Entrectinib as first treatment for infants with certain brain tumors

Trial details

Who runs it

St. Jude Children's Research Hospital

Trial code

NCT06528691

Record updated

3 November 2026

Original description

This clinical trial tests how well entrectinib works to treat patients less than 3 years of age with NTRK 1/2/3 or ROS1 fused, high grade glioma or other central nervous system (CNS) tumors.Read full original descriptionShow less

PHASE 2 Study of Entrectinib as a Single Agent in Upfront Therapy for Children <3 Years of Age With NTRK1/2/3 or ROS1-FUSED CNS Tumors (GLOBOTRK)

This clinical trial tests how well entrectinib works to treat patients less than 3 years of age with NTRK 1/2/3 or ROS1 fused, high grade glioma or other central nervous system (CNS) tumors.

Inclusion: Age from birth to age <3 years at the time of diagnosis (date of surgical resection/biopsy); Participant with presumed newly diagnosed tumor in the supratentorial compartment; Patient must have measurable disease based on RAPNO criteria; ≤84 days since surgery (resection or biopsy); Available tumor tissue for central review; Parent/guardian has the ability to understand and the willingness to sign a written informed consent document according to institutional guidelines; Patients must be <3 years of age at the time of diagnosis (date of surgical resection/biopsy); High-grade glioma (World Health Organization [WHO] grade III or IV) harboring NTRK1/2/3 or ROS1 gene fusions as determined by central pathology review; Patients must have measurable disease as defined by RAPNO criteria; Patients are eligible at the time of diagnosis, prior to any exposure to chemotherapy, targeted therapy, immunotherapy, cellular therapy or radiation; ≤28 days since study screening; Lansky score ≥50% and a minimum life expectancy of ≥ 12 weeks; Neurologic deficits must have been stable for at least 7 days prior to study enrollment; Hemoglobin ≥ 8 g/dL (without transfusion or erythropoietin use within 7 days prior to enrollment); Platelet count ≥ 75,000/µL (without transfusion within 7-day period prior to enrollment); Absolute neutrophil count >1,000/µL; Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5x the upper limit of normal (ULN); Bilirubin ≤ 1.5 x ULN; Adequate renal function as defined by the following age-based serum creatinine concentrations:; 0 to <1 year: 0.5 mg/dL; 1 to <2 years: 0.6 mg/dL; 2 to 3 years: 0.8 mg/dL; Adequate cardiac function as defined by electrocardiogram (ECG) with Fridericia's corrected QT interval (QTc) ≤ 450 msec and echocardiogram left ventricular ejection fraction (LVEF) >50%; Screening and enrollment consents signed; Willingness and ability to comply with treatment plan, scheduled visits, laboratory tests and other study procedures; Patients must be <3 years of age at the time of diagnosis (date of surgical resection/biopsy); CNS tumor other than HGG harboring NTRK1/2/3 or ROS1 gene fusions as determined by central pathology review; Patients must have measurable disease as defined by RAPNO criteria; Patients are eligible at the time of diagnosis, prior to any exposure to chemotherapy, targeted therapy, immunotherapy, cellular therapy or radiation; ≤28 days since study screening; Lansky score ≥50% and a minimum life expectancy of ≥ 12 weeks; Neurologic deficits must have been stable for at least 7 days prior to study enrollment; Hemoglobin ≥ 8 g/dL (without transfusion or erythropoietin use within 7 days prior to enrollment); Platelet count ≥ 75,000/µL (without transfusion within 7-day period prior to enrollment); Absolute neutrophil count >1,000/µL; ALT and ALT ≤2.5x the upper limit of normal (ULN); Bilirubin ≤ 1.5 x ULN; Adequate renal function as defined by the following age-based serum creatinine concentrations:; 0 to <1 year: 0.5 mg/dL; 1 to <2 years: 0.6 mg/dL; 2 to 3 years: 0.8 mg/dL; Adequate cardiac function as defined by ECG with QTc ≤ 450 msec and echocardiogram LVEF >50%; Screening and enrollment consents signed; Willingness and ability to comply with treatment plan, scheduled visits, laboratory tests and other study procedures.

Exclusion: Previous exposure to cytotoxic chemotherapy or radiotherapy; Receiving enzyme inducing antiepileptic drugs (EIAEDs); Receiving another investigational agent concurrently; Surgery within 2 weeks prior to treatment enrollment; Inability to take medication enterally; Clinically significant medical disorder that could compromise the ability to tolerate study therapy or would interfere with the study procedures or results history; History of recent (3 months) symptomatic congestive heart failure; Known active, uncontrolled infection (bacterial, fungal, or viral); Any prior cancer therapy including chemotherapy (excluding Bridging Chemotherapy Cycle), targeted therapy, immunotherapy, cellular therapy, or radiation; Patients with known hypersensitivity to excipients of the investigational medicinal product; Active gastrointestinal disease or malabsorption disorder (e.g. Crohn's disease, ulcerative colitis, short-gut syndrome) that would impair drug absorption.

ClinicalTrials.gov NCT06528691