NCT03598244

Volitinib in Treating Patients With Recurrent or Refractory Primary CNS Tumors

A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Medulloblastoma, High-Grade Glioma, Diffuse Intrinsic Pontine Glioma, and CNS Tumors Harboring MET Aberrations

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 41 (actual)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 6 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

This phase I trial studies the side effects and best dose of volitinib in treating patients with primary central nervous system (CNS) tumors that have come back (recurrent) or does not respond to treatment (refractory). Volitinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Detailed description

PRIMARY OBJECTIVES: I. To estimate the maximum tolerated dose (MTD) and recommend a phase II dose of volitinib (savolitinib) administered orally daily in children with refractory, progressive or recurrent primary CNS tumors. II. To define and describe the toxicities of savolitinib in children with refractory, progressive, or recurrent primary CNS tumors. III. To characterize the pharmacokinetics of savolitinib in children with refractory, progressive, or recurrent primary CNS tumors. SECONDARY OBJECTIVES: I. To preliminarily define the antitumor activity of savolitinib within the confines of a phase I study. II. To perform a genomic analysis within the confines of a phase I study to investigate correlation between response to treatment (as measured by objective response or progression free survival \[PFS\]) and the presence of specific genomic alterations (e.g., MET or HGF amplification, MET mutations, or MET fusion) and/or specific subgroups of disease. OUTLINE: This is a dose-escalation study of volitinib followed by a dose-expansion study. Patients receive volitinib orally (PO) once daily (QD). Treatment repeats every 28 days for up to 39 courses in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo blood sample collection X-ray imaging, and magnetic resonance imaging (MRI) scans throughout study. After completion of study treatment, patients are followed up at 30 days, then periodically for up to 2 years.

Conditions

• Recurrent Diffuse Intrinsic Pontine Glioma
• Recurrent Malignant Glioma
• Recurrent Medulloblastoma
• Recurrent Primary Central Nervous System Neoplasm
• Refractory Diffuse Intrinsic Pontine Glioma
• Refractory Malignant Glioma
• Refractory Medulloblastoma
• Refractory Primary Central Nervous System Neoplasm

Interventions

Type	Name	Description
PROCEDURE	Biospecimen Collection	Undergo blood sample collection
PROCEDURE	Magnetic Resonance Imaging	Undergo MRI scan
DRUG	Savolitinib	Given PO
PROCEDURE	X-Ray Imaging	Undergo X-ray imaging

Timeline

• Start date: 2018-11-27
• Primary completion: 2025-04-14
• Completion: 2026-04-14
• First posted: 2018-07-26
• Last updated: 2025-04-20

Locations

12 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study