OncoMatch/Clinical Trials/NCT02390752
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 ...
Is NCT02390752 recruiting? Yes, currently enrolling (May 2026). This Phase 1 trial studies TURALIO(R) for neurofibroma, plexiform.
Treatment: TURALIO(R) — 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.
Check if I qualifyExtracted eligibility criteria
Cancer type
Acute Lymphoblastic Leukemia
Non-Hodgkin Lymphoma
Acute Myeloid Leukemia
Sarcoma
Prior therapy
Must have received: effective standard therapies
Individuals must have relapsed after or be refractory to effective standard therapies. There are no limits on number of prior therapeutic regimens.
Lab requirements
Blood counts
Absolute neutrophil count >= 1.5 x 10^9/L; Hemoglobin > 10 g/dL; Platelet count >= 100 x 10^9/L
Kidney function
Serum creatinine <= 1.5 x ULN
Liver function
AST and ALT <= upper limit of normal (ULN); TBil and DBil <= ULN (Gilbert's syndrome: TBil <= 1.5 x ULN)
Cardiac function
Ejection fraction >= 50%, QTcF < 450 ms (male) or <470 ms (female) on ECG at Baseline
Absolute neutrophil count >= 1.5 x 10^9/L; Hemoglobin > 10 g/dL; Platelet count >= 100 x 10^9/L; AST and ALT <= upper limit of normal (ULN); TBil and DBil <= ULN with an exception of patients with confirmed Gilbert's syndrome. For patients with confirmed Gilberts syndrome, the TBil should be <= 1.5 x ULN; Serum creatinine <= 1.5 x ULN; Cardiac ejection fraction >= 50%, and QTcF < 450 ms (male) or <470 ms (female) on ECG at Baseline.
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- National Institutes of Health Clinical Center · Bethesda, Maryland
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