Optimal two-stage designs for phase II clinical trials.

Targeting BTK with ibrutinib in relapsed or refractory mantle-cell lymphoma. N Engl J Med (2013) 6.70

Phase II trial of sorafenib in advanced thyroid cancer. J Clin Oncol (2008) 5.31

Axitinib is an active treatment for all histologic subtypes of advanced thyroid cancer: results from a phase II study. J Clin Oncol (2008) 5.04

A phase II trial of gefitinib as first-line therapy for advanced non-small cell lung cancer with epidermal growth factor receptor mutations. Br J Cancer (2006) 4.46

Molecular profiling and targeted therapy for advanced thoracic malignancies: a biomarker-derived, multiarm, multihistology phase II basket trial. J Clin Oncol (2015) 4.38

Clinical trials in head injury. J Neurotrauma (2002) 4.34

Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med (2012) 3.93

PI3Kδ inhibition by idelalisib in patients with relapsed indolent lymphoma. N Engl J Med (2014) 3.86

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 (2006) 3.83

Phase II trial of sorafenib in metastatic thyroid cancer. J Clin Oncol (2009) 3.73

KIT as a therapeutic target in metastatic melanoma. JAMA (2011) 3.49

Phase II multi-institutional trial of the histone deacetylase inhibitor romidepsin as monotherapy for patients with cutaneous T-cell lymphoma. J Clin Oncol (2009) 3.13

Phase II clinical trial of sorafenib in metastatic medullary thyroid cancer. J Clin Oncol (2010) 2.96

Phase II study of sorafenib in patients with metastatic or recurrent sarcomas. J Clin Oncol (2009) 2.89

Phase 2 trial of romidepsin in patients with peripheral T-cell lymphoma. Blood (2011) 2.64

Multicenter, phase II trial of sunitinib in previously treated, advanced non-small-cell lung cancer. J Clin Oncol (2008) 2.59

Effect of bipolar androgen therapy for asymptomatic men with castration-resistant prostate cancer: results from a pilot clinical study. Sci Transl Med (2015) 2.55

Setting the bar in phase II trials: the use of historical data for determining "go/no go" decision for definitive phase III testing. Clin Cancer Res (2007) 2.50

TBCRC 001: randomized phase II study of cetuximab in combination with carboplatin in stage IV triple-negative breast cancer. J Clin Oncol (2012) 2.45

Pralatrexate in patients with relapsed or refractory peripheral T-cell lymphoma: results from the pivotal PROPEL study. J Clin Oncol (2011) 2.40

A Phase II trial of the oral mTOR inhibitor everolimus in relapsed Hodgkin lymphoma. Am J Hematol (2010) 2.27

Primary mFOLFOX6 plus bevacizumab without resection of the primary tumor for patients presenting with surgically unresectable metastatic colon cancer and an intact asymptomatic colon cancer: definitive analysis of NSABP trial C-10. J Clin Oncol (2012) 2.19

Etanercept, mycophenolate, denileukin, or pentostatin plus corticosteroids for acute graft-versus-host disease: a randomized phase 2 trial from the Blood and Marrow Transplant Clinical Trials Network. Blood (2009) 2.16

Hypothesis testing for two-stage designs with over or under enrollment. Stat Med (2015) 2.13

RTOG 0529: a phase 2 evaluation of dose-painted intensity modulated radiation therapy in combination with 5-fluorouracil and mitomycin-C for the reduction of acute morbidity in carcinoma of the anal canal. Int J Radiat Oncol Biol Phys (2012) 2.12

Lenalidomide plus Rituximab as Initial Treatment for Mantle-Cell Lymphoma. N Engl J Med (2015) 2.10

Prolonged myelosuppression with clofarabine in the treatment of patients with relapsed or refractory, aggressive non-Hodgkin lymphoma. Leuk Lymphoma (2009) 2.06

Phase 2 study of weekly irinotecan in adults with recurrent malignant glioma: final report of NABTT 97-11. Neuro Oncol (2004) 2.03

Cixutumumab and temsirolimus for patients with bone and soft-tissue sarcoma: a multicentre, open-label, phase 2 trial. Lancet Oncol (2013) 1.99

Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. Blood (2013) 1.97

Approaches to phase 1 clinical trial design focused on safety, efficiency, and selected patient populations: a report from the clinical trial design task force of the national cancer institute investigational drug steering committee. Clin Cancer Res (2010) 1.93

Phase 2 study of CEP-701, an orally available JAK2 inhibitor, in patients with primary or post-polycythemia vera/essential thrombocythemia myelofibrosis. Blood (2009) 1.82

A phase II trial of the oral mTOR inhibitor everolimus in relapsed aggressive lymphoma. Leukemia (2010) 1.81

Low-dose, single-agent temsirolimus for relapsed mantle cell lymphoma: a phase 2 trial in the North Central Cancer Treatment Group. Cancer (2008) 1.80

Alemtuzumab treatment of intermediate-1 myelodysplasia patients is associated with sustained improvement in blood counts and cytogenetic remissions. J Clin Oncol (2010) 1.79

A phase 2 clinical trial of sequential neoadjuvant chemotherapy with ifosfamide, doxorubicin, and gemcitabine followed by cisplatin, gemcitabine, and ifosfamide in locally advanced urothelial cancer: final results. Cancer (2012) 1.76

Mocetinostat for relapsed classical Hodgkin's lymphoma: an open-label, single-arm, phase 2 trial. Lancet Oncol (2011) 1.75

Is etoricoxib effective in preventing heterotopic ossification after primary total hip arthroplasty? Int Orthop (2013) 1.74

Randomized phase II designs. Clin Cancer Res (2009) 1.73

Evaluation of an unconventional cancer treatment (the Di Bella multitherapy): results of phase II trials in Italy. Italian Study Group for the Di Bella Multitherapy Trails. BMJ (1999) 1.67

Phase 2 trial of carboplatin, weekly paclitaxel, and biweekly bevacizumab in patients with unresectable stage IV melanoma: a North Central Cancer Treatment Group study, N047A. Cancer (2009) 1.66

Phase II trial of bevacizumab, thalidomide, docetaxel, and prednisone in patients with metastatic castration-resistant prostate cancer. J Clin Oncol (2010) 1.65

A multicenter phase II trial of decitabine as first-line treatment for older patients with acute myeloid leukemia judged unfit for induction chemotherapy. Haematologica (2011) 1.63

Salvage chemotherapy with rituximab DHAP for relapsed non-Hodgkin lymphoma: a phase II trial in the North Central Cancer Treatment Group. Leuk Lymphoma (2008) 1.61

A phase II study of biweekly oxaliplatin plus infusional 5-fluorouracil and folinic acid (FOLFOX-4) as first-line treatment of advanced gastric cancer patients. Br J Cancer (2005) 1.61

Ofatumumab in combination with ICE or DHAP chemotherapy in relapsed or refractory intermediate grade B-cell lymphoma. Blood (2013) 1.60

A phase II trial of 17-allylamino-17-demethoxygeldanamycin in patients with hormone-refractory metastatic prostate cancer. Clin Cancer Res (2008) 1.56

Phase 2 multi-institutional trial evaluating gemcitabine and stereotactic body radiotherapy for patients with locally advanced unresectable pancreatic adenocarcinoma. Cancer (2014) 1.55

Phase II trial of the histone deacetylase inhibitor vorinostat (Zolinza, suberoylanilide hydroxamic acid, SAHA) in patients with recurrent and/or metastatic head and neck cancer. Invest New Drugs (2007) 1.53

Phase II trial of temozolomide plus o6-benzylguanine in adults with recurrent, temozolomide-resistant malignant glioma. J Clin Oncol (2009) 1.53

Efficient identification and evaluation of effective Helicobacter pylori therapies. Clin Gastroenterol Hepatol (2008) 1.52

Evaluation of fetuses in a study of intravenous immunoglobulin as preventive therapy for congenital heart block: Results of a multicenter, prospective, open-label clinical trial. Arthritis Rheum (2010) 1.52

Phase II study of the mitogen-activated protein kinase 1/2 inhibitor selumetinib in patients with advanced hepatocellular carcinoma. J Clin Oncol (2011) 1.52

Cladribine and cytarabine in refractory multisystem Langerhans cell histiocytosis: results of an international phase 2 study. Blood (2015) 1.51

Cardiac toxicity and efficacy of trastuzumab combined with pertuzumab in patients with [corrected] human epidermal growth factor receptor 2-positive metastatic breast cancer. Clin Cancer Res (2008) 1.50

Irinotecan plus raltitrexed vs raltitrexed alone in patients with gemcitabine-pretreated advanced pancreatic adenocarcinoma. Br J Cancer (2003) 1.48

Multicenter phase 2 trial of sirolimus for tuberous sclerosis: kidney angiomyolipomas and other tumors regress and VEGF- D levels decrease. PLoS One (2011) 1.48

A multi-center phase II evaluation of the small molecule survivin suppressor YM155 in patients with unresectable stage III or IV melanoma. Invest New Drugs (2009) 1.47

Phase II clinical trial of cediranib in patients with metastatic castration-resistant prostate cancer. BJU Int (2013) 1.47

Phase II study of cetuximab in combination with cisplatin and docetaxel in patients with untreated advanced gastric or gastro-oesophageal junction adenocarcinoma (DOCETUX study). Br J Cancer (2009) 1.46

OCTET-CY: a phase II study to investigate the efficacy of post-transplant cyclophosphamide as sole graft-versus-host prophylaxis after allogeneic peripheral blood stem cell transplantation. Eur J Haematol (2015) 1.45

Phase I/II trial and pharmacokinetic study of cixutumumab in pediatric patients with refractory solid tumors and Ewing sarcoma: a report from the Children's Oncology Group. J Clin Oncol (2011) 1.45

Therapeutic regulation of myeloid-derived suppressor cells and immune response to cancer vaccine in patients with extensive stage small cell lung cancer. Cancer Immunol Immunother (2013) 1.43

Optimal timing of surgery after chemoradiation for advanced rectal cancer: preliminary results of a multicenter, nonrandomized phase II prospective trial. Ann Surg (2011) 1.43

Phase II trial of imatinib mesylate in patients with metastatic melanoma. Br J Cancer (2008) 1.43

Phase II evaluation of IPI-926, an oral Hedgehog inhibitor, in patients with myelofibrosis. Leuk Lymphoma (2015) 1.42

Cixutumumab for patients with recurrent or refractory advanced thymic epithelial tumours: a multicentre, open-label, phase 2 trial. Lancet Oncol (2014) 1.40

Lack of therapeutic effect of the histone deacetylase inhibitor vorinostat in patients with metastatic radioiodine-refractory thyroid carcinoma. J Clin Endocrinol Metab (2008) 1.40

Alemtuzumab in T-cell large granular lymphocytic leukaemia: interim results from a single-arm, open-label, phase 2 study. Lancet Haematol (2015) 1.40

A phase 2 trial of dasatinib in advanced melanoma. Cancer (2010) 1.40

Comparison of error rates in single-arm versus randomized phase II cancer clinical trials. J Clin Oncol (2010) 1.40

Fool's gold, lost treasures, and the randomized clinical trial. BMC Cancer (2013) 1.39

Final analysis of a phase II trial using sorafenib for metastatic castration-resistant prostate cancer. BJU Int (2009) 1.37

A predictive probability design for phase II cancer clinical trials. Clin Trials (2008) 1.37

Capecitabine plus oxaliplatin as first-line treatment in patients with advanced biliary system adenocarcinoma: a prospective multicentre phase II trial. Br J Cancer (2008) 1.35

Outpatient chemotherapy with gemcitabine and oxaliplatin in patients with biliary tract cancer. Br J Cancer (2006) 1.35

Phase II study and biomarker analysis of cetuximab combined with modified FOLFOX6 in advanced gastric cancer. Br J Cancer (2009) 1.34

Phase II trial of sunitinib in patients with metastatic non-clear cell renal cell carcinoma. Invest New Drugs (2010) 1.33

Shortcomings in the clinical evaluation of new drugs: acute myeloid leukemia as paradigm. Blood (2010) 1.33

A Phase II trial of 17-allylamino, 17-demethoxygeldanamycin (17-AAG, tanespimycin) in patients with metastatic melanoma. Invest New Drugs (2010) 1.30

Phase II trial of pemetrexed as second-line therapy in patients with metastatic urothelial carcinoma. Invest New Drugs (2006) 1.29

Phase II study of sorafenib in children with recurrent or progressive low-grade astrocytomas. Neuro Oncol (2014) 1.28

Phase II study of belinostat (PXD101), a histone deacetylase inhibitor, for second line therapy of advanced malignant pleural mesothelioma. J Thorac Oncol (2009) 1.28

Rituximab in combination with high-dose methylprednisolone for the treatment of fludarabine refractory high-risk chronic lymphocytic leukemia. Leukemia (2008) 1.27

Bayesian hierarchical modeling of patient subpopulations: efficient designs of Phase II oncology clinical trials. Clin Trials (2013) 1.27

A phase II trial of erlotinib in combination with bevacizumab in patients with metastatic breast cancer. Clin Cancer Res (2008) 1.25

A phase II study of AT-101 (Gossypol) in chemotherapy-sensitive recurrent extensive-stage small cell lung cancer. J Thorac Oncol (2011) 1.24

A simulation study to evaluate the impact of the number of lesions measured on response assessment. Eur J Cancer (2008) 1.24

A randomised phase II study of pegylated arginine deiminase (ADI-PEG 20) in Asian advanced hepatocellular carcinoma patients. Br J Cancer (2010) 1.23

Phase II trial of the histone deacetylase inhibitor belinostat in women with platinum resistant epithelial ovarian cancer and micropapillary (LMP) ovarian tumours. Eur J Cancer (2010) 1.22

A phase II study of pazopanib in patients with recurrent or metastatic invasive breast carcinoma: a trial of the Princess Margaret Hospital phase II consortium. Oncologist (2010) 1.22

XELOX (capecitabine plus oxaliplatin) as first-line treatment for elderly patients over 70 years of age with advanced colorectal cancer. Br J Cancer (2006) 1.21

A phase II trial of trastuzumab in combination with low-dose interleukin-2 (IL-2) in patients (PTS) with metastatic breast cancer (MBC) who have previously failed trastuzumab. Breast Cancer Res Treat (2008) 1.21

Imatinib mesylate (Gleevec) in advanced breast cancer-expressing C-Kit or PDGFR-beta: clinical activity and biological correlations. Ann Oncol (2008) 1.21

Maternal use of hydroxychloroquine is associated with a reduced risk of recurrent anti-SSA/Ro-antibody-associated cardiac manifestations of neonatal lupus. Circulation (2012) 1.20

Phase II trial of sorafenib in patients with advanced anaplastic carcinoma of the thyroid. Thyroid (2013) 1.19

Belinostat in Patients With Relapsed or Refractory Peripheral T-Cell Lymphoma: Results of the Pivotal Phase II BELIEF (CLN-19) Study. J Clin Oncol (2015) 1.19

Effective incorporation of biomarkers into phase II trials. Clin Cancer Res (2009) 1.17

Randomized phase II trials: a long-term investment with promising returns. J Natl Cancer Inst (2011) 1.17

What inference for two-stage phase II trials? BMC Med Res Methodol (2012) 1.17