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Anti-metabolites

Azacitidine + Enasidenib for Myelodysplastic Syndrome

Phase 2

Recruiting

Led By Courtney DiNardo

Research Sponsored by M.D. Anderson Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subjects must have an IDH2 gene mutation (IDH2-R140 or R172) as determined by local laboratory result

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

Must not have

Clinically significant gastrointestinal conditions or disorders that may interfere with study drug absorption, including prior gastrectomy

Subject has received a prior targeted IDH2 inhibitor

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 3 years

Awards & highlights

No Placebo-Only Group

Summary

This trial looks at the side effects and effectiveness of azacitidine and enasidenib for treating patients with myelodysplastic syndrome caused by an IDH2 mutation.

Who is the study for?

This trial is for patients with IDH2-mutant myelodysplastic syndrome who have specific gene mutations, normal liver and kidney function, can consent to the study's requirements, and are not pregnant or nursing. Men must use contraception if with a partner of childbearing potential. It includes those new to treatment (Arm A) or who didn't respond well to previous therapies (Arm B).

What is being tested?

The trial is testing how effective azacitidine combined with enasidenib is in treating myelodysplastic syndrome linked to IDH2 mutations. The goal is to see if these drugs can halt cancer cell growth by inhibiting certain enzymes.

What are the potential side effects?

Potential side effects may include issues related to organ inflammation, digestive system disturbances due to enzyme inhibition, fatigue from anemia management, and possible reactions at the infusion site where medication enters the body.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My cancer has an IDH2 gene mutation.

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I have been diagnosed with MDS, RAEB-T, or CMML.

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All my side effects from previous cancer treatments, except hair loss, are mild or gone.

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I did not respond to or relapsed after 6 cycles of treatment with hypomethylating agents.

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I can take care of myself and am up and about more than half of the day.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have major stomach issues that could affect medication absorption.

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I have previously been treated with a drug targeting IDH2.

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I do not have severe heart issues like recent heart attacks or advanced heart failure.

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I am currently pregnant or breastfeeding.

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I do not have an active, uncontrolled infection or HIV/Hepatitis B/C.

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I have an active brain or spinal cord disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of adverse events

Overall response rate

Secondary study objectives

Anti-tumor activity

Drug exposure levels

Event-free survival (EFS)

+2 more

Other study objectives

Biomarkers analysis

Side effects data

From 2023 Phase 2 trial • 6 Patients • NCT04281498

50%

Anemia

50%

White blood cell decreased

50%

Aspartate aminotransferase increased

50%

Headache

50%

Hyperuricemia

50%

Platelet count decreased

50%

Epistaxis

50%

Blood bilirubin increased

33%

Constipation

33%

Dyspnea

33%

Edema limbs

33%

Fever (Pyrexia)

33%

Hyperkalemia

33%

Bone pain

33%

Neutrophil count decreased

33%

Creatinine increased

33%

Leukocytosis

33%

Hypocalcemia

17%

Vertigo

17%

Abdominal pain

17%

Alkaline phosphatase increased

17%

Anorexia

17%

Back pain

17%

Belching

17%

Bloating

17%

Bruising

17%

Fall

17%

Fatigue

17%

Heart failure

17%

Hyperphosphatemia

17%

Hypertension

17%

Hypokalemia

17%

OTHER Ear lobe pain

17%

Oral pain

17%

Alanine aminotransferase increased

17%

Nasal congestion

17%

Dizziness

17%

OTHER COVID-19

17%

Skin hyperpigmentation

17%

OTHER Unspecified contact dermatitis

17%

Upper respiratory infection

17%

Hypermagnesemia

17%

Hypernatremia

17%

OTHER early satiety

17%

OTHER Night sweats

17%

OTHER Petechial

17%

Febrile neutropenia

17%

Muscle cramp

17%

OTHER Heartburn

100%

80%

60%

40%

20%

Study treatment Arm

Patients With MPN

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Arm II (enasidenib)Experimental Treatment2 Interventions

Patients relapsed and/or refractory to HMA therapy receive enasidenib PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Group II: Arm I (enasidenib, azacitidine)Experimental Treatment3 Interventions

Patients who are HMA-naive receive enasidenib PO QD on days 1-28 and azacitidine IV over 30-60 minutes or SC on days 1-7. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Azacitidine

2012

Completed Phase 3

~1440

Enasidenib

2020

Completed Phase 2

~610