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 ...
This is not medical advice. AI-assisted translation — inaccuracies may occur. Always verify the original and consult your oncologist before taking any steps.
About the trial
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.
Original English text from ClinicalTrials.gov
Who can (and can't) join
✓ Qualifies
- •Wiek od 3 do 35 lat.
- •Zdiagnozowana białaczka (AML lub ALL) lub guz lity, który nawrócił lub nie zareagował na dotychczasowe leczenie.
- •Guz lity, w tym związane z nerwiakowłókniakowatością typu 1 (NF1) i złośliwymi nowotworami osłonek nerwów obwodowych (MPNST).
- •Choroba musi być możliwa do oceny (w przypadku guzów) lub mierzalna/ocenialna (w przypadku białaczki).
- •Zgoda na udział w badaniu od pacjenta lub jego opiekuna prawnego.
- •Zdolność do połykania kapsułek.
- •Ogólny stan zdrowia pozwalający na udział w badaniu (oceniany za pomocą skali Karnofsky'ego lub Lansky'ego).
✗ Disqualifies
- •Choroby inne niż te objęte badaniem.
- •Przyjmowanie pewnych leków, które mogą wpływać na wyniki badania.
- •Ciężkie choroby serca, wątroby lub nerek.
- •Ciąża lub karmienie piersią.
- •Niezdolność do poddania się badaniom wymaganym w protokole badania.
Simplified criteria — AI translation
Trial details
- Minimum age
- 3 Years
- Maximum age
- 35 Years
- Last updated (source)
- July 21, 2026
- Sex
- No restrictions
Therapies / drugs in trial
Locations (1)
National Institutes of Health Clinical Center
Bethesda, United States
Trial contact
NCI POB Solid Tumor Referral Team
Rosandra N Kaplan, M.D.
Contact information from ClinicalTrials.gov. Contact in English.
Share this trial
Data from ClinicalTrials.gov. AI-assisted translation, last sync: 7/3/2026.
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