Actively Recruiting
Phase I Trial of TURALIO (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 Associated Plexiform Neurofibromas and Tenosynovial Giant Cell Tumor
Led by National Cancer Institute (NCI) · Updated on 2026-06-08
54
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying TURALIO(R) (Pexidartinib, PLX3397), an oral drug, in children and young adults aged 3 to 35 years who have refractory solid tumors or leukemias that have returned or not responded to previous treatments. This phase I trial aims to find the highest safe dose of TURALIO(R), observe its side effects, and see if it helps treat certain cancers including neurofibromatosis type 1 associated plexiform neurofibromas and tenosynovial giant cell tumor. Participants will take TURALIO(R) capsules once daily in 28-day cycles, continuing for up to 2 years if tolerated. The dosing is based on body surface area and adjusted using a dosing nomogram. The study uses a rolling-six design to find the maximum tolerated dose during the first cycle, followed by expansion of the recommended dose group to gain more experience. Treatment continues without breaks between cycles. Throughout the study, participants will undergo various tests including medical history reviews, physical exams, blood and urine tests, heart assessments, scans or x-rays for solid tumors, and blood or bone marrow tests for leukemia. They will keep a symptom diary. After stopping TURALIO(R), follow-up visits will repeat these assessments and monitor side effects. Researchers will evaluate safety, drug tolerability, pharmacokinetics, and immune responses over time.
CONDITIONS
Brief 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...
- Ages 3 to 35 years with body surface area at least 0.55 m^2
- Diagnosed with recurrent or refractory solid tumors, including CNS neoplasms and NF1-associated malignant peripheral nerve sheath tumors, or acute leukemias (AML or ALL)
- Able to swallow capsules
- Karnofsky performance status at least 50% if over 16 years, Lansky score at least 50% if 16 or younger
- Fully recovered from prior anti-cancer therapy toxic effects
- Specific time intervals since last chemotherapy, biologic therapy, immunotherapy, radiation, stem cell transplant, surgery, or growth factor use
- Adequate hematologic, liver, and kidney function as defined by specified laboratory values
- Women of childbearing potential and fertile men must agree to use effective contraception during and for one month after treatment
- Ability to understand and sign informed consent
You will not qualify if you...
- Pregnant or breastfeeding individuals
- Currently receiving other cancer therapies or investigational agents, except intrathecal chemotherapy for leukemia when indicated
- Require warfarin therapy
- Uncontrolled or active infections, symptomatic heart failure, unstable angina, arrhythmias, or psychiatric/social conditions limiting compliance
- Known active hepatitis A, B, C or HIV infection, or inactive hepatitis B carrier status
- History of allergic reactions to compounds similar to TURALIO(R)
- Prolonged PT/INR unless cleared by hematology
- Use of drugs strongly affecting CYP3A4 or UGT enzymes, or proton pump inhibitors within 14 days prior to study start
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Repeated 28-day cycles until disease progression or discontinuation
Participants take the study drug TURALIO(R) orally once daily in 28-day cycles continuously without a rest period between cycles, to assess safety, tolerability, and determine the recommended phase II dose.
Visits prior to cycles 3, 5, 9, 13 and every 6 cycles for safety assessments; pharmacokinetic assessments during cycles 1 and 2; regular assessments before cycle 1, on day 7 of cycle 1, and at each restaging evaluation
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
R
Rosandra N Kaplan, M.D.
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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