 30 days post treatment completion
Toxicity will be graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v5.0).