Phase 1 study evaluating combination therapy with the receptor tyrosine kinase inhibitor Pexidartinib (PLX3397) and Sirolimus in patients with unresectable sarcoma and Phase 2 study in malignant peripheral nerve sheath tumors


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The purpose of this study is to determine if treatment with PLX3397 and Sirolimus will be tolerated and result in shrinking of the cancer or stopping the cancer from growing. In the phase I portion, the maximum tolerate dose of the study drug will be determined. In the Phase II potion, progression free survival will be assessed at the dose level found in Phase I. Participants will continue to take the study drug until they experiance an unacceptable side effect or their disease progresses.

Are you Eligible? (Inclusion Criteria)

  • Individuals over the age of 18 with Malignant peripheral nerve sheath tumors
  • (MPNSTs) that can not be removed surgically or that have a sarcoma that has
  • been treated with chemotherapy and is growing.

Specialty Area(s)

Sarcomas (Bone and Soft Tissue), Soft Tissue Sarcoma

Principal Investigator

Profile Headshot
  • Director of Gastrointestinal Oncology
  • Director of Pancreas Translational Research

Trial Location(s)

CUIMC/Herbert Irving Pavilion
161 Fort Washington Avenue
New York, NY 10032