The following article features coverage from the American Society of Clinical Oncology 2019 meeting. Click here to read more of Cancer Therapy Advisor‘s conference coverage.

In a phase 1/1B trial, entrectinib showed antitumor activity and appeared safe in pediatric patients with recurrent or refractory solid tumors harboring NTRK1/2/3, ROS1, or ALK aberrations, including those in the central nervous system (CNS) ( identifier: NCT02650401). The trial results will be presented at the upcoming 2019 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago.

A total of 29 children and adolescents with solid tumors were enrolled in the trial and received entrectinib, an oral inhibitor of TRKA/B/C, ROS1, and ALK proteins that can penetrate the CNS. The trial had 2 phases: a dose-finding phase that established a recommended dose of 550 mg/m2 daily, and a basket trial that is currently ongoing.

Patients had a median age of 7 years (range, 0–20 years), 15 patients were male, and 14 were female. Tumor types included neuroblastoma (16 patients), high-grade glioma (5 patients), inflammatory myofibroblastic tumor (3 patients), infantile fibrosarcoma (2 patients), synovial sarcoma (1 patient), CNS embryonal tumor (1 patient), and melanoma (1 patient).

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In all, 28 patients were evaluable for a response. All patients (11 of 11) harboring a NTRK1/2/3, ROS1, or ALK gene fusion had responses (3 complete responses and 8 partial responses). One patient with an ALK-mutated neuroblastoma responded to treatment with entrectinib. No responses were seen in patients with tumors lacking aberrations in target kinases.

“This is early results and many of our patients continue to derive a benefit from this drug,” said study presenter Giles W. Robinson, MD, a pediatric neuro-oncologist at St. Jude Children’s Research Hospital in Memphis, Tennessee, during an ASCO press conference.

“Overall [entrectinib] was quite well tolerated,” Dr Robinson said. Toxicities included elevated creatinine, dysgeusia, fatigue, and pulmonary edema. Weight gain was also observed as a side effect of treatment.

The trial is ongoing.

Read more of Cancer Therapy Advisor‘s coverage of ASCO’s annual meeting by visiting the conference page.


Robinson GW, Gajjar AJ, Gauvain KM, et al. Phase 1/1B trial to assess the activity of entrectinib in children and adolescents with recurrent or refractory solid tumors including central nervous system (CNS) tumors. Presented at: 2019 American Society of Clinical Oncology Annual Meeting; Chicago, IL; May 31-June 4, 2019. Abstract 10009.