Brain Malignancy Steering Committee clinical trials planning workshop: Report from the Targeted Therapies Working Group

Neuro-Oncology

Volumn 17, Issue 2, 2015, Pages 180-188

  Alexander, Brian M ^a   Galanis, Evanthia ^b   Yung, W K Alfred ^c   Ballman, Karla V ^b   Boyett, James M ^d   Cloughesy, Timothy F ^e   Degroot, John F ^c   Huse, Jason T ^f   Mann, Bhupinder ^g   Mason, Warren ^h   Mellinghoff, Ingo K ^f   Mikkelsen, Tom ⁱ   Mischel, Paul S ^j   O'Neill, Brian P ^b   Prados, Michael D ^k   Sarkaria, Jann N ^b   Tawab Amiri, Abdul ^g   Trippa, Lorenzo ^a   Ye, Xiaobu ^l   Ligon, Keith L ^a   Berry, Donald A ^c   Wen, Patrick Y ^a   more..

^a HARVARD MEDICAL SCHOOL   (United States)

^b MAYO CLINIC   (United States)

^c UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

^d ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^e UNIVERSITY OF CALIFORNIA   (United States)

^f MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

^g NATIONAL CANCER INSTITUTE   (United States)

^h PRINCESS MARGARET HOSPITAL   (Canada)

ⁱ HENRY FORD HOSPITAL   (United States)

^j UNIVERSITY OF CALIFORNIA   (United States)

^k UNIVERSITY OF CALIFORNIA   (United States)

^l JOHNS HOPKINS UNIVERSITY   (United States)

more..

Author keywords

adaptive randomization; biomarkers; clinical trials; glioblastoma

Indexed keywords

3 (2 AMINO 5 BENZOXAZOLYL) 1 ISOPROPYL 1H PYRAZOLO[3,4 D]PYRIMIDIN 4 AMINE; ABEMACICLIB; BEVACIZUMAB; BIOLOGICAL MARKER; BUPARLISIB; DACOMITINIB; ISOCITRATE DEHYDROGENASE 1; LAPATINIB; METHYLATED DNA PROTEIN CYSTEINE METHYLTRANSFERASE; NERATINIB; PALBOCICLIB; PHOSPHATIDYLINOSITOL 3 KINASE; PROTEIN P53; RIBOCICLIB; TEMOZOLOMIDE; VOXTALISIB;

BLOOD BRAIN BARRIER; BRAIN CANCER; CANCER PROGNOSIS; CLINICAL TRIAL (TOPIC); GLIOBLASTOMA; HUMAN; IMMUNOHISTOCHEMISTRY; MOLECULARLY TARGETED THERAPY; OVERALL SURVIVAL; PROGRESSION FREE SURVIVAL; QUALITY OF LIFE; REVIEW; BIOASSAY; BRAIN NEOPLASMS;

BIOMARKERS; BRAIN NEOPLASMS; CLINICAL TRIALS AS TOPIC; ENDPOINT DETERMINATION; GLIOBLASTOMA; HUMANS;

EID: 84924972234     PISSN: 15228517     EISSN: 15235866     Source Type: Journal    
DOI: 10.1093/neuonc/nou154     Document Type: Review

References (48)

1. Quant EC, Drappatz J, Wen PY, et al. Recurrent high-grade glioma. Curr Treat Options Neurol. 2010;12(4):321-333.
2. Wen PY, Kesari S. Malignant gliomas in adults. N Engl J Med. 2008; 359(5):492-507.
3. Noushmehr H, Weisenberger DJ, Diefes K, et al. Identification of a CpG island methylator phenotype that defines a distinct subgroup of glioma. Cancer Cell. 2010;17(5):510-522.
4. Verhaak RG, Hoadley KA, Purdom E, et al. Integrated genomic analysis identifies clinically relevant subtypes of glioblastoma characterized by abnormalities in PDGFRA, IDH1, EGFR, and NF1. Cancer Cell. 2010;17(1):98-110.
5. Brennan CW, Verhaak RG, McKenna A, et al. The somatic genomic landscape of glioblastoma. Cell. 2013;155(2):462-477.
6. Comprehensive genomic characterization defines human glioblastoma genes and core pathways. Nature. 2008; 455(7216):1061-1068.
7. Phillips HS, Kharbanda S, Chen R, et al. Molecular subclasses of high-grade glioma predict prognosis, delineate a pattern of disease progression, and resemble stages in neurogenesis. Cancer Cell. 2006;9(3):157-173.
8. Parsons DW, Jones S, Zhang X, et al. An integrated genomic analysis of human glioblastoma multiforme. Science. 2008; 321(5897):1807-1812.
9. Reitman ZJ, Jin G, Karoly ED, et al. Profiling the effects of isocitrate dehydrogenase 1 and 2 mutations on the cellular metabolome. Proc Natl Acad Sci USA. 2011;108(8):3270-3275.
10. Hegi ME, Diserens AC, Gorlia T, et al. MGMT gene silencing and benefit from temozolomide in glioblastoma. N Engl J Med. 2005; 352(10):997-1003.
11. Stupp R, Mason WP, van den Bent MJ, et al. Radiotherapy plus concomitant and adjuvant temozolomide for glioblastoma. N Engl J Med. 2005;352(10):987-996.
12. Alexander BM, Mehta MP. Role of isocitrate dehydrogenase in glioma. Expert Rev Neurother. 2011;11(10):1399-1409.
13. Malmström A, Grønberg BH, Marosi C, et al. Temozolomide versus standard 6-week radiotherapy versus hypofractionated radiotherapy in patients older than 60 years with glioblastoma: the Nordic randomised, phase 3 trial. Lancet Oncol. 2012;13(9): 916-926.
14. Wick W, Platten M, Meisner C, et al. Temozolomide chemotherapy alone versus radiotherapy alone for malignant astrocytoma in the elderly: the NOA-08 randomised, phase 3 trial. Lancet Oncol. 2012; 13(7):707-715.
15. United States Department of Health and Human Services FDA. Guidance for Industry: Enrichment Strategies for Clinical Trials to Support Approval of Human Drugs and Biological Products. FDA: Maryland, USA; 2012.
16. Gilbert MR, Dignam JJ, Armstrong TS, et al. A randomized trial of bevacizumab for newly diagnosed glioblastoma. New Engl J Med. 2014;370(8):699-708.
17. Northcott PA, Shih DJ, Remke M, et al. Rapid, reliable, and reproducible molecular sub-grouping of clinical medulloblastoma samples. Acta Neuropathol. 2012;123(4): 615-626.
18. Dias-Santagata D, Akhavanfard S, David SS, et al. Rapid targeted mutational analysis of human tumours: a clinical platform to guide personalized cancer medicine. EMBO Mol Med. 2010;2(5): 146-158.
19. MacConaill LE, Campbell CD, Kehoe SM, et al. Profiling critical cancer gene mutations in clinical tumor samples. PLoS One. 2009;4(11):e7887.
20. Omuro A, Chan TA, Abrey LE, et al. Phase II trial of continuous low-dose temozolomide for patients with recurrent malignant glioma. Neuro Oncol. 2013;15(2):242-250.
21. Wagle N, Berger MF, Davis MJ, et al. High-throughput detection of actionable genomic alterations in clinical tumor samples by targeted, massively parallel sequencing. Cancer Discov. 2012; 2(1):82-93.
22. Frampton GM, Fichtenholtz A, Otto GA, et al. Development and validation of a clinical cancer genomic profiling test based on massively parallel DNA sequencing. Nature Biotech. 2013;31(11): 1023-1031.
23. Craig JM, Vena N, Ramkissoon S, et al. DNA fragmentation simulation method (FSM) and fragment size matching improve aCGH performance of FFPE tissues. PLoS One. 2012;7(6):e38881.
24. United States Department of Health and Human Services FDA. Guidance for Industry: Clinical Pharmacogenomics: Premarket Evaluation in Early-Phase Clinical Studies and Recommendations for Labeling, FDA: Maryland, USA; 2013.
25. United States Department of Health and Human Services FDA. Guidance for Industry: Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics, FDA: Maryland, USA; 2007.
26. Stupp R, Hegi ME, Mason WP, et al. Effects of radiotherapy with concomitant and adjuvant temozolomide versus radiotherapy alone on survival in glioblastoma in a randomised phase III study: 5-year analysis of the EORTC-NCIC trial. Lancet Oncol. 2009;10(5):459-466.
27. Wen PY, Macdonald DR, Reardon DA, et al. Updated response assessment criteria for high-grade gliomas: response assessment in neuro-oncology working group. J Clin Oncol. 2010;28(11):1963-1972.
28. Brandes AA, Franceschi E, Tosoni A, et al. MGMT promoter methylation status can predict the incidence and outcome of pseudoprogression after concomitant radiochemotherapy in newly diagnosed glioblastoma patients. J Clin Oncol. 2008; 26(13):2192-2197.
29. Norden AD, Drappatz J, Muzikansky A, et al. An exploratory survival analysis of anti-angiogenic therapy for recurrent malignant glioma. J Neurooncol. 2009;92(2):149-155.
30. Broglio KR, Berry DA. Detecting an overall survival benefit that is derived from progression-free survival. J Natl Cancer Inst. 2009; 101(23):1642-1649.
31. Ballman KV, Buckner JC, Brown PD, et al. The relationship between six-month progression-free survival and 12-month overall survival end points for phase II trials in patients with glioblastoma multiforme. Neuro Oncol. 2007;9(1):29-38.
32. Polley MY, Lamborn KR, Chang SM, et al. Six-month progression-free survival as an alternative primary efficacy endpoint to overall survival in newly diagnosed glioblastoma patients receiving temozolomide. Neuro Oncol. 2010;12(3): 274-282.
33. Han K, Ren M, Wick W, et al. Progression-free survival as a surrogate endpoint for overall survival in glioblastoma: a literature-based meta-analysis from 91 trials. Neuro Oncol. 2014;16(5):696-706.
34. Ye X, Grossman SA, Desideri S, et al. Correlation of treatment effects on progression-free survival at 6 months (PFS-6) and overall survival (OS) in patients with newly diagnosed glioblastoma (GBM). J Clin Oncol. 2011;29: (suppl; abstr 2008).
35. Chinot OL, Wick W, Mason W, et al. Bevacizumab plus radiotherapy-temozolomide for newly diagnosed glioblastoma. New Engl J Med. 2014;370(8):709-722.
36. Alexander BM, Trippa L. Progression-free survival: too much risk, not enough reward? Neuro Oncol. 2014;16(5):615-616.
37. Tajik P, Zwinderman AH, Mol BW, et al. Trial designs for personalizing cancer care: a systematic review and classification. Clin Cancer Res. 2013;19(17):4578-4588.
38. Freidlin B, Korn EL. Adaptive randomization versus interim monitoring. J Clin Oncol. 2013;31(7):969-970.
39. Berry DA. Adaptive clinical trials in oncology. Nat Rev Clin Oncol. 2012;9(4):199-207.
40. Trippa L, Lee EQ, Wen PY, et al. Reply to B. Freidlin et al. J Clin Oncol. 2013;31(7):970-971.
41. Trippa L, Lee EQ, Wen PY, et al. Bayesian adaptive randomized trial design for patients with recurrent glioblastoma. J Clin Oncol. 2012; 30(26):3258-3263.
42. Barker AD, Sigman CC, Kelloff GJ, et al. I-SPY 2: an adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clin Pharmacol Ther. 2009;86(1):97-100.
43. Alexander BM, Wen PY, Trippa L, et al. Biomarker-based adaptive trials for patients with glioblastoma-lessons from I-SPY 2. Neuro Oncol. 2013;15(8):972-978.
44. Galanis E, Wu W, Cloughesy T, et al. Phase 2 trial design in neuro-oncology revisited: a report from the RANO group. Lancet Oncol. 2012;13(5):e196-e204.
45. Berry DA. Adaptive Clinical Trials: The Promise and the Caution. J Clin Oncol. 2011;29(6):606-609.
46. Gilbert MR, Wang M, Aldape KD, et al. Dose-dense temozolomide for newly diagnosed glioblastoma: a randomized phase III clinical trial. J Clin Oncol. 2013;31(32):4085-4091.
47. Johnson BE, Mazor T, Hong C, et al. Mutational analysis reveals the origin and therapy-driven evolution of recurrent glioma. Science. 2014;343(6167):189-193.
48. Malmstrom A, Gronberg BH, Marosi C, et al. Temozolomide versus standard 6-week radiotherapy versus hypofractionated radiotherapy in patients older than 60 years with glioblastoma: the Nordic randomised, phase 3 trial. Lancet Oncol. 2012;13(9): 916-926.