RecruitingPhase 1NCT02390752

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 ...

Phase I Trial of TURALIO (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 (TGCT)


Sponsor

National Cancer Institute (NCI)

Enrollment

54 participants

Start Date

Apr 29, 2015

Study Type

INTERVENTIONAL

Conditions

Summary

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.


Eligibility

Min Age: 3 YearsMax Age: 35 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a drug called pexidartinib (TURALIO) in children and young adults whose solid tumors or leukemia have come back or stopped responding to standard treatments. **You may be eligible if...** - You are between 3 and 35 years old - You have a solid tumor (such as bone cancer, muscle cancer, or brain tumor) or a blood cancer (AML or ALL) that has returned or stopped responding to treatment - You are able to swallow capsules - Your blood counts, liver, and kidney function are within acceptable ranges - Enough time has passed since your last treatment (chemotherapy, radiation, or surgery) **You may NOT be eligible if...** - You are pregnant or breastfeeding - You are currently being treated with another cancer therapy (with limited exceptions) - You need to take the blood thinner warfarin - You have active infections, uncontrolled heart failure, or unstable heart problems - You have active hepatitis A, B, C, or HIV - You take certain medications that strongly interact with the study drug Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGTURALIO(R)

take oral drug daily for a 28 day cycle


Locations(1)

National Institutes of Health Clinical Center

Bethesda, Maryland, United States

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NCT02390752


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