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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 2016 Phase 1 & 2 trial • 21 Patients • NCT02273739

67%

Asthenia

67%

Decreased appetite

67%

Cough

67%

Dyspnoea

33%

Pyrexia

33%

Hyperglycaemia

33%

Musculoskeletal chest pain

33%

Tachypnoea

33%

Sinus tachycardia

33%

Hypoalbuminaemia

33%

Partial seizures

33%

Dysphagia

33%

Disease progression

33%

Sepsis

33%

Dehydration

33%

Mental status changes

33%

Pneumonia aspiration

33%

Anaemia

33%

Supraventricular tachycardia

33%

Constipation

33%

Nausea

33%

Fatigue

33%

Oedema peripheral

33%

Skin bacterial infection

33%

Fall

33%

Shunt malfunction

33%

Blood alkaline phosphatase increased

33%

Blood lactate dehydrogenase increased

33%

Lipase decreased

33%

Costochondritis

33%

Musculoskeletal pain

33%

Osteonecrosis

33%

Dizziness

33%

Dysuria

33%

Dyspnoea exertional

33%

Acne

33%

Pneumonia

100%

80%

60%

40%

20%

Study treatment Arm

Enasidenib 100 mg

Enasidenib 200 mg

Enasidenib 400 mg

Enasidenib 650 mg

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