Azacitidine + Enasidenib for Myelodysplastic Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This phase II trial studies the side effects and how well azacitidine and enasidenib work in treating patients with IDH2-mutant myelodysplastic syndrome. Azacitidine and enasidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
Will I have to stop taking my current medications?
The trial protocol does not specify whether you need to stop taking your current medications. However, if you have previously taken a targeted IDH2 inhibitor, you cannot participate in this trial.
Is the combination of Azacitidine and Enasidenib safe for humans?
Azacitidine has been studied for safety in patients with myelodysplastic syndromes, acute myeloid leukemia, and chronic myelomonocytic leukemia. Common side effects include low blood cell counts and gastrointestinal issues, but these can often be managed with supportive care. The combination with other drugs like panobinostat showed more severe side effects, suggesting careful monitoring is needed.12345
How is the drug Azacitidine + Enasidenib unique for treating myelodysplastic syndrome?
The combination of Azacitidine and Enasidenib is unique because Azacitidine is a hypomethylating agent that can prolong survival in higher-risk myelodysplastic syndrome (MDS) patients, while Enasidenib targets specific genetic mutations (IDH2 mutations) that may be present in some MDS cases, potentially offering a more tailored treatment approach.25678
What data supports the effectiveness of the drug Azacitidine for treating myelodysplastic syndrome?
Who Is on the Research Team?
Courtney DiNardo, MD
Principal Investigator
M.D. Anderson Cancer Center
Are You a Good Fit for This Trial?
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).Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Patients receive enasidenib and azacitidine or enasidenib alone in 28-day cycles
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Azacitidine
- Enasidenib
Azacitidine is already approved in European Union, United States, Canada, Japan for the following indications:
- Acute myeloid leukemia
- Chronic myelomonocytic leukemia
- Myelodysplastic syndromes
- Myelodysplastic syndromes
- Chronic myelomonocytic leukemia
- Myelodysplastic syndromes
- Acute myeloid leukemia
- Myelodysplastic syndromes
- Acute myeloid leukemia
Find a Clinic Near You
Who Is Running the Clinical Trial?
M.D. Anderson Cancer Center
Lead Sponsor
National Cancer Institute (NCI)
Collaborator
Celgene
Industry Sponsor
Jay Backstrom
Celgene
Chief Medical Officer since 2016
MD
Mark Alles
Celgene
Chief Executive Officer since 2016
Bachelor's degree from Lock Haven University of Pennsylvania