RARE 1 Nilotinib and Paclitaxel for Rare Cancers
This study is testing a combination of two drugs, nilotinib and paclitaxel, for people with rare solid tumors. Researchers want to see if these drugs, which have shown promise in lab studies and in other cancers, can shrink or stop the growth of rare cancers. You may be able to join if you are 18 or older, have a rare cancer that has gotten worse after standard treatments, or if there are no other effective treatments available. The study will measure how many participants' tumors respond to the treatment over 12 months. The current status of this study is unclear, and it plans to enroll 82 participants.
- Study design
- This is an interventional study, meaning participants will receive a specific treatment. It aims to enroll 82 participants.
- What's involved
- You will be screened with a medical history, physical exam, blood and urine tests, and a pregnancy test if needed.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary outcome, objective response, is measured at 12 months.
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NCT04449549
Rapid Analysis and Response Evaluation of Combination Anti-Neoplastic Agents in Rare Tumors (RARE CANCER) Trial: RARE 1 Nilotinib and Paclitaxel
Recruiting
PHASE2Ages 18+InterventionalTreatmentNational Cancer Institute (NCI)Government-funded
~82 participants
Updated 2026-08-28 on ClinicalTrials.gov
What's tested:Nilotinib and Paclitaxel
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Objective response
Measured over 12 months
Conditions
Where it's being run
1 sites across 1 statesMaryland1
Study leadership
- Alice P Chen, M.D. · PRINCIPAL_INVESTIGATOR · National Cancer Institute (NCI)
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Patients must have histologically confirmed rare solid tumors that have progressed on standard therapy known to prolong survival or for which no standard treatment options exist. With Amendment G (v 6-17-24), eligibility will be limited to patients with adult granulosa cell ovarian cancer, clear cell ovarian cancer, anal cancer, or Ewing sarcoma. Patients must have measurable and evaluable disease.
Age \>= 18 years.
ECOG performance status \<= 2.
Patients must have normal organ and marrow function as defined below:
Absolute neutrophil count \>=1,500/mcL
Platelets \>=100,000/mcL
Total bilirubin \<=1.5 X institutional ULN
AST(SGOT)/ALT(SGPT) \<=3 X institutional upper limit of normal; \<= 5.0 x ULN in patients with liver metastases
creatinine \<=1.5 X institutional ULN OR
creatinine clearance \>=60 mL/min/1.73 m\^2 for patients with creatinine levels \>1.5 mg/dL
Nilotinib and paclitaxel have both been assigned to pregnancy category D by the FDA. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation and for at least 3 months after dosing with study drugs ceases. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 3 months after completion of study drug administration.
Patients must have completed radiation therapy or major surgery \>= 3 weeks, or biologic therapy or chemotherapy \>= 5 half-lives or 3 weeks, whichever is shorter (6 weeks for nitrosoureas and mitomycin C) prior to entering the study. Patients must be \>= 2 weeks since any prior administration of a study drug in a Phase 0 or equivalent study and be \>= 1 week from palliative radiation therapy (patients on study may be eligible for palliative radiotherapy to non-targeted lesions after 2 cycles of therapy at the PI's discretion). Patients must have recovered to eligibility levels from prior toxicity or adverse events. Treatment with bisphosphonates is permitted.
Biopsies are optional on this study. In lieu of baseline biopsies, patients are encouraged to submit at registration archival tumor biopsy tissue from a previous research study or medical care providing it meets the minimum collection and preservations requirements outlined for the submission of archival tissue are:
Tissue must have been collected within 3 months prior to registration.
Patient must not have received any intervening therapy for their cancer since the collection of the tumor sample.
Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial. For these patients, an HIV viral load test must be completed within 28 days prior to enrollment.
Exclusion
QTcF interval of \>=450 msec at study entry; congenital long QT syndrome
Sensory/motor neuropathy \>= Grade 2
Patients who are receiving any other investigational agents.
Patients with known primary central nervous system (CNS) malignancy or symptomatic CNS metastases are excluded, with the following exceptions:
Patients with asymptomatic untreated CNS disease may be enrolled, provided all of the following criteria are met:
Evaluable or measurable disease outside the CNS
No metastases to brain stem, midbrain, pons, medulla, or cerebellum
No history of intracranial hemorrhage or spinal cord hemorrhage
No ongoing requirement for dexamethasone for CNS disease; patients on a stable dose of anticonvulsants are permitted.
No neurosurgical resection or brain biopsy within 28 days prior to Cycle 1, Day 1
Patients with asymptomatic treated CNS metastases may be enrolled, provided all the criteria listed above are met as well as the following:
Radiographic demonstration of improvement upon the completion of CNS-directed therapy and no evidence of interim progression between the completion of CNS-directed therapy and radiographic screening for the current study
No stereotactic radiation or whole-brain radiation within 28 days prior to Cycle 1, Day 1
Screening CNS radiographic study \>=4 weeks from completion of radiotherapy and \>=2 weeks from discontinuation of corticosteroids
History of allergic reactions attributed to compounds of similar chemical or biologic composition to study drugs.
Uncontrolled intercurrent illness including, but not limited to, serious untreated infection, symptomatic respiratory failure/congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
Pregnant women are excluded from this study because nilotinib and paclitaxel have been assigned to pregnancy category D by the FDA. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with the study drugs, breastfeeding should be discontinued prior to the first dose of study drug and women should refrain from nursing throughout the treatment period and for 3 months following the last dose of study drug.
What this trial measures
- Objective response12 months
If at least 4/30 patients experience an objective response, defined as a complete or partial response by RECIST 1.1, the combination of nilotinib and paclitaxel will be considered promising. This design provides approximately 88% power to reject a null ORR of 0.05 when the true ORR is 0.2 (with one-sided type-I error of approximately 0.062).