Single-arm trial
A trial where everyone gets the new drug and there is no comparison group; success is judged by how many tumours shrink. Fast and small, and the basis of many accelerated approvals, but it cannot show that patients live longer.
Single-arm phase 2 trials with objective response rate and duration of response as endpoints supported accelerated approvals for many targeted drugs in rare or heavily pretreated populations (larotrectinib, sotorasib, tarlatamab, most CAR-T products) where randomisation was impractical or considered unethical. Their weaknesses are patient selection, lack of a comparator for time-to-event endpoints, and susceptibility to response-evaluable population definitions; the FDA increasingly asks for a randomised confirmatory trial to be under way at approval and has issued guidance on when single-arm data suffice. External or synthetic control arms are attempts to add a comparator after the fact.
Pages like this
not linked directly; found by shared links- TermBlinded independent central review (BICR)
Shares Blinding (double-blind, open-label, placebo-controlled), Objective response rate (ORR).
- TermControl arm and comparator (investigator's choice)
Shares Blinding (double-blind, open-label, placebo-controlled), External and synthetic control arms.
- TermConfirmatory trial
Shares Pivotal (registrational) trial, Accelerated approval.
- TermComplete response (CR) and partial response (PR)
Shares Duration of response (DoR) and disease control rate (DCR), Objective response rate (ORR).
- TermPrimary, secondary and co-primary endpoints
Shares Pivotal (registrational) trial, Objective response rate (ORR).
- Key paperSunRISe-1: TAR-200, a gemcitabine-releasing device placed in the bladder, for BCG-unresponsive non-muscle-invasive bladder cancer
- TermDuration of response
- TermOff-label
Shares Accelerated approval, Basket, umbrella, and platform trials.