Single-arm phase II trial design under parametric cure models

Phase II trial designs for complex interventions: a single-arm single-stage design for clustered continuous outcomes

To incorporate formal assessments of potential efficacy into feasibility and pilot studies of complex interventions, appropriate methods for sample size determination are required. In particular, the implications of clustered patient outcomes resulting from cluster randomisation or treatment provision must be addressed. In this paper we will show how small-sample methods used to design phase II...

Notes on Hypothesis Testing under a Single-Stage Design in Phase II Trial

Weekly ascorbic acid infusion in castration-resistant prostate cancer patients: a single-arm phase II trial

BACKGROUND Ascorbic acid (AA) has in vivo cytotoxic properties at concentrations that can only be achieved through intravenous (IV) administration in humans. Treatment with intravenous AA is widely and increasingly used in complementary medicine despite a lack of clinical evidence for the efficacy of this treatment. METHODS This non-comparative, single-center, phase II trial included patients...

Assessing the fit of parametric cure models.

Survival data can contain an unknown fraction of subjects who are "cured" in the sense of not being at risk of failure. We describe such data with cure-mixture models, which separately model cure status and the hazard of failure among non-cured subjects. No diagnostic currently exists for evaluating the fit of such models; the popular Schoenfeld residual (Schoenfeld, 1982. Partial residuals for...

Exact Operating Characteristics for Single-arm Phase II Trials

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