Phase I Study of TURALIOR Pexidartinib Oral Capsules in Children and Young Adults Aged 3 to 35 With Refractory Leukemias and Solid Tumors Including NF1-Associated Plexiform Neurofibromas and Tenosynovial Giant Cell Tumors

What this Trial Is About

Researchers are investigating TURALIOR Pexidartinib, an oral drug, for children and young adults aged 3 to 35 years who have hard-to-treat cancers, including refractory solid tumors like neurofibromatosis type 1 NF1 associated plexiform neurofibromas and refractory leukemias such as acute myelogenous leukemia AML and acute lymphoblastic leukemia ALL. The study aims to find the safest dose and to evaluate side effects while exploring how the drug affects tumor growth or shrinkage. Participants will take TURALIOR capsules once daily in 28-day cycles for up to two years, with dosing adjusted based on body surface area. The trial uses a rolling-six phase I design to gradually find the best dose, involving 2 to 6 subjects per dose level, and will expand at the recommended dose to include more patients for further safety and pharmacokinetic analysis. The study includes patients with various refractory tumors and leukemias, including those with NF1 and malignant peripheral nerve sheath tumors. Throughout the study, participants will undergo multiple assessments such as physical exams, blood and urine tests, heart monitoring, and imaging scans for solid tumors or blood tests for leukemia. They will keep symptom diaries and may have biopsies if needed. After stopping the drug, follow-up visits will repeat these tests to monitor side effects and overall health. The primary outcome is determining the recommended phase II dose based on safety and tolerability during the first treatment cycle.

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 ...