Early drug study for children and young adults with hard-to-treat cancers
Part of Blood & lymphatic, Brain & nervous system, Cancer, Immune system & allergy clinical trials.
This Phase 1 trial tests pexidartinib (TURALIO) to see how safe it is and how well it works against refractory (hard-to-treat) leukemias and solid tumors, including tumors seen with NF1 and MPNST. It mainly helps researchers learn the best way to dose the medication and gather early signals of benefit.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are age 3 to 35 (and your body size must meet a minimum body surface area).
- You have a confirmed hard-to-treat cancer: recurrent or refractory solid tumors or AML/ALL leukemia, or NF1 with malignant peripheral nerve sheath tumor (MPNST) and related nerve tumors.
- Your cancer must be measurable or otherwise evaluable at enrollment.
- You must be able to swallow capsules and travel to NIH for study visits.
- You must have recovered from past cancer treatments (timing rules apply for chemo, radiation, immune treatments, transplants, and surgery).
- Your blood, liver, kidney, and heart tests must meet safety limits (and you must not be pregnant or breastfeeding).
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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