# Pivotal (registrational) trial

Source: https://onco.cc/terms/pivotal-trial/  
OnCo record `pivotal-trial` (Term). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

The trial (usually a randomised phase 3, sometimes a large phase 2) whose results are submitted to regulators as the main evidence for approval. When a company calls a trial 'registrational' it is signalling that this is the one that decides.

## Summary

Regulators traditionally required two adequate and well-controlled trials, but in oncology one pivotal trial with confirmatory supporting evidence is the norm. Pivotal trials define the approved population, dose and comparator, so their design is negotiated with the FDA in advance (end-of-phase-2 meetings, special protocol assessment). Phase 2 single-arm cohorts serve as pivotal for accelerated approval in rare or refractory diseases. A 'registration-directed' or 'registration-enabling' phase 2 signals a company's intent to file on it. Pivotal trials are the units this site's evidence and trial pages are built around.

## Fields

- Kind: Term
- Last checked: 2026-09-09
- Also known as: pivotal; pivotal trial; pivotal study; pivotal phase 3; registrational; registrational trial; registrational study; registration trial; registration-enabling; registration-directed; approval-enabling; pivotal cohort; registrational cohort; supported approval; basis of approval; approval was based on; registration trials; registrational trials

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Phases_of_clinical_research
- Wikipedia: https://en.wikipedia.org/wiki/Phases_of_clinical_research

## Connected records

- terms: [BLA / NDA / MAA (marketing applications) and PDUFA dates](https://onco.cc/terms/bla-nda/), [Confirmatory trial](https://onco.cc/terms/confirmatory-trial/), [Group sequential design, stopping rules and alpha spending](https://onco.cc/terms/group-sequential-design/), [Phase 1, 2 and 3 trials](https://onco.cc/terms/trial-phases/), [Primary, secondary and co-primary endpoints](https://onco.cc/terms/primary-endpoint/), [Single-arm trial](https://onco.cc/terms/single-arm/), [Trial lifecycle: from protocol to label](https://onco.cc/terms/trial-lifecycle/), [Trial registration and results reporting (ClinicalTrials.gov, EU CTR)](https://onco.cc/terms/trial-registration/)
- fronts: [Drug Discovery Platforms](https://onco.cc/fronts/drug-discovery/)
- trials: [ADAURA](https://onco.cc/trials/adaura/), [KEYNOTE-522](https://onco.cc/trials/keynote-522/), [NAVIGATE](https://onco.cc/trials/navigate/)
- drugs: [Linvoseltamab](https://onco.cc/drugs/linvoseltamab/), [Moxetumomab pasudotox](https://onco.cc/drugs/moxetumomab-pasudotox/), [Olutasidenib](https://onco.cc/drugs/olutasidenib/)
- ideas: [Exposure-response modelling with a pre-set target exposure before any dose is chosen](https://onco.cc/ideas/idea-tr1-exposure-response-before-dose-selection/)
- institutions: [Chongqing University Cancer Hospital](https://onco.cc/institutions/chongqing-university-cancer-hospital/)
- companies: [Abbisko Therapeutics](https://onco.cc/companies/abbisko/), [ABX advanced biochemical compounds](https://onco.cc/companies/abx-advanced-biochemical-compounds/), [Acacia Pharma](https://onco.cc/companies/acacia-pharma/), [Active Biotech](https://onco.cc/companies/active-biotech/), [Advenchen Laboratories](https://onco.cc/companies/advenchen-laboratories/), [Aeterna Zentaris](https://onco.cc/companies/aeterna-zentaris/), [Agennix](https://onco.cc/companies/agennix/), [Almirall](https://onco.cc/companies/almirall/), [Angiocrine Bioscience](https://onco.cc/companies/angiocrine-bioscience/), [AOP Health](https://onco.cc/companies/aop-health/), [Argos Therapeutics](https://onco.cc/companies/argos-therapeutics/), [Arog Pharmaceuticals](https://onco.cc/companies/arog-pharmaceuticals/), [Ascletis Pharma](https://onco.cc/companies/ascletis/), [Atossa Therapeutics](https://onco.cc/companies/atossa-therapeutics/), [Bavarian Nordic](https://onco.cc/companies/bavarian-nordic/), [Bellicum Pharmaceuticals](https://onco.cc/companies/bellicum/), [Blaze Bioscience](https://onco.cc/companies/blaze-bioscience/), [Can-Fite BioPharma](https://onco.cc/companies/can-fite/), [Cancer Prevention Pharmaceuticals](https://onco.cc/companies/cancer-prevention-pharmaceuticals/), [Cartesian Therapeutics](https://onco.cc/companies/cartesian-therapeutics/), [CEBIOTEX](https://onco.cc/companies/cebiotex/), [CellSeed](https://onco.cc/companies/cellseed/), [Changchun GeneScience Pharmaceutical](https://onco.cc/companies/changchun-genescience/), [Chongqing Precision Biotech](https://onco.cc/companies/chongqing-precision-biotech/), [Corbus Pharmaceuticals](https://onco.cc/companies/corbus-pharmaceuticals/), [CSL Limited](https://onco.cc/companies/csl/), [Cyclacel Pharmaceuticals](https://onco.cc/companies/cyclacel/), [D3 Bio (Wuxi)](https://onco.cc/companies/d3-bio-wuxi/), [Daehwa Pharmaceutical](https://onco.cc/companies/daehwa-pharmaceutical/), [Delta-Fly Pharma](https://onco.cc/companies/delta-fly-pharma/), [Denovo Biopharma](https://onco.cc/companies/denovo-biopharma/), [Diffusion Pharmaceuticals](https://onco.cc/companies/diffusion-pharmaceuticals/), [Dong-A ST](https://onco.cc/companies/dong-a-st/), [Emergent BioSolutions](https://onco.cc/companies/emergent-biosolutions/), [Endo (now Keenova Therapeutics and Par Health)](https://onco.cc/companies/endo/), [Eurofarma](https://onco.cc/companies/eurofarma/), [Foresee Pharmaceuticals](https://onco.cc/companies/foresee-pharmaceuticals/), [Forward Pharmaceuticals](https://onco.cc/companies/forward-pharmaceuticals/), [FutureChem](https://onco.cc/companies/futurechem/), [Galena Biopharma](https://onco.cc/companies/galena-biopharma/), [Galera Therapeutics](https://onco.cc/companies/galera-therapeutics/), [GC Cell](https://onco.cc/companies/gc-cell/), [Georgiamune](https://onco.cc/companies/georgiamune/), [Grifols](https://onco.cc/companies/grifols/), [Halozyme 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Pharmaceutical](https://onco.cc/companies/kissei/), [Lee's Pharmaceutical](https://onco.cc/companies/lees-pharmaceutical/), [LEO Pharma](https://onco.cc/companies/leo-pharma/), [Lisata Therapeutics](https://onco.cc/companies/lisata/), [Luye Pharma Group](https://onco.cc/companies/luye-pharma/), [Mabion](https://onco.cc/companies/mabion/), [Mast Therapeutics](https://onco.cc/companies/mast-therapeutics/), [Medigene](https://onco.cc/companies/medigene/), [Merrimack Pharmaceuticals](https://onco.cc/companies/merrimack-pharmaceuticals/), [Moleculin Biotech](https://onco.cc/companies/moleculin/), [Monopar Therapeutics](https://onco.cc/companies/monopar/), [Mundipharma](https://onco.cc/companies/mundipharma/), [Nippon Kayaku](https://onco.cc/companies/nippon-kayaku/), [Nobelpharma](https://onco.cc/companies/nobelpharma/), [Norgine](https://onco.cc/companies/norgine/), [Oakwood Laboratories](https://onco.cc/companies/oakwood-laboratories/), [Organon & Co.](https://onco.cc/companies/organon/), 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JSON: https://onco.cc/api/v1/entities/pivotal-trial.json