Background:
People with rare cancers often have limited treatment options. The biology of rare cancers is not well understood. Researchers want to find better treatments for these cancers. They want to test 2 drugs that, taken separately, have helped people with non-rare cancers. They want to see if these drugs together can make rare cancers shrink or stop growing.
Objective:
To learn if nilotinib and paclitaxel will benefit people with rare cancers.
Eligibility:
People age 18 and older who have a rare, advanced cancer that has progressed after receiving standard treatment, or for which no effective therapy exists.
Design:
Participants will be screened with medical history and physical exam. They will have blood and urine tests. They will have a pregnancy test if needed. They will have an electrocardiogram to check their heart. They will have imaging scans to measure their tumors.
Participants will repeat the screening tests during the study.
Participants will receive nilotinib and paclitaxel. The drugs are given in 28-day cycles. Nilotinib is a capsule taken by mouth twice a day. Paclitaxel will be given intravenously by peripheral line or central line once a week for the first 3 weeks of each cycle.
Participants will keep a medicine diary. They will track when they take the study drugs and any side effects they may have.
Participants may have optional tumor biopsies.
Participants can stay on the study until their disease gets worse or they have intolerable side effects.
Participants will have a follow-up phone call about 30 days after taking the last dose of study drugs.
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.
Absolute neutrophil count >=1,500/mcL
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.
Evaluable or measurable disease outside the CNS
Single group
Nilotinib and Paclitaxel
DrugThe BCR-Abl kinase inhibitor nilotinib demonstrated greater than additive activity in combination with the anti-tubulin agent paclitaxel in preclinical xenograft models, justifying the clinical evaluation of this combination for its antitumor activity
Objective response
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).
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