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Study identifier: NCT02390752 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...

Condition: Neurofibroma, Plexiform · Precursor Cell Lymphoblastic Leukemia-Lymphoma · Leukemia, Promyelocytic, Acute  ·  Sponsor: National Cancer Institute (NCI)

PhasePhase 1
Planned participants54
Who can joinAll sexes, 3 Years to 35 Years
Healthy volunteersNo

About this study

Background: \- Some people with cancer have solid tumors. Others have refractory leukemia. This may not go away after treatment. Researchers want to see if a drug called TURALIO(R) can shrink tumors or stop them from growing. Objectives: \- To find the highest safe dose and side effects of TURALIO(R). To see if it helps treat certain types of cancer. Eligibility: \- People ages 3-35 with a solid tumor or leukemia that has returned or not responded to cancer therapies. Design: * Individuals will be screened with: * Medical history * Physical exam * Blood and urine tests * Heart tests * Scans or other tests of the tumor * Individuals will take TURALIO(R) 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. Individuals will keep a diary of symptoms. * Individuals with solid tumors will have scans or x-rays. * Individuals with leukemia will have blood tests. They may have a bone marrow sample taken. * Some individuals may have a biopsy. * When finished taking TURALIO(R), individuals will have follow-up visits. They will repeat the screening tests and note side effects.

This description comes directly from the study's public registry record.

Talk to the study team

NCI POB Solid Tumor Referral Team  ·  (240) 858-7013  ·  ncipobstreferrals@mail.nih.gov

Rosandra N Kaplan, M.D.  ·  (240) 760-6198  ·  kaplanrn@mail.nih.gov

Always discuss trial participation with your own doctor first.

Locations (1)

National Institutes of Health Clinical CenterBethesda, Maryland, United StatesRecruiting

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Source record: clinicaltrials.gov/study/NCT02390752