Infant-Type Hemispheric Gliomas: A Review of Clinical, Radiologic, Histopathologic, and Molecular Features.
Aditi Bagchi, Jason Chiang, Soniya Pinto...
https://pubmed.ncbi.nlm.nih.gov/41213248Actively Recruiting
Early Phase 1
Age: 0 - 21Years
All Genders
ID04655404
A Pilot and Surgical Study of Larotrectinib for Treatment of Children With Newly-Diagnosed High-Grade Glioma With NTRK Fusion
Led by Nationwide Children's Hospital · Updated on 2026-03-18
15
Participants Needed
21
Research Sites
521 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate disease status in children newly diagnosed with high-grade glioma that have a TRK fusion. It is a pilot study focusing on children up to 21 years old, assessing disease control and survival rates. The study also examines the safety of larotrectinib given with chemotherapy or after focal radiation therapy in this group. Participants will receive larotrectinib orally twice daily at a dose based on body surface area, in 28-day cycles. After 2 cycles of larotrectinib alone, patients showing a complete or continued complete response will continue larotrectinib as maintenance therapy for up to 12 cycles, with possible extension to 24 cycles if beneficial. Those with partial response or stable disease may receive additional chemotherapy or focal radiation depending on age and clinical factors. A surgical cohort will receive larotrectinib for 3-5 days before surgery followed by larotrectinib treatment. During the study, participants will be closely monitored for disease control using imaging and for safety by tracking side effects and adverse events. Researchers will measure drug levels in blood and tumor tissue in the surgical group. The main outcomes include disease control after 2 cycles and treatment-related side effects, with survival rates assessed over time. Participation involves regular visits, treatment cycles, and follow-up evaluations for up to several years.
CONDITIONS
Brief Title
A Pilot Study of Larotrectinib for Newly-Diagnosed High-Grade Glioma With NTRK Fusion
Who Can Participate
Age: 0 - 21Years
All Genders
Eligibility Criteria
You may qualify if you...
- Patients aged from birth up to 21 years at enrollment
- Newly diagnosed high-grade glioma or diffuse intrinsic pontine glioma with confirmed NTRK fusion by approved testing
- Histologically verified high-grade glioma such as anaplastic astrocytoma, glioblastoma, or H3 K27-mutant diffuse midline glioma
- Patients with disseminated disease eligible if receiving chemotherapy only, no craniospinal radiation planned
- Patients with primary spinal tumors eligible if receiving chemotherapy or focal radiation only
- Surgical cohort eligible if prior biopsy done and further resection planned (excluding DIPG patients)
- Karnofsky performance score ≥ 50% for patients older than 16 years or Lansky score ≥ 50 for those 16 years or younger
- No prior anti-cancer chemotherapy
- Adequate bone marrow, renal, liver, pulmonary, and neurologic function
- Patients with controlled seizure disorder on medication
- Written informed consent and assent as appropriate
You will not qualify if you...
- Pregnant or breastfeeding females; pregnancy tests required for post-menarchal girls
- Patients currently or previously on other investigational drugs
- Prior or current treatment with other anti-cancer agents including chemotherapy, immunotherapy, or targeted therapy
- Active, uncontrolled systemic infections
- Previous solid organ transplantation
- Malabsorption syndrome or conditions affecting oral absorption
- Use of strong CYP3A4 inhibitors or inducers from 7 days before enrollment through study end
- Inability to comply with safety monitoring requirements
AI-Screening
AI-Powered Screening
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Initial 2 cycles of 28 days each, followed by up to 12 cycles maintenance and possible extension up to 24 cycles
Participants receive larotrectinib monotherapy twice daily orally on a 28-day cycle for 2 cycles, followed by disease evaluation. Those with continued complete or complete response continue larotrectinib monotherapy maintenance for up to 12 cycles, with possible extension to 24 cycles if benefiting clinically. Participants with partial response or stable disease may receive larotrectinib combined with chemotherapy or undergo focal radiation therapy depending on age and other factors.
2 cycles (each 28 days) followed by additional cycles depending on response and treatment plan
Surgery and Immediate Post-operative Care
Duration - 3 to 5 days pre-surgery plus subsequent treatment cycles
Participants in the surgical cohort receive larotrectinib orally twice daily for 3 to 5 days prior to definitive tumor resection surgery, followed by larotrectinib monotherapy treatment cycles as described in the treatment stage.
1 pre-surgery visit plus treatment visits post-surgery
Trial Site Locations
Total: 21 locations
1
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
2
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
3
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Withdrawn
4
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Actively Recruiting
5
Duke University Health System
Durham, North Carolina, United States, 27708
Actively Recruiting
6
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
7
Nationwide Children's Hospital
Columbus, Ohio, United States, 43235
Actively Recruiting
8
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
9
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
10
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
11
Sydney Children's Hospital
Randwick, New South Wales, Australia, 2031
Actively Recruiting
12
Queensland Children's Hospital
South Brisbane, Queensland, Australia, 4101
Actively Recruiting
13
Perth Children's Hospital
Perth, Western Australia, Australia, 6000
Actively Recruiting
14
The Hospital for Sick Children (SickKids)
Toronto, Ontario, Canada, M5G1X8
Actively Recruiting
15
Montreal Children's Hospital
Montreal, Quebec, Canada, H4A3J1
Actively Recruiting
16
Hopp Children's Cancer Center at NCT Heidelberg (KiTZ)
Heidelberg, Baden-Wurttemberg, Germany, 69120
Actively Recruiting
17
University Medical Center Augsburg
Augsburg, Germany
Actively Recruiting
18
University Hospital Berlin
Berlin, Germany
Actively Recruiting
19
University Hospital Koln
Cologne, Germany
Actively Recruiting
20
University Medical Center Gottingen
Göttingen, Germany
Actively Recruiting
21
Starship Children's Hospital
Auckland, Grafton, New Zealand, 1023
Actively Recruiting
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Research Team
K
Kelsey H Troyer, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
2
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