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Table 1.

Three early-phase designs for convergent prevention and therapy trials

NameCohortEnd point(s)AgentsDurationNCorrelative opportunities
Dose-finding Cancer (one type) Toxicity Oral molecular-targeted agents Highly variable* Highly variable* Preliminary biomarker responses 
Therapy with imbedded prevention end points Advanced cancer (one type) Tumor response; imbedded IEN response Oral molecular-targeted agents Highly variable Highly variable Target modulation and drug effects (dosing, mechanism); predictive markers of response, resistance 
Preresection, window of opportunity IEN and early-stage cancer (stratified) Tumor and IEN response Oral molecular-targeted agents 3-6 weeks 20-30 patients per arm Target modulation and drug effects (dosing, mechanism); predictive markers of response, resistance 
NameCohortEnd point(s)AgentsDurationNCorrelative opportunities
Dose-finding Cancer (one type) Toxicity Oral molecular-targeted agents Highly variable* Highly variable* Preliminary biomarker responses 
Therapy with imbedded prevention end points Advanced cancer (one type) Tumor response; imbedded IEN response Oral molecular-targeted agents Highly variable Highly variable Target modulation and drug effects (dosing, mechanism); predictive markers of response, resistance 
Preresection, window of opportunity IEN and early-stage cancer (stratified) Tumor and IEN response Oral molecular-targeted agents 3-6 weeks 20-30 patients per arm Target modulation and drug effects (dosing, mechanism); predictive markers of response, resistance 
*

Reflecting the variability seen in optimal biological dose-finding phase I therapy trials.

Ranging from the durations and sample sizes of phase II therapy trials to durations and sizes of phase III adjuvant trials.

The number of patients can vary widely and beyond the variables noted in the column, depending on target response rates and other factors of the design.

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