Recruiting
Phase 2

Entrectinib

Sponsor:

St. Jude Children's Research Hospital

Code:

NCT06528691

Conditions

High Grade Glioma

CNS Tumor

Eligibility Criteria

Sex: All

Age: 0 - 3

Healthy Volunteers: Not accepted

Interventions

Entrectinib

Cyclophosphamide

Etoposide

Carboplatin

G-CSF

Study Details

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.

Conditions

High Grade Glioma

CNS Tumor

Study ID

NCT06528691

Start date

May 1, 2026

Status verified date

May, 2026

Completion date

Nov, 2032

Anticipated

Primary completion date

Nov, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 3

Healthy Volunteers: Not accepted

Inclusion Criteria: 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 Criteria: 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

Exclusion Criteria: COHORT 1 AND 2

  • 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)
  • Receiving enzyme inducing antiepileptic drugs (EIAEDs)
  • Any prior cancer therapy including chemotherapy (excluding Bridging Chemotherapy Cycle), targeted therapy, immunotherapy, cellular therapy, or radiation
  • Receiving another investigational agent concurrently
  • Surgery within 2 weeks prior to treatment enrollment
  • 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
  • Inability to take medication enterally

Study Design

Enrollment

52 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Entrectinib therapy, Cohort 1 and Cohort 2

Cohort 1: Patients who are younger than 3 years of age diagnosed with NTRK1/2/3- or ROS1-fused high-grade glioma (HGG) will receive therapy as outline in Detailed Description.

Cohort 2: Patients who are younger than 3 years of age diagnosed with NTRK1/2/3- or ROS1-fused CNS tumors other than HGG will receive therapy as outline in Detailed Description.

Interventions

Entrectinib

Given orally (PO) or enterally

Cyclophosphamide

Given intravenous (IV)

Etoposide

Given IV

Carboplatin

Given IV

G-CSF

Given subcutaneous (SQ) or IV

Pegfilgrastim

Given SQ as part of recommended Bridging Therapy instead of G-CSF.

Surgery

A gross total resection or significant debulking may become possible if a response to entrectinib is seen.

Primary outcome measure

  • Overall response rate (ORR) (Cohort 1) [ Time Frame: After cycle 4 (each cycle is 28 days). ]

Central Contacts and Locations

Central contacts

Locations

St. Jude Children's Research Hospital

Recruiting

Memphis, Tennessee, United States, 38105

Contacts

Principal Investigator:

Daniel Moreira, MD, MEd

More Information

Sponsor

St. Jude Children's Research Hospital

Last update posted

May 13, 2026

Last verified

May, 2026

Keywords

  • CNS Tumors
  • High Grade Glioma with NTRK1/2/3 gene fusion
  • High Grade Glioma with ROS1 gene fusion
  • CNS tumor other than HGG harboring NTRK1/2/3 gene fusion
  • CNS tumor other than HGG harboring ROS1 gene fusion
  • Children

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by St. Jude Children's Research Hospital on 2026-05-13.