Azacitidine and Enasidenib in Treating Patients With IDH2-Mutant Myelodysplastic Syndrome
Targeted Therapy With the IDH2-Inhibitor Enasidenib (AG221) for High-Risk IDH2-Mutant Myelodysplastic Syndrome
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 12 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Time periods mentioned: 3 years
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "After completion of study treatment, patients are followed up every 3 months for up to 3 years."
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This phase 2 study tests the side effects of azacitidine and enasidenib and how well they work. It is for people with myelodysplastic syndrome (MDS), a bone marrow disease, or certain related blood cancers that have an IDH2 gene change. These drugs may block enzymes that cancer cells need to grow.
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.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Have MDS or an allowed related blood cancer, confirmed by a tissue test. These include certain forms of acute myeloid leukemia (AML) and chronic myelomonocytic leukemia (CMML).
- Have an IDH2-R140 or IDH2-R172 gene change confirmed by a lab test.
- Group A: Have higher-risk MDS, or intermediate-risk MDS with certain high-risk gene changes. You must not have taken azacitidine, decitabine, or SGI-110. Other MDS treatments are allowed.
- Group B: Your disease did not respond after 6 cycles of a hypomethylating drug, a type of MDS treatment, or came back after responding.
- Be able to care for yourself and be out of bed for at least half of your waking hours.
- Meet the study's liver and kidney blood test limits. Important side effects from past cancer treatment, other than blood problems or hair loss, must be mild or gone.
- Understand and sign the consent form before study procedures, and be willing and able to follow study rules.
- If you can become pregnant, have a negative pregnancy test within 7 days before treatment and use two birth control methods during treatment and for at least 3 months after. Men having sex with someone who can become pregnant must use barrier birth control for the same period.
Who can't join
- Have a medical condition that the study doctor thinks would greatly increase the risks of joining.
- Have taken a drug that targets IDH2 before, or have a known allergy to either study drug or its ingredients.
- Have mental health or thinking problems that prevent understanding consent or completing study steps.
- Have an active, uncontrolled infection, including known HIV or chronic active hepatitis B or C.
- Have serious stomach or bowel problems that could affect how the drug enters your body, including past surgery to remove the stomach.
- Have known active disease in the brain, spinal cord, or the tissues covering them.
- Have serious heart disease, poor heart function, uncontrolled heart rhythm problems, or a heart attack within the past 6 months. Certain abnormal heart tracing results also exclude you.
- Be pregnant or breastfeeding.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: * Signed, informed consent must be obtained prior to any study specific procedures * 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 * Subjects must have an IDH2 gene mutation (IDH2-R140 or R172) as determined by local laboratory result * (Arm A only): Subject must be hypomethylating agent naive (i.e. prior azacitidine, decitabine, SGI-110 is exclusionary). Receipt of other MDS-directed therapy such as lenalidomide is allowed * (Arm A only): Subjects with high-risk MDS (i.e. International Prostate Symptom Score \[IPSS\] intermediate-2 or high-risk; or revised \[R\]-IPSS high or very-high risk). Patients with intermediate-1 risk by IPSS or intermediate risk by R-IPSS with high-risk molecular features including TP53, ASXL1, EZH2, and/or RUNX1 mutations are also eligible * (Arm B only): Subject must be relapsed or refractory to prior hypomethylating agent therapy, defined as prior receipt of 6 cycles of HMA therapy with failure to attain a response, or relapse after prior response to HMA therapy * Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 * Serum bilirubin =\< 2 x the upper limit of normal (ULN) (except for patients with Gilbert's disease) * Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) =\< 3 x the laboratory ULN * Serum creatinine =\< 2 x the ULN * Able to understand and voluntarily sign a written informed consent, and willing and able to comply with protocol requirements * Resolution of all clinically significant treatment-related, non-hematological toxicities, except alopecia, from any previous cancer therapy to =\< grade 1 prior to the first dose of study treatment * Female patients of childbearing potential must have a negative serum or urine pregnancy test within 7 days of the first dose of study drug and agree to use dual methods of contraception during the study and for a minimum of 3 months following the last dose of study drug. Post-menopausal females (\> 45 years old and without menses for \> 1 year) and surgically sterilized females are exempt from these requirements. Male patients must use an effective barrier method of contraception during the study and for a minimum of 3 months following the last dose of study drug if sexually active with a female of childbearing potential Exclusion Criteria: * Any prior or coexisting medical condition that in the investigator's judgment will substantially increase the risk associated with the subject's participation in the study * Subject has received a prior targeted IDH2 inhibitor * Psychiatric disorders or altered mental status precluding understanding of the informed consent process and/or completion of the necessary study procedures * Active uncontrolled infection at study enrollment including known diagnosis of human immunodeficiency virus or chronic active hepatitis B or C infection * Clinically significant gastrointestinal conditions or disorders that may interfere with study drug absorption, including prior gastrectomy * Patients with known active central nervous system (CNS) disease, including leptomeningeal involvement * Impaired cardiac function, uncontrolled cardiac arrhythmia, or clinically significant cardiac disease including the following: a) New York Heart Association grade III or IV congestive heart failure, b) myocardial infarction within the last 6 months * Subjects with a corrected QT (QTc) \> 480 ms (QTc \> 510 msec for subjects with a bundle branch block at baseline * Nursing or pregnant women * Subjects with known hypersensitivity to study drugs or their excipients
Study sites (3)
MD
- Johns Hopkins University/Sidney Kimmel Cancer Center · Baltimore, MD 21287More trials in Baltimore
OH
- Cleveland Clinic Foundation · Cleveland, OH 44195More trials in Cleveland
TX
- M D Anderson Cancer Center · Houston, TX 77030Courtney DiNardo · Study contact · 713-794-1141Courtney DiNardo · Principal investigatorMore trials in Houston