TABLE 1.

Recommended study designs in the assessment of focal therapy for prostate cancer

Study type	Population	Intervention	Comparator	Outcome
Phase I/II	Men due for RP	Focal ablation before RP	None	Whole-mount step-section pathology  evaluating absence of disease in  treated volume
Phase II	Low- and/or  intermediate-risk  cancer	Prostate mapping with  imaging and 3-D TPM  biopsy; focal ablation;  repeat TPM biopsy	None	Absence of (clinically significant) cancer  in treated area (derived from either  targeted TRUS-guided biopsies [with  high density of cores per mL residual  tissue] or 3-D TPM biopsy)
Phase II	Low- and/or  intermediate-risk  cancer	Image-guided biopsy; focal  ablation; post-ablation  imaging	None	Lesion ablation as defined by  post-treatment imaging
Phase III	Low- and/or low-  intermediate-risk  cancer	Focal therapy	Active surveillance	Rate of progression to radical  treatment
Phase III	Low- and/or  intermediate to  intermediate-high risk  cancer	Focal therapy	Radical whole-gland  treatment (surgery or  radiotherapy)	Freedom from metastases
Phase III	Low- and/or low-  intermediate risk  disease	Prostate mapping with 3-D  TPM biopsy + MRI,  followed by focal ablation  of all known lesions	Active surveillance	Cancer on 3-D TPM biopsy + MRI at 1  year
Phase II/III	Low- and/or  intermediate to  intermediate-high risk  cancer	Prostate mapping with  imaging and 3-D TPM  biopsy; focal ablation  monotherapy; repeat TPM  biopsy	Prostate mapping with  imaging and 3-D TPM  biopsy; focal ablation with  adjuvant chemoprevention  or low-dose radiotherapy;  repeat TPM biopsy	Rate of clinically significant prostate  cancer lesion in untreated tissue
Phase III  (pragmatic  design)	Low- and/or  intermediate-risk  disease	Focal therapy (adaptive, i.e.  incorporates any new  ablative therapy  demonstrating phase II  efficacy)	Standard of care (determined  by individual equipoise)  active surveillance or  radical therapy	Rate of systemic therapy; freedom from  progression defined according to  each treatment modality