The goal of this study is to perform genetic sequencing on brain tumors from children, adolescents, and young adult patients who have been newly diagnosed with a high-grade glioma. This molecular profiling will decide if patients are eligible to participate in a subsequent treatment-based clinical trial based on the genetic alterations identified in their tumor.
A novel, molecularly-guided, multi-arm phase umbrella II trial is proposed in children, adolescents, and young adults with newly diagnosed HGG, including DIPG, in which we will (1) conduct comprehensive molecular screening of tumor tissue using a multi-omic approach (WES/WGS, gene fusion panels/RNASeq, DNA methylation microarray) across international CONNECT genomics cores with rapid return of clinical results, (2) stratify patients to biologically-targeted treatment arms, based on the tumor molecular profile and histopathology, and (3) perform longitudinal evaluation of peripheral blood, cerebrospinal fluid (CSF), and/or tumor tissue as well as advanced neuro-imaging to determine genomic, immune, and radiologic biomarkers predictive of response, recurrence, resistance, and toxicity. Based on results of the above tumor molecular profiling and pathology-based confirmation of HGG diagnosis, eligible patients will be assigned to one of several biologically guided treatment arms on a phase II trial. Approximately 300-350 patients will be enrolled on the screening protocol through which biospecimens (paired tumor DNA/RNA and normal comparator samples) will undergo extensive molecular profiling and/or there will be comprehensive central molecular and pathology review of previously obtained molecular results to assess eligibility to any of the therapeutic subprotocols of the phase II study.
Study Type
OBSERVATIONAL
Enrollment
350
Children's Hospital Colorado
Aurora, Colorado, United States
NOT_YET_RECRUITINGChildren's National Medical Center
Washington D.C., District of Columbia, United States
RECRUITINGAnn & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
NOT_YET_RECRUITINGDana-Farber Cancer Institute
Boston, Massachusetts, United States
NOT_YET_RECRUITINGC.S. Mott Children's Hospital
Ann Arbor, Michigan, United States
RECRUITINGDuke University Health System
Durham, North Carolina, United States
NOT_YET_RECRUITINGCincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
RECRUITINGNationwide Children's Hospital
Columbus, Ohio, United States
RECRUITINGChildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
NOT_YET_RECRUITINGTexas Children's Hospital
Houston, Texas, United States
RECRUITING...and 11 more locations
Molecular profiling
Utilize molecular, clinical, and histopathologic data to assess eligibility for specific biologically-guided treatment subprotocols among pediatric, adolescent and young adult patients with newly diagnosed HGG, including DIPG.
Time frame: 4 years
Feasibility of molecular profiling and enrollment to a TarGeT treatment protocol
Determine the percent of pediatric, adolescent, and young adult patients newly diagnosed with HGG, including DIPG, who undergo screening through one of three TarGeT-SCR screening mechanisms and are assigned to a TarGeT treatment arm.
Time frame: 4 years
Genomic Research
Increase knowledge of the genomic and immunologic landscape of newly-diagnosed pediatric and young adult HGGs, including DIPG, through comprehensive molecular characterization.
Time frame: 6 years
Germline susceptibility testing
Determine the frequency and spectrum of germline cancer susceptibility mutations in children and young adults with HGG and DIPG and assess the feasibility of return of those results.
Time frame: 4 years
Biobanking
Prospectively collect tumor tissue from diagnostic biopsy/resection as well as baseline peripheral blood and cerebrospinal fluid (CSF) samples for the CONNECT biorepository to be used in correlative research for the present trial as well as future studies.
Time frame: 4 years
Progression Free Survival
Prospectively collect data to estimate the distribution of PFS in HGG and DIPG patients, respectively, who do not enroll on any TarGeT treatment subprotocol.
Time frame: 4 years
Feasibility of Assignment
Determine the percent of pediatric, adolescent, and young adult patients newly diagnosed with HGG, including DIPG, who are assigned to a TarGeT treatment arm through one of the three TarGeT-SCR screening mechanisms, and begin treatment on a TarGeT treatment subprotocol within the required timelines.
Time frame: 4 years
Descriptively analyze patients not assigned
For patients who consent to TarGeT-SCR but do not enroll on a TarGeT treatment subprotocol: Descriptively capture and analyze reasons for lack of treatment arm enrollment.
Time frame: 4 years
Overall Survival
Prospectively collect data to estimate the distribution of OS in HGG and DIPG patients, respectively, who do not enroll on any TarGeT treatment subprotocol.
Time frame: 4 years
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