Status:

RECRUITING

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

Lead Sponsor:

St. Jude Children's Research Hospital

Collaborating Sponsors:

Hoffmann-La Roche

Conditions:

High Grade Glioma

CNS Tumor

Eligibility:

All Genders

Up to 3 years

Phase:

PHASE2

Brief Summary

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.

Detailed Description

PRIMARY OBJECTIVE * To determine the overall response rate of entrectinib when used as first line therapy in patients who are younger than 3 years of age with NTRK1/2/3- or ROS1-fused high-grade glio...

Eligibility Criteria

Inclusion

  • Screening Phase
  • 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

Exclusion

  • Screening Phase
  • Previous exposure to cytotoxic chemotherapy or radiotherapy
  • Inclusion Criteria: COHORT 1
  • 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
  • Inclusion Criteria: COHORT 2
  • 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

Key Trial Info

Start Date :

January 1 2026

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

November 1 2032

Estimated Enrollment :

52 Patients enrolled

Trial Details

Trial ID

NCT06528691

Start Date

January 1 2026

End Date

November 1 2032

Last Update

November 28 2025

Active Locations (1)

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1

St. Jude Children's Research Hospital

Memphis, Tennessee, United States, 38105