Targeted therapy with the mutant IDH2 inhibitor enasidenib for high-risk IDH2-mutant myelodysplastic syndrome.
Courtney D DiNardo, Sangeetha Venugopal, Curtis Lachowiez...
https://pubmed.ncbi.nlm.nih.gov/35973199Actively Recruiting
Phase 2
Age: 12Years +
All Genders
ID03383575
Targeted Therapy With Enasidenib and Azacitidine for High-Risk IDH2-Mutant Myelodysplastic Syndrome
Led by M.D. Anderson Cancer Center · Updated on 2026-02-17
63
Participants Needed
3
Research Sites
N/A
Total Duration
On this page
Sponsors
M
M.D. Anderson Cancer Center
Lead Sponsor
N
National Cancer Institute (NCI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating enasidenib and azacitidine in patients with myelodysplastic syndrome (MDS) that has an IDH2 gene mutation. This phase II trial aims to study the side effects, safety, and how well these drugs work alone or together. The study also looks at survival rates, molecular markers, and quality of life in patients with this specific type of blood cancer. Patients are divided into two groups based on their previous treatment. One group, who have not received hypomethylating agents (HMA), will take enasidenib orally every day and receive azacitidine either intravenously or by injection for seven days in 28-day cycles. The other group, who have relapsed or are resistant to HMA therapy, will take only enasidenib orally every day in 28-day cycles. Treatment continues unless the disease worsens or side effects become unacceptable. Participants will have regular visits for treatment and monitoring, including assessments of side effects and response to therapy. After treatment ends, they will be followed every three months for up to three years to check safety and outcomes. The primary measures include tracking adverse events and overall response rate during this time. Secondary measures involve survival and molecular activity related to the treatments.
CONDITIONS
Brief Title
Azacitidine and Enasidenib in Treating Patients With IDH2-Mutant Myelodysplastic Syndrome
Who Can Participate
Age: 12Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Signed informed consent before any study procedures
- Confirmed diagnosis of myelodysplastic syndrome (MDS), RAEB-T, or chronic myelomonocytic leukemia (CMML)
- Presence of IDH2 gene mutation (R140 or R172)
- For Arm A: No prior hypomethylating agent (HMA) treatment; high-risk MDS by IPSS or R-IPSS criteria
- For Arm B: Relapsed or refractory to prior HMA therapy after at least 6 cycles
- ECOG performance status of 0 to 2
- Serum bilirubin not more than 2 times upper limit of normal (ULN), except Gilbert's disease
- ALT and/or AST not more than 3 times ULN
- Serum creatinine not more than 2 times ULN
- Able and willing to sign consent and follow protocol
- All prior significant treatment-related non-blood toxicities resolved to grade 1 or less, except hair loss
- Female patients of childbearing potential must have negative pregnancy test and agree to dual contraception; post-menopausal or sterilized females exempt
- Male patients must use barrier contraception if sexually active with females of childbearing potential
You will not qualify if you...
- Any medical condition increasing risk for study participation
- Prior treatment with targeted IDH2 inhibitors
- Psychiatric or mental conditions preventing informed consent or study compliance
- Active uncontrolled infections including HIV or chronic hepatitis B or C
- Significant gastrointestinal conditions affecting drug absorption
- Active central nervous system disease including leptomeningeal involvement
- Severe heart conditions including NYHA class III/IV heart failure or recent heart attack within 6 months
- Corrected QT interval longer than 480 ms (or 510 ms with bundle branch block)
- Pregnant or nursing women
- Known allergy to study drugs or their ingredients
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Repeated 28-day cycles until disease progression or discontinuation
Participants receive treatment with enasidenib alone or in combination with azacitidine in repeated 28-day cycles until disease progression or unacceptable toxicity occurs.
Monthly visits for treatment cycles
Follow-up
Duration - Up to 3 years
After completing treatment, participants are followed up every 3 months for up to 3 years to monitor safety and overall outcomes.
Quarterly visits for up to 3 years
Trial Site Locations
Total: 3 locations
1
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, United States, 21287
Active, Not Recruiting
2
Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
Active, Not Recruiting
3
M D Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
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Research Team
C
Courtney DiNardo, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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