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Azacitidine and Enasidenib for IDH2-Mutant Myelodysplastic Syndrome

This study is looking at how safe and effective two medicines, azacitidine and enasidenib, are for people with myelodysplastic syndrome (MDS) that has a specific change in the IDH2 gene. Azacitidine and enasidenib may work by blocking enzymes that help cancer cells grow. The study will test enasidenib alone, and enasidenib combined with azacitidine. You could be eligible if you have MDS, including certain types of acute myeloid leukemia (AML), and have the IDH2 gene mutation. The researchers want to see if these treatments reduce side effects and improve how patients respond to treatment over up to three years. This study is currently recruiting up to 63 participants.

Study design
This is an interventional study, meaning participants will receive specific treatments. It plans to enroll 63 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for adverse events and overall response rate for up to 3 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.

NCT03383575

Azacitidine and Enasidenib in Treating Patients With IDH2-Mutant Myelodysplastic Syndrome

Recruiting
PHASE2Ages 12+InterventionalTreatment
M.D. Anderson Cancer Center
~63 participants
Updated 2026-08-10 on ClinicalTrials.gov
What's tested:AzacitidineEnasidenibQuality-of-Life Assessment

At a glance

Recruiting sites
1 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of adverse events
Measured over Up to 3 years
+1 more outcome measured

Conditions

Acute Myeloid Leukemia
Blasts 20-30 Percent of Bone Marrow Nucleated Cells
Chronic Myelomonocytic Leukemia
IDH2 Gene Mutation
Myelodysplastic Syndrome With Excess Blasts
Recurrent High Risk Myelodysplastic Syndrome
Refractory High Risk Myelodysplastic Syndrome

Explore related trials

Acute Myeloid LeukemiaChronic Myelomonocytic LeukemiaMyelodysplastic SyndromeIDH2 Gene

Where it's being run

3 sites across 3 states
Maryland1
Ohio1
Texas1

Study leadership

  • Courtney DiNardo · PRINCIPAL_INVESTIGATOR · M.D. Anderson Cancer Center

Who to contact

Courtney DiNardo, MD
[email protected]
713-794-1141
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Signed, informed consent must be obtained prior to any study specific procedures
Subjects with a histologically confirmed diagnosis of MDS, including both MDS and refractory anemia with excess blasts in transformation (RAEB-T) (acute myeloid leukemia \[AML\] with 20-30% blasts and multilineage dysplasia by French-American-British \[FAB\] criteria) by World Health Organization (WHO), and chronic myelomonocytic leukemia (CMML) are eligible
Subjects must have an IDH2 gene mutation (IDH2-R140 or R172) as determined by local laboratory result
(Arm A only): Subject must be hypomethylating agent naive (i.e. prior azacitidine, decitabine, SGI-110 is exclusionary). Receipt of other MDS-directed therapy such as lenalidomide is allowed
(Arm A only): Subjects with high-risk MDS (i.e. International Prostate Symptom Score \[IPSS\] intermediate-2 or high-risk; or revised \[R\]-IPSS high or very-high risk). Patients with intermediate-1 risk by IPSS or intermediate risk by R-IPSS with high-risk molecular features including TP53, ASXL1, EZH2, and/or RUNX1 mutations are also eligible
(Arm B only): Subject must be relapsed or refractory to prior hypomethylating agent therapy, defined as prior receipt of 6 cycles of HMA therapy with failure to attain a response, or relapse after prior response to HMA therapy
Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
Serum bilirubin =\< 2 x the upper limit of normal (ULN) (except for patients with Gilbert's disease)
Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) =\< 3 x the laboratory ULN
Serum creatinine =\< 2 x the ULN
Able to understand and voluntarily sign a written informed consent, and willing and able to comply with protocol requirements
Resolution of all clinically significant treatment-related, non-hematological toxicities, except alopecia, from any previous cancer therapy to =\< grade 1 prior to the first dose of study treatment
Female patients of childbearing potential must have a negative serum or urine pregnancy test within 7 days of the first dose of study drug and agree to use dual methods of contraception during the study and for a minimum of 3 months following the last dose of study drug. Post-menopausal females (\> 45 years old and without menses for \> 1 year) and surgically sterilized females are exempt from these requirements. Male patients must use an effective barrier method of contraception during the study and for a minimum of 3 months following the last dose of study drug if sexually active with a female of childbearing potential

Exclusion

Any prior or coexisting medical condition that in the investigator's judgment will substantially increase the risk associated with the subject's participation in the study
Subject has received a prior targeted IDH2 inhibitor
Psychiatric disorders or altered mental status precluding understanding of the informed consent process and/or completion of the necessary study procedures
Active uncontrolled infection at study enrollment including known diagnosis of human immunodeficiency virus or chronic active hepatitis B or C infection
Clinically significant gastrointestinal conditions or disorders that may interfere with study drug absorption, including prior gastrectomy
Patients with known active central nervous system (CNS) disease, including leptomeningeal involvement
Impaired cardiac function, uncontrolled cardiac arrhythmia, or clinically significant cardiac disease including the following: a) New York Heart Association grade III or IV congestive heart failure, b) myocardial infarction within the last 6 months
Subjects with a corrected QT (QTc) \> 480 ms (QTc \> 510 msec for subjects with a bundle branch block at baseline
Nursing or pregnant women
Subjects with known hypersensitivity to study drugs or their excipients

What this trial measures

  • Incidence of adverse eventsUp to 3 years

    Will use the Bayesian method by Thall, Simon and Estey for toxicity monitoring. For purpose of toxicity monitoring, toxicity is defined as any grade 3 or higher treatment related-toxicities by Common Terminology Criteria for Adverse Events criteria.

  • Overall response rateUp to 3 years

    Defined as complete response (CR), partial response, and marrow CR assessed by International Working Group criteria. Will be estimated along with the 90% credible interval.