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138 active trials for Sarcoma

PLX3397 in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN)

Background: - Some people with cancer have solid tumors. Others have refractory leukemia. This doesn t go away after treatment. Researchers want to see if a drug called PLX3397 can shrink tumors or stop them from growing. Objectives: - To find the highest safe dose and side effects of PLX3397. To see if it helps treat certain types of cancer. Eligibility: People ages 3 22 with a solid tumor or leukemia that has returned or not responded to cancer therapies. For Phase II, people ages 3 31 with a Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibroma (PN) that cannot be removed with surgery. Design: Participants will be screened with: Medical history Physical exam Blood and urine tests Heart tests Scans or other tests of the tumor Participants will take PLX3397 as a capsule once daily for a 28-day cycle. They can do this for up to 2 years. During the study, participants will have many tests and procedures. They include repeats of the screening tests. Participants will keep a diary of symptoms. Participants with solid tumors will have scans or x-rays. Participants with NF1 PN will have MRI scans. Participants with leukemia will have blood tests. They may have a bone marrow sample taken. Some participants may have a biopsy. When finished taking PLX3397, participants will have follow-up visits. They will repeat the screening tests and note side effects. Phase II will follow the same procedures as Phase I above, but participants will also fill out questionnaires about their pain and quality of life.

Start: April 2015