CTNI-39. UPDATE ON GBM AGILE: A GLOBAL, PHASE 2/3 ADAPTIVE PLATFORM TRIAL TO EVALUATE MULTIPLE REGIMENS IN NEWLY DIAGNOSED AND RECURRENT GLIOBLASTOMA

Abstract BACKGROUND GBM AGILE (Glioblastoma Adaptive, Global, Innovative Learning Environment) is a biomarker based, multi-arm, international, seamless Phase 2/3 platform trial designed to rapidly identify and confirm experimental therapies that improve overall survival for approval along with their associated biomarker signatures. GBM AGILE is a collaboration between academic investigators, patient organizations, and industry to support new drug applications for newly diagnosed (ND) and recurrent glioblastoma. METHODS The primary objective of GBM AGILE is to identify therapies that improve the overall survival in patients with ND or recurrent glioblastoma. Bayesian response adaptive randomization is used within subtypes of the disease to assign participants to investigational arms based on their performance. GBM AGILE operates under a Master Protocol allowing multiple drugs to be evaluated simultaneously and over time against a common control. New experimental therapies are added as information about promising new drugs is identified, while therapies are removed as they complete evaluation. Six investigational arms have been included to date. Along with an adaptive trial design, shared control arm and operational processes, GBM AGILE continues to incorporate new design and operational elements. One recent design element added to the Master Protocol for consideration for future arms is an extended evaluation period, of up to 11 months, for arms that reach maximum sample size in Stage 1 (Stage 1: learn most responsive signature) without having reached a decision for futility or graduation. Allowing data to mature and additional events to accumulate may increase the ability of the trial to detect efficacy, and if appropriate, for an arm to graduate to Stage 2 (Stage 2: confirm activity in graduating signature). Using improved and flexible processes, GBM AGILE continues to serve as a global trial that supports the efficient and rapid incorporation and evaluation of new experimental therapies for patients with glioblastoma. NCT number: NCT03970447.

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Publication Details

Journal
Neuro-Oncology
Published
2024-11-01
DOI
https://doi.org/10.1093/neuonc/noae165.0406
Primary Topic
Glioma Diagnosis and Treatment
Type
article
Field-Weighted Citation Impact
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article

CTNI-39. UPDATE ON GBM AGILE: A GLOBAL, PHASE 2/3 ADAPTIVE PLATFORM TRIAL TO EVALUATE MULTIPLE REGIMENS IN NEWLY DIAGNOSED AND RECURRENT GLIOBLASTOMA

Mustafa Khasraw, Emma Maria Viktoria Hyddmark, Hui Kong Gan, Michael Lim et al.
Neuro-Oncology
Glioma Diagnosis and Treatment
article

CTNI-39. UPDATE ON GBM AGILE: A GLOBAL, PHASE 2/3 ADAPTIVE PLATFORM TRIAL TO EVALUATE MULTIPLE REGIMENS IN NEWLY DIAGNOSED AND RECURRENT GLIOBLASTOMA

Mustafa Khasraw, Emma Maria Viktoria Hyddmark, Hui Kong Gan, Michael Lim, Michael Weller, Ingo K. Mellinghoff, Patrick Y. Wen, Benjamin M. Ellingson, A. Peter Johnson, E. Lee, Kirk Tanner, Phioanh Leia Nghiemphu, Antje Wick, Ashley A. Powell, Howard Colman, Brian M. Alexander, Meredith Buxton, Erik P. Sulman, John de Groot, François Ducray, Andrew B. Lassman, Jan Drappatz, Timothy F. Cloughesy, Sarah Untch, Tom Mikkelsen, Donald A. Berry, Nicholas Berry, Anna McGlothlin, James Perry, Macarena de la Fuente, Alfred Yung, Gary Gordon
article en

Abstract

Abstract BACKGROUND GBM AGILE (Glioblastoma Adaptive, Global, Innovative Learning Environment) is a biomarker based, multi-arm, international, seamless Phase 2/3 platform trial designed to rapidly identify and confirm experimental therapies that improve overall survival for approval along with their associated biomarker signatures. GBM AGILE is a collaboration between academic investigators, patient organizations, and industry to support new drug applications for newly diagnosed (ND) and recurrent glioblastoma. METHODS The primary objective of GBM AGILE is to identify therapies that improve the overall survival in patients with ND or recurrent glioblastoma. Bayesian response adaptive randomization is used within subtypes of the disease to assign participants to investigational arms based on their performance. GBM AGILE operates under a Master Protocol allowing multiple drugs to be evaluated simultaneously and over time against a common control. New experimental therapies are added as information about promising new drugs is identified, while therapies are removed as they complete evaluation. Six investigational arms have been included to date. Along with an adaptive trial design, shared control arm and operational processes, GBM AGILE continues to incorporate new design and operational elements. One recent design element added to the Master Protocol for consideration for future arms is an extended evaluation period, of up to 11 months, for arms that reach maximum sample size in Stage 1 (Stage 1: learn most responsive signature) without having reached a decision for futility or graduation. Allowing data to mature and additional events to accumulate may increase the ability of the trial to detect efficacy, and if appropriate, for an arm to graduate to Stage 2 (Stage 2: confirm activity in graduating signature). Using improved and flexible processes, GBM AGILE continues to serve as a global trial that supports the efficient and rapid incorporation and evaluation of new experimental therapies for patients with glioblastoma. NCT number: NCT03970447.

Neuro-OncologyVol. 26(Supplement_8)
Memorial Sloan Kettering Cancer Center (US), NewYork–Presbyterian Hospital (US), The University of Texas MD Anderson Cancer Center (US), University of Miami (US), Lyon College (US), University of Toronto (CA), University of Zurich (CH), University of Utah (US), Heidelberg University (DE), UCLA Health (US), New York Hospital Queens (US), Huntsman Cancer Institute (US), University Hospital Heidelberg (DE), Presbyterian Hospital (US), Berry & Associates (United States) (US), University Hospital of Zurich (CH), Dana-Farber Cancer Institute (US), Duke Medical Center (US), University of Pittsburgh Medical Center (US), National Brain Tumor Society (US), Dana-Farber Brigham Cancer Center (US), Austin Health (AU), Herbert Irving Comprehensive Cancer Center, New York University (US), Columbia University (US), Stanford University (US)
Openalex Percentile: Top 64%
Glioma Diagnosis and Treatment
DOIOpen Access PDFOpenAlex
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