Most phase 1 clinical trials for anticancer agents base dose escalation on toxicity and are employed to find the maximum tolerated dose. Recently, Hunsberger et al. (Statistics in Medicine 2005; 24: 2171–2181) proposed proportion designs that are useful for molecularly targeted agents, because these new agents do not yield severe toxicity. The aim of the proportion designs is to find a dose to produce a specific high response rate. We generalize the proportion designs and derive the exact formulas of key statistical properties of the generalized proportion designs. Using the exact formulas, we compute the expected response rate at the recommended dose and the expected number of patients to finish the trials. Based on the numeric results, we recommend six generalized proportion designs.
- Exact formula
- Phase 1 trials
ASJC Scopus subject areas
- Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
- Public Health, Environmental and Occupational Health
- Pharmacology (medical)