A Bayesian predictive strategy for an adaptive two-stage design in phase II clinical trials

Statistics in Medicine
Valeria Sambucini

Abstract

Phase II clinical trials are typically designed as two-stage studies, in order to ensure early termination of the trial if the interim results show that the treatment is ineffective. Most of two-stage designs, developed under both a frequentist and a Bayesian framework, select the second stage sample size before observing the first stage data. This may cause some paradoxical situations during the practical carrying out of the trial. To avoid these potential problems, we suggest a Bayesian predictive strategy to derive an adaptive two-stage design, where the second stage sample size is not selected in advance, but depends on the first stage result. The criterion we propose is based on a modification of a Bayesian predictive design recently presented in the literature (see (Statist. Med. 2008; 27:1199-1224)). The distinction between analysis and design priors is essential for the practical implementation of the procedure: some guidelines for choosing these prior distributions are discussed and their impact on the required sample size is examined.

References

Mar 1, 1989·Controlled Clinical Trials·R Simon
Aug 30, 1997·Statistics in Medicine·D F Heitjan
Feb 28, 2002·Annals of Oncology : Official Journal of the European Society for Medical Oncology·K F FooD Machin
Jul 12, 2002·Statistics in Medicine·Say-Beng Tan, David Machin
Mar 17, 2004·Controlled Clinical Trials·Matthew S Mayo, Byron J Gajewski
Jun 8, 2004·Biometrics·Yong Lin, Weichung J Shih
Dec 14, 2005·Statistics in Medicine·Byron J Gajewski, Matthew S Mayo
Feb 16, 2006·Statistics in Medicine·Anindita Banerjee, Anastasios A Tsiatis
Jul 19, 2006·Statistics in Medicine·Say-Beng Tan, David Machin
Sep 1, 2007·Statistics in Medicine·Valeria Sambucini
Jan 22, 2008·Statistics in Medicine·Pierpaolo BruttiStefania Gubbiotti

❮ Previous
Next ❯

Citations

Aug 13, 2011·Journal of Biopharmaceutical Statistics·H Amy XiaBradley P Carlin
Aug 13, 2011·Journal of Biopharmaceutical Statistics·Gregory Campbell
Aug 13, 2011·Journal of Biopharmaceutical Statistics·Wen Li, Haoda Fu
Jan 22, 2013·Journal of Biopharmaceutical Statistics·Christy Chuang-Stein, H Amy Xia
Jul 19, 2012·Statistics in Medicine·Satoshi TeramukaiSarah Zohar
Dec 30, 2014·Statistics in Medicine·Matteo Cellamare, Valeria Sambucini
Jun 5, 2014·Journal of Biopharmaceutical Statistics·Valeria Sambucini
Sep 1, 2018·Pharmaceutical Statistics·Kosuke Kashiwabara, Yutaka Matsuyama
Sep 28, 2010·Journal of Pharmacokinetics and Pharmacodynamics·Alan MaloneyUlrika S H Simonsson
Aug 13, 2011·Journal of Biopharmaceutical Statistics·John A ScottLayla S Sian
Jan 1, 2015·Therapeutic Innovation & Regulatory Science·Oleksandr SverdlovSheng Wu

❮ Previous
Next ❯

Related Concepts

Trending Feeds

COVID-19

Coronaviruses encompass a large family of viruses that cause the common cold as well as more serious diseases, such as the ongoing outbreak of coronavirus disease 2019 (COVID-19; formally known as 2019-nCoV). Coronaviruses can spread from animals to humans; symptoms include fever, cough, shortness of breath, and breathing difficulties; in more severe cases, infection can lead to death. This feed covers recent research on COVID-19.

Blastomycosis

Blastomycosis fungal infections spread through inhaling Blastomyces dermatitidis spores. Discover the latest research on blastomycosis fungal infections here.

Nuclear Pore Complex in ALS/FTD

Alterations in nucleocytoplasmic transport, controlled by the nuclear pore complex, may be involved in the pathomechanism underlying multiple neurodegenerative diseases including Amyotrophic Lateral Sclerosis and Frontotemporal Dementia. Here is the latest research on the nuclear pore complex in ALS and FTD.

Applications of Molecular Barcoding

The concept of molecular barcoding is that each original DNA or RNA molecule is attached to a unique sequence barcode. Sequence reads having different barcodes represent different original molecules, while sequence reads having the same barcode are results of PCR duplication from one original molecule. Discover the latest research on molecular barcoding here.

Chronic Fatigue Syndrome

Chronic fatigue syndrome is a disease characterized by unexplained disabling fatigue; the pathology of which is incompletely understood. Discover the latest research on chronic fatigue syndrome here.

Evolution of Pluripotency

Pluripotency refers to the ability of a cell to develop into three primary germ cell layers of the embryo. This feed focuses on the mechanisms that underlie the evolution of pluripotency. Here is the latest research.

Position Effect Variegation

Position Effect Variagation occurs when a gene is inactivated due to its positioning near heterochromatic regions within a chromosome. Discover the latest research on Position Effect Variagation here.

STING Receptor Agonists

Stimulator of IFN genes (STING) are a group of transmembrane proteins that are involved in the induction of type I interferon that is important in the innate immune response. The stimulation of STING has been an active area of research in the treatment of cancer and infectious diseases. Here is the latest research on STING receptor agonists.

Microbicide

Microbicides are products that can be applied to vaginal or rectal mucosal surfaces with the goal of preventing, or at least significantly reducing, the transmission of sexually transmitted infections. Here is the latest research on microbicides.