Table 2: Selected ongoing and recently concluded phase II trials in castration refractory prostate cancer.

TargetDrugTrialPhasePrimary endpointClinical trial identifier number

Androgen dependent pathways

CYP17TOK-001 (galeterone)Single arm, open label, dose escalation, study of TOK-001 in chemotherapy naive mCRPC I/IISafety, PSA response (≥50%)NCT00959959

AREnzalutamide (MDV 3100)Randomized, double-blind, MDV3100 versus bicalutamide in CRPCIIPFS at 24 monthsNCT01664923
Enzalutamide (MDV 3100)Randomized, double-blind MDV3100 versus bicalutamide in CRPCIIPFS at 24 monthsNCT01288911
ARN-509Open label, single arm, ARN-509 in CRPCI/IIPSA responseNCT01171898

AR mRNA antagonistEZN-4176Open label, single arm study of EZN-4176 in CRPCIMaximum tolerated dose, safetyNCT01337518

Androgen independent pathways

Src kinaseDasatinibRandomized, open label of cediranib and dasatinib versus cediranib and placebo in CRPC resistant to docetaxelIIPFSNCT01260688
DasatinibRandomized, open label of abiraterone and prednisone with or without dasatinib in mCRPCIIPFSNCT01685125
SaracatinibRandomized, double-blind, saracatinib versus placebo in mCRPC with prior docetaxel IITime to disease progression, PFSNCT01267266
SaracatinibSingle arm, saracatinib in mCRPC IIPSA response NCT00513071

mTOREverolimus (RAD001)Single arm, RAD001 in mCRPCIIBiochemical response rateNCT00629525
Everolimus (RAD001)Single arm, RAD001 with docetaxel and bevacizumab in mCRPCI/IISafety NCT00574769
Everolimus (RAD001)Single arm, RAD001 with docetaxel in mCRPCI/IISafety, objective responseNCT00459186
Everolimus (RAD001)Single arm, carboplatin, everolimus, and prednisone in mCRPC with prior docetaxelIITime to progressionNCT01051570
TemsirolimusSingle arm, cixutumumab with temsirolimus in chemotherapy naive CRPCI/IISafety, objective response/PSA responseNCT01026623
RidaforolimusRandomized, placebo controlled, bicalutamide with or without ridaforolimus in mCRPCIIPSA response, dose limiting toxicitiesNCT00777959

IGF-1RFigitumumab (CP-751871)Randomized, noncomparative, two arm, open label, docetaxel and prednisone with and without figitumumab in mCRPCIIPSA response, objective responseNCT00313781
Cixutumumab (IMC-A12)Single arm, open label, cixutumumab in chemotherapy naive mCRPCIITime to progression, pharmacokineticsNCT00520481
Cixutumumab (IMC-A12)Single arm, cixutumumab with temsirolimus in chemotherapy naive CRPCI/IISafety, objective response/PSA response, and time to progressionNCT01026623

Bcl-2OblimersenRandomized docetaxel with or without oblimersen in mCRPCIIPSA response >30%, major toxic event rate <45%NCT00085228
R-(-)-gossypol acetic acid (AT-101)Randomized, docetaxel and prednisone with or without AT-101 in chemotherapy naive mCRPCIIOS*NCT00571675

Hsp90STA-9090Single arm, open label, STA-9090 in mCRPC with prior docetaxelIIPFS at 6 monthsNCT01270880

Hsp90AT13387Two arm, open-label, parallel group, randomized, AT13387 with or without abiraterone in mCRPC progressing on abirateroneI/IISafety and tolerability
response per PCWG2

HDACPanobinostat (LBH589)Single arm, panobinostat in mCRPC with prior docetaxelIIPFS at 24 weeksNCT00667862
Panobinostat (LBH589)Randomized panobinostat at two dose levels combined with bicalutamide for CRPCI/IISafety, dosing schedule, proportion free of progression and without symptomatic deterioration at 9 monthsNCT00878436
VorinostatSingle arm, vorinostat for mCPRC with prior chemotherapyIIPFS at 6 months**NCT00330161
SB939Open label, single arm, SB939 for recurrent or mCRPCIIPFS
PSA response

DNA methyltransferaseAzacitidineSingle arm, docetaxel retreatment, and prednisone with azacitidine in mCRPC with prior docetaxelI/IISafety
PSA and objective response

ImmunotherapyProstvac-VFRandomized, open label, flutamide with and without Prostvac-VF in non-mCRPC IITime to treatment failureNCT00450463
Prostvac-VFRandomized, open label, docetaxel and prednisone with or without Prostvac-VF in mCRPCIIOSNCT01145508
177Lu radiolabeled monoclonal antibody (Ab) HuJ591 (177Lu-J591)Randomized ketoconazole and hydrocortisone plus 177Lu-J591 or placebo (111In-J591 ) in non-mCRPC IIProportion free of radiographically evident metastases at 18 monthsNCT00859781

CYP17: Cytochrome P450 17 alpha-hydroxylase and C17,20-lyase; mCRPC: metastatic castration refractory prostate cancer; PSA: prostate-specific antigen; AR: androgen receptor; PFS: progression free survival; OS: overall survival; mTOR: mammalian target of rapamycin; IGF-1: insulin like growth factor-1; Hsp: heat shock protein; HDAC: histone deacetylases.
Primary endpoints not met.
*Primary endpoint not met.
**Significant toxicities, no one met the primary endpoint.