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Acute Myeloid LeukemiaPhase 2NCT03728335

Enasidenib (AG-221) Maintenance Post Allogeneic HCT in Patients With IDH2 Mutation

A Phase 2 study testing Enasidenib Mesylate in people with Acute Myeloid Leukemia, run by City of Hope Medical Center at 2 sites in United States.

Suspended

Paused. Enrolment is on hold for now and may resume.

Official record
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Trial summary

This phase II trial studies the side effects of using enasidenib as maintenance therapy in treating patients with acute myeloid leukemia with IDH2 mutation following donor stem cell transplant. Enasidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Acute Myeloid Leukemia
What is being tested?
Enasidenib Mesylate
Could I receive a placebo?
No, everyone receives an active treatment
How many people
35 participants
Where
2 sitesUnited States
Study dates
23 Dec 2019to 11 Jan 2027
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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)

Who cannot join

  • 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
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  • 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)

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • City of Hope Medical Center

    Duarte, California, 91010

  • Moffitt Cancer Center

    Tampa, Florida, 33612

Other trials in United States

About this study

Official title: Phase 2 Trial of Enasidenib (AG-221) Maintenance Post Allogeneic Hematopoietic Cell Transplantation in Patients With IDH2 Mutation

This phase II trial studies the side effects of using enasidenib as maintenance therapy in treating patients with acute myeloid leukemia with IDH2 mutation following donor stem cell transplant. Enasidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Read the detailed description

PRIMARY OBJECTIVES:

  1. Evaluate the efficacy of enasidenib as post-HCT maintenance therapy in patients with mIDH2 AML, at 2-years post-HCT.

SECONDARY OBJECTIVES:

  1. Assess overall (OS) and leukemia-free survival (LFS) at 1 and 2 years post-HCT;
  2. Estimate the cumulative incidence of chronic GVHD at 1 and 2 years post-HCT;
  3. Estimate the cumulative incidence of relapse, non-relapse mortality (NRM), GVHD and relapse free survival (GRFS) at 1 and 2 years post-HCT.

EXPLORATORY OBJECTIVES:

  1. Monitor disease status among subset of patients with MRD positive disease when start to receive Enasidenib by multiparameter flow cytometry post allogeneic HCT on patients BM on days +100 and +365;
  2. Investigate clearance of IDH2 mutation post HCT by NGS-PCR testing on the bone marrow specimens on days +100 and +365 and in peripheral blood every 3 months till 2 year follow up; and

OUTLINE:

Patients receive enasidenib orally (PO) once daily (QD) on days 1-28. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up at 30 days and periodically thereafter up to 2 years.

Being tested
Enasidenib Mesylate
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Enasidenib MesylateDrug

    Also called 2-Methyl-1-[(4-[6-(trifluoromethyl)pyridin-2-yl]-6-{[2-(trifluoromethyl)pyridin-4-yl]amino}-1,3,5-triazin-2-yl)amino]propan-2-ol Methanesulfonate, 2-Propanol, 2-Methyl-1-((4-(6-(trifluoromethyl)-2-pyridinyl)-6-((2-(trifluoromethyl)-4-pyridinyl)amino)-1,3,5-triazin-2-yl)amino)-, Methanesulfonate (1:1), AG-221 Mesylate, CC-90007

    Given PO

Study groups in NCT03728335
Study groupRoleWhat they receive
Treatment (enasidenib mesylate)ExperimentalPatients receive enasidenib PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity.
Receives: Drug: Enasidenib Mesylate

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of adverse events (AEs)

    Toxicity will be graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v5.0).

    Measured: Up to 30 days post treatment completion

Secondary outcomes (5)

  1. Overall survival (OS)

    Will be analyzed using the Kaplan-Meier curves.

    Measured: From starting enasidenib to date of death, assessed up to 2 years

  2. Leukemia free survival (LFS)

    Will be analyzed using the Kaplan-Meier curves.

    Measured: From starting enasidenib to date of relapse or death, assessed up to 2 years

  3. Time to relapse

    Time to relapse will be censored at the last disease assessment if patients are known to be alive and leukemia free.

    Measured: From starting enasidenib to date of relapse, assessed up to 2 years

  4. Non-relapse mortality (NRM)

    Will be analyzed using the curves of cumulative incidence.

    Measured: From starting enasidenib to date of death from other causes than relapse, assessed up to 2 years

  5. Graft versus host disease (GvHD)-free relapse free survival (GRFS)

    Will be analyzed using the Kaplan-Meier curves.

    Measured: At 1 year mark of starting enasidenib

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Nov 2018
  2. Study start23 Dec 2019
  3. Primary completion11 Jan 2027 (estimated)
  4. Study completion11 Jan 2027 (estimated)

Why it stopped: Accrual goal met

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
35 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03728335
Sponsor's protocol ID
18117
Other IDs
NCI-2018-02371, 18117
Status last verified
Aug 2026
First submitted
31 Oct 2018
Last updated
21 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Shallis RM, Podoltsev NA. Maintenance therapy for acute myeloid leukemia: sustaining the pursuit for sustained remission. Curr Opin Hematol. 2021 Mar 1;28(2):110-121. doi: 10.1097/MOH.0000000000000637.

    PubMed 33394722

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Enasidenib Mesylate in people with Acute Myeloid Leukemia, run by City of Hope Medical Center at 2 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 26 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: paused, as of the sponsor's last update on 21 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of adverse events (AEs) (time frame: Up to 30 days post treatment completion). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03728335, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Aug 2026.

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