Actively Recruiting
Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors
Led by National Cancer Institute (NCI) · Updated on 2026-04-28
40
Participants Needed
1
Research Sites
356 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Cancers of the nasal cavity or skull base are rare. They often are not diagnosed until they are at an advanced stage, and they often spread to other parts of the body. These cancers may have mutations in a gene called IDH2. Researchers want to find out if a drug (enasidenib) that targets the IDH2 mutation can help people with these cancers. Objective: To test enasidenib in people with cancers of the nasal cavity or skull base. Eligibility: People aged 18 years and older with rare cancers of the nasal cavity or the base of the skull. Their cancer must have an IDH2 gene mutation, and it must have recurred locally or spread to other parts of the body. These cancers can include sinonasal undifferentiated carcinoma; olfactory neuroblastoma; sinonasal large-cell neuroendocrine carcinoma; poorly differentiated sinonasal adenocarcinoma; or chondrosarcoma. Design: Participants will be screened. They will have a physical exam with blood and urine tests and tests of their heart function. They will have imaging scans of their brain, skull base, neck, chest, abdomen, and pelvis. A sample of tumor tissue will be collected. Enasidenib is a tablet taken by mouth with a glass of water. Participants will take the drug once a day, every day, in 28-day cycles. They will not have resting periods between cycles. Participants will visit the clinic on the first day of each cycle to receive the tablets they will need to take at home until the beginning of the next cycle. They will keep a diary to record the time of each dose they take. Participants may remain in the study as long as the drug is helping them....
CONDITIONS
Official Title
Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Histologically or cytologically confirmed locally advanced or metastatic sinonasal undifferentiated carcinoma, olfactory neuroblastoma, large-cell neuroendocrine carcinoma, poorly differentiated sinonasal adenocarcinoma, or chondrosarcoma with documented tumor IDH2 mutations R140 or R172
- Primary tumors located in the sinonasal cavity and/or skull base
- Locally advanced disease not suitable for curative surgery or radiotherapy
- Disease recurrence or progression after prior systemic therapy for recurrent or metastatic cancer
- Measurable disease according to RECIST 1.1 criteria
- Age 18 years or older
- ECOG performance status of 0 to 2
- Adequate organ and marrow function including hemoglobin ≥9 g/dL, ANC ≥1,000/mcL, platelets ≥75,000/mcL, bilirubin ≤1.5 times upper limit of normal (ULN), AST/ALT ≤1.5 times ULN (or ≤2.5 times ULN if liver metastases), and creatinine ≤1.5 times ULN or creatinine clearance ≥40 mL/min
- Treated brain or central nervous system metastases with no progression after at least 4 weeks
- For participants with HIV, CD4 count ≥200 cells/mm3, stable antiretroviral therapy for at least 4 weeks, and no recent opportunistic infections
- For participants with hepatitis B or C, viral load undetectable on suppressive therapy if applicable
- Women of reproductive potential and men with partners of reproductive potential must agree to use effective contraception during and for 2 months after treatment
- Agreement to discontinue or postpone breastfeeding during and for 2 months after treatment
- Ability and willingness to provide informed consent and undergo blood sampling and tumor biopsy for research
- Co-enrollment in companion biospecimen procurement study (and olfactory neuroblastoma natural history study if applicable)
You will not qualify if you...
- Prior treatment with any IDH1/2 inhibitor
- Use of other investigational agents within 3 weeks or 5 half-lives before starting study treatment
- Systemic anticancer treatment within 3 weeks before starting study treatment, except bisphosphonates and denosumab
- Large-field radiotherapy within 4 weeks before starting treatment
- Major surgery within 2 weeks before starting treatment, with insufficient recovery
- New or active brain metastases or leptomeningeal disease
- Allergic reactions to compounds similar to enasidenib
- Treatment with botanical or herbal supplements intended for general health or study disease within 2 weeks before treatment
- Use of certain sensitive cytochrome P450 substrate medications with narrow therapeutic range unless switched prior to enrollment
- Use of sensitive substrates of specific drug transporters unless dose adjustments or switches are made prior to enrollment
- Use of medications known to prolong QT interval unless alternatives are used or monitored
- Significant cardiovascular disease or impaired function including recent stroke, uncontrolled hypertension, recent acute coronary syndrome, symptomatic heart failure, severe arrhythmia, or QTcF >450 msec
- Conditions limiting oral drug absorption such as short-gut syndrome or gastroparesis
- Active infections requiring intravenous antibiotics
- History of second malignancy within 3 years except certain treated localized cancers
- Pregnancy
- Uncontrolled illnesses or social situations limiting study participation or safety
AI-Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
N
NCI Medical Oncology Referral Office
CONTACT
C
Charalampos Floudas, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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