Study on Azacitidine and Enasidenib for Treating Patients with IDH2-Mutant Myelodysplastic Syndrome

Sponsor: M.D. Anderson Cancer Center

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT03383575

Provider View

This clinical trial is testing the safety and effectiveness of two drugs, azacitidine and enasidenib, in treating patients with a specific type of blood disorder called IDH2-mutant myelodysplastic syndrome (MDS). These drugs may help stop cancer cells from growing by blocking certain enzymes.

Patient Parameters

Program Overview

The purpose of this study is to see how well the drugs azacitidine and enasidenib work together to treat patients with myelodysplastic syndrome (MDS) that has a specific genetic mutation called IDH2. The study will also look at how safe these drugs are when used alone or in combination. Azacitidine and enasidenib may help stop the growth of cancer cells by blocking certain enzymes needed for cell growth.

Description

This study has two main goals:

  1. To find out if enasidenib alone, or enasidenib combined with azacitidine, is safe and can be tolerated by patients with IDH2-mutant myelodysplastic syndrome (MDS).
  2. To see how effective the combination of enasidenib and azacitidine is in patients who have not been treated with hypomethylating agents (HMA) before, and how effective enasidenib alone is in patients whose MDS has not responded to HMA therapy.

The study will also look at other factors, such as how long patients live without the disease getting worse and how long the treatment response lasts. Researchers will also study changes in the cancer cells and DNA, as well as the patients' quality of life.

Patients will be divided into two groups:

  • Group 1: Patients who have not received HMA treatment before will take enasidenib by mouth daily and receive azacitidine through an IV or injection for 7 days. This cycle repeats every 28 days unless the disease progresses or side effects become unacceptable.
  • Group 2: Patients whose MDS has not responded to HMA therapy will take enasidenib by mouth daily. This cycle also repeats every 28 days unless the disease progresses or side effects become unacceptable.

After finishing the study treatment, patients will have follow-up visits every 3 months for up to 3 years.

Eligibility Criteria

Inclusion Criteria

  • Must provide signed, informed consent before any study procedures.
  • Must have a confirmed diagnosis of myelodysplastic syndrome (MDS) or related conditions.
  • Must have an IDH2 gene mutation as confirmed by a local lab.
  • For Group 1: Must not have received hypomethylating agent therapy before.
  • For Group 1: Must have high-risk MDS or certain genetic features if intermediate risk.
  • For Group 2: Must have MDS that did not respond to previous HMA therapy.
  • Must have a performance status score of 0 to 2, indicating good overall health.
  • Must have certain blood test results within normal limits.
  • Must be able to understand and sign informed consent.
  • Must have resolved any significant side effects from previous cancer treatments.
  • Women of childbearing potential must have a negative pregnancy test and agree to use contraception. Men must also agree to use contraception.

Exclusion Criteria

  • Any medical condition that increases the risk of participating in the study.
  • Previous treatment with an IDH2 inhibitor.
  • Mental health conditions that prevent understanding the study.
  • Active, uncontrolled infections, including HIV or hepatitis B/C.
  • Gastrointestinal conditions that affect drug absorption.
  • Active central nervous system disease.
  • Significant heart problems or recent heart attack.
  • Abnormal heart rhythm or prolonged QT interval.
  • Pregnant or nursing women.
  • Known allergies to study drugs.

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