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

• Status: Recruiting
• Trial end date: 2021-12-01

Summary

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.

• Phase: Phase 1/Phase 2

Details

• Lead Sponsor: National Cancer Institute (NCI)