MedPath

A Biospecimen Collection Study in BRAF-V600E Mutated Recurrent Gliomas

Terminated
Conditions
BRAF V600E
Glioma
Pleomorphic Xantho-Astrocytoma
Glioblastoma
Interventions
Procedure: Surgical Cohort
Registration Number
NCT03593993
Lead Sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Brief Summary

This is a surgical biospecimen collection study. The purpose of this study is to understand how much of two drugs (dabrafenib and trametinib) are able to penetrate brain tumors and turn off the RAF signaling pathway. This is important because these drugs are currently FDA approved for other tumors and may have efficacy in brain tumors with the BRAF V600E mutation.

Detailed Description

The primary goal of this study is to establish concentrations of dabrafenib and trametinib in enhancing brain tissue and cerebrospinal fluid from people with BRAF-V600E mutant recurrent gliomas who undergo tumor resection. The exploratory goals are to measure the amount of ERK signaling pathway activity, to identify mechanisms of resistance to these drugs, and evaluate feasibility of measuring tumor DNA in cerebrospinal fluid.

People (adults or children) with brain tumors who are already taking dabrafenib and / or trametinib and are in need of a surgical resection are potentially eligible. On the day of surgery, blood, cerebrospinal fluid, and tumor tissue will be collected for research purposes.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
3
Inclusion Criteria
  • Subjects must have a history of primary brain tumor (including but not limited to glioblastoma (GBM), anaplastic astrocytoma (AA), anaplastic ganglioglioma (AG), and anaplastic pleomorphic xanthoastrocytoma (PXA)).
  • Subjects must have a BRAF-V600 mutation identified in previous tissue analysis (may be IHC or PCR based). Allowable mutations include V600E, V600K, V600R, and V600D.
  • Subjects must be taking dabrafenib at a dose of at least 50mg twice daily (adults only) and / or trametinib at a dose of at least 1mg daily (adults only) for at least 7 days prior to surgery as prescribed by their treating physician. Note: Pediatric patients may be taking any dose of dabrafenib and / or trametinib as prescribed by their treating physician for at least 7 days prior to surgery.
  • Subjects must be undergoing surgery for clinical purposes.
  • Written informed consent - a signed informed consent and/or assent (as age appropriate) for study participation will be obtained according to institutional guidelines.
Exclusion Criteria
  • Subjects who are receiving any other investigational agents or chemotherapeutic agents.
  • Subjects for whom collection of blood, spinal fluid, or tissue samples is unsafe or clinically inadvisable.

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
Surgical CohortSurgical CohortBlood, cerebrospinal fluid, and tumor tissue will be obtained from each participant on this arm.
Primary Outcome Measures
NameTimeMethod
Concentration of trametinib in brain tumorDay 1

Obtain single time-point concentration of trametinib in enhancing brain tissue (ng/mL) using liquid chromatography/mass spectrometry with one single, random sample

Concentration of dabrafenib in brain tumorDay 1

Obtain single time-point concentration of dabrafenib in enhancing brain tissue (ng/mL) using liquid chromatography/mass spectrometry with one single, random sample

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (5)

Johns Hopkins Sidney Kimmel Comprehensive Cancer Center

🇺🇸

Baltimore, Maryland, United States

Massachusettes General Hospital

🇺🇸

Boston, Massachusetts, United States

University of Pennsylvania Abramson Cancer Center

🇺🇸

Philadelphia, Pennsylvania, United States

Wake Forest Baptist Medical Center

🇺🇸

Winston-Salem, North Carolina, United States

Penn State Hershey Medical Center

🇺🇸

Hershey, Pennsylvania, United States

© Copyright 2025. All Rights Reserved by MedPath