Actively 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 ...
Led by National Cancer Institute (NCI) · Updated on 2026-04-24
54
Participants Needed
1
Research Sites
713 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
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 ...
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 3 and 35 years with body surface area of at least 0.55 m^2
- Diagnosis of recurrent or refractory solid tumors including central nervous system tumors, NF1-associated malignant peripheral nerve sheath tumor, or refractory acute leukemia (AML or ALL)
- Ability to swallow capsules
- Karnofsky performance status of 50% or higher for patients older than 16 years; Lansky performance status of 50% or higher for patients 16 years or younger
- Fully recovered from acute toxic effects of prior anti-cancer therapy
- Adequate hematologic, liver, and kidney function as defined by specific blood counts and lab values
- Women of childbearing potential and fertile men must agree to use effective contraception during treatment and for 1 month after last dose
- Ability or legal guardian's willingness to provide informed consent
You will not qualify if you...
- Pregnant or breastfeeding individuals
- Current treatment with other cancer therapies or investigational agents except intrathecal chemotherapy for leukemia when indicated
- Need for warfarin therapy
- Uncontrolled illness including active infection, symptomatic heart failure, unstable angina, arrhythmia, or psychiatric/social issues limiting compliance
- Active untreated infection
- Known active hepatitis A, B, C, or HIV infection
- History of allergic reactions to TURALIO(R) or similar compounds
- Prolonged clotting times (PT/INR) unless cleared by hematology
- Recent use of drugs affecting CYP3A4 enzyme or acid reducing agents including proton pump inhibitors within 14 days before study start
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
N
NCI POB Solid Tumor Referral Team
CONTACT
R
Rosandra N Kaplan, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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