NCT03728335

Brief 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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
35

participants targeted

Target at P25-P50 for phase_2

Timeline
8mo left

Started Jul 2019

Longer than P75 for phase_2

Geographic Reach
1 country

2 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress91%
Jul 2019Jan 2027

First Submitted

Initial submission to the registry

October 31, 2018

Completed
2 days until next milestone

First Posted

Study publicly available on registry

November 2, 2018

Completed
8 months until next milestone

Study Start

First participant enrolled

July 11, 2019

Completed
7.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 11, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 11, 2027

Last Updated

March 13, 2026

Status Verified

March 1, 2026

Enrollment Period

7.5 years

First QC Date

October 31, 2018

Last Update Submit

March 11, 2026

Conditions

Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (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).

    Up to 30 days post treatment completion

Secondary Outcomes (5)

  • Overall survival (OS)

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

  • Leukemia free survival (LFS)

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

  • Time to relapse

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

  • Non-relapse mortality (NRM)

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

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

    At 1 year mark of starting enasidenib

Other Outcomes (3)

  • Minimal residual disease (MRD) disappearance (bone marrow [BM])

    At days 100 and 365

  • IDH2 mutation clearance (BM and peripheral blood)

    At days 100 and 365 and up to 2 years

  • mIDH2 variant allele fraction (BM)

    At days 100 and 365

Study Arms (1)

Treatment (enasidenib mesylate)

EXPERIMENTAL

Patients 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.

Drug: Enasidenib Mesylate

Interventions

Enasidenib MesylateDRUG

Given PO

Also known as: 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, Enasidenib Methanesulfonate, Idhifa
Treatment (enasidenib mesylate)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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)
  • +11 more criteria

You may not qualify if:

  • 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
  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

City of Hope Medical Center

Duarte, California, 91010, United States

RECRUITING

Moffitt Cancer Center

Tampa, Florida, 33612, United States

RECRUITING

Related Publications (1)

  • 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.

    PMID: 33394722DERIVED

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

2-Propanolmethanesulfonic acidenasidenib

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

PropanolsAlcoholsOrganic Chemicals

Study Officials

  • Amandeep Salhotra

    City of Hope Medical Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 31, 2018

First Posted

November 2, 2018

Study Start

July 11, 2019

Primary Completion (Estimated)

January 11, 2027

Study Completion (Estimated)

January 11, 2027

Last Updated

March 13, 2026

Record last verified: 2026-03

Locations

US(2)