Across 121 European CTIS prostate cancer Phase II/III trials, safety/adverse events were the most frequently measured endpoint family, appearing in 85 trials (70.2%). Overall survival followed in 78 trials (64.5%), quality-of-life or patient-reported outcomes in 64 trials (52.9%), and radiographic progression-free survival in 57 trials (47.1%). Phase III trials concentrated on OS and rPFS, while Phase II trials more often used safety, PSA response, ORR, pharmacokinetics, and duration-of-response endpoints.
The ten most common endpoint families were led by safety/adverse events in 85/121 trials (70.2%), overall survival in 78/121 (64.5%), quality-of-life or patient-reported outcomes in 64/121 (52.9%), and radiographic progression-free survival in 57/121 (47.1%).
The prostate cancer endpoint stack is not built around a single efficacy measure. CTIS submissions most often combine safety, survival, radiographic progression, patient-reported outcomes, PSA biology and response endpoints, reflecting both registrational survival expectations and prostate-specific disease monitoring.
As primary endpoints, safety/adverse events appeared in 33/121 trials (27.3%), rPFS in 30/121 (24.8%) and OS in 29/121 (24.0%). As secondary endpoints, safety appeared in 67/121 (55.4%), OS in 64/121 (52.9%), QoL/PRO in 60/121 (49.6%), and PSA response plus ORR each in 41/121 (33.9%). Explicit exploratory/other endpoint labeling was uncommon: biomarkers/pharmacodynamics led that group at 5/121 trials (4.1%).
| Endpoint family | Primary | Secondary | Exploratory / other |
|---|---|---|---|
| Safety / adverse events | 33 (27.3%) | 67 (55.4%) | 3 (2.5%) |
| Overall survival | 29 (24.0%) | 64 (52.9%) | 2 (1.7%) |
| Radiographic PFS | 30 (24.8%) | 32 (26.4%) | 0 (0.0%) |
| Quality of life / PRO | 4 (3.3%) | 60 (49.6%) | 3 (2.5%) |
| PSA response / decline | 15 (12.4%) | 41 (33.9%) | 0 (0.0%) |
| Objective response rate | 6 (5.0%) | 41 (33.9%) | 0 (0.0%) |
| Biomarkers / pharmacodynamics | 1 (0.8%) | 20 (16.5%) | 5 (4.1%) |
Primary endpoint choices remain split between early-phase tolerability and later-phase disease-control or survival outcomes. The secondary endpoint layer is where sponsors most consistently add QoL, PSA kinetics, response, PK and biomarker evidence for CTIS/EU submissions.
The largest sub-disease group was mCRPC or castration-resistant prostate cancer with 51/121 trials (42.1%). In mCRPC, OS appeared in 41/51 trials (80.4%), safety in 40/51 (78.4%) and rPFS in 36/51 (70.6%). In mHSPC/castration-sensitive disease, OS appeared in 13/15 trials (86.7%), while both QoL/PRO and rPFS appeared in 12/15 (80.0%).
| Subtype | n | Endpoint #1 | Endpoint #2 | Endpoint #3 |
|---|---|---|---|---|
| mCRPC / castration-resistant | 51 | OS: 41 (80.4%) | Safety: 40 (78.4%) | rPFS: 36 (70.6%) |
| Prostate cancer, stage not specified | 25 | Safety: 15 (60.0%) | QoL/PRO: 13 (52.0%) | OS: 11 (44.0%) |
| Metastatic, subtype not specified | 16 | Safety: 10 (62.5%) | PFS: 7 (43.8%) | ORR / PK: 7 (43.8%) |
| mHSPC / castration-sensitive | 15 | OS: 13 (86.7%) | QoL/PRO: 12 (80.0%) | rPFS: 12 (80.0%) |
| Localized / recurrent / oligometastatic | 11 | Safety: 8 (72.7%) | OS: 7 (63.6%) | PFS / QoL: 6 (54.5%) |
Advanced metastatic settings drive the strongest survival and rPFS signal. mHSPC trials especially emphasize OS, rPFS and QoL together, while Phase II-heavy metastatic and unspecified cohorts retain more safety, PSA, ORR and PK content.
Among 57 Phase II-like trials, safety/adverse events appeared in 47 (82.5%), PSA response in 32 (56.1%), and ORR plus OS each in 30 (52.6%). Among 62 Phase III trials, OS appeared in 48 (77.4%), safety and QoL/PRO each in 37 (59.7%), and rPFS in 29 (46.8%). Two Phase II/III bridge trials were present and are counted in the overall total.
| Endpoint family | Pha |
|---|