Literature DB >> 24528176

Bayesian adaptive designs in single ascending dose trials in healthy volunteers.

David Guédé1, Bruno Reigner, Francois Vandenhende, Mike Derks, Ulrich Beyer, Paul Jordan, Eric Worth, Cheikh Diack, Nicolas Frey, Richard Peck.   

Abstract

AIM: Recent publications indicate a strong interest in applying Bayesian adaptive designs in first time in humans (FTIH) studies outside of oncology. The objective of the present work was to assess the performance of a new approach that includes Bayesian adaptive design in single ascending dose (SAD) trials conducted in healthy volunteers, in comparison with a more traditional approach.
METHODS: A trial simulation approach was used and seven different scenarios of dose-response were tested.
RESULTS: The new approach provided less biased estimates of maximum tolerated dose (MTD). In all scenarios, the number of subjects needed to define a MTD was lower with the new approach than with the traditional approach. With respect to duration of the trials, the two approaches were comparable. In all scenarios, the number of subjects exposed to a dose greater than the actual MTD was lower with the new approach than with the traditional approach.
CONCLUSIONS: The new approach with Bayesian adaptive design shows a very good performance in the estimation of MTD and in reducing the total number of healthy subjects. It also reduces the number of subjects exposed to doses greater than the actual MTD.
© 2014 The British Pharmacological Society.

Entities:  

Keywords:  Bayesian adaptive design; safety; single ascending dose trial

Mesh:

Substances:

Year:  2014        PMID: 24528176      PMCID: PMC4137831          DOI: 10.1111/bcp.12344

Source DB:  PubMed          Journal:  Br J Clin Pharmacol        ISSN: 0306-5251            Impact factor:   4.335


  17 in total

1.  "Classical 3 + 3 design" versus "accelerated titration designs": analysis of 270 phase 1 trials investigating anti-cancer agents.

Authors:  Nicolas Penel; Nicolas Isambert; Pierre Leblond; Charles Ferte; Alain Duhamel; Jacques Bonneterre
Journal:  Invest New Drugs       Date:  2009-01-10       Impact factor: 3.850

2.  Bayesian decision procedures based on logistic regression models for dose-finding studies.

Authors:  J Whitehead; D Williamson
Journal:  J Biopharm Stat       Date:  1998-07       Impact factor: 1.051

3.  Defining the maximum tolerated dose: investigator, academic, industry and regulatory perspectives.

Authors:  N R Cutler; J J Sramek; D J Greenblatt; P Chaikin; N Ford; L J Lesko; B Davis; R L Williams
Journal:  J Clin Pharmacol       Date:  1997-09       Impact factor: 3.126

4.  Practical implementation of a modified continual reassessment method for dose-finding trials.

Authors:  S Piantadosi; J D Fisher; S Grossman
Journal:  Cancer Chemother Pharmacol       Date:  1998       Impact factor: 3.333

5.  Continual reassessment method: a practical design for phase 1 clinical trials in cancer.

Authors:  J O'Quigley; M Pepe; L Fisher
Journal:  Biometrics       Date:  1990-03       Impact factor: 2.571

6.  Some practical improvements in the continual reassessment method for phase I studies.

Authors:  S N Goodman; M L Zahurak; S Piantadosi
Journal:  Stat Med       Date:  1995-06-15       Impact factor: 2.373

7.  Practical modifications of the continual reassessment method for phase I cancer clinical trials.

Authors:  D Faries
Journal:  J Biopharm Stat       Date:  1994-07       Impact factor: 1.051

8.  A comparison of two phase I trial designs.

Authors:  E L Korn; D Midthune; T T Chen; L V Rubinstein; M C Christian; R M Simon
Journal:  Stat Med       Date:  1994-09-30       Impact factor: 2.373

9.  Some suggestions for measuring predictive performance.

Authors:  L B Sheiner; S L Beal
Journal:  J Pharmacokinet Biopharm       Date:  1981-08

10.  The continual reassessment method: comparison of Bayesian stopping rules for dose-ranging studies.

Authors:  S Zohar; S Chevret
Journal:  Stat Med       Date:  2001-10-15       Impact factor: 2.373

View more
  3 in total

1.  Results and evaluation of a first-in-human study of RG7342, an mGlu5 positive allosteric modulator, utilizing Bayesian adaptive methods.

Authors:  Stefan Sturm; Marie-Laure Delporte; Salah Hadi; Scott Schobel; Lothar Lindemann; Robert Weikert; Georg Jaeschke; Michael Derks; Giuseppe Palermo
Journal:  Br J Clin Pharmacol       Date:  2017-12-18       Impact factor: 4.335

2.  A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier.

Authors:  Stefan Sturm; Andreas Günther; Birgit Jaber; Paul Jordan; Nada Al Kotbi; Nikhat Parkar; Yumi Cleary; Nicolas Frances; Tobias Bergauer; Katja Heinig; Heidemarie Kletzl; Anne Marquet; Hasane Ratni; Agnès Poirier; Lutz Müller; Christian Czech; Omar Khwaja
Journal:  Br J Clin Pharmacol       Date:  2018-11-16       Impact factor: 4.335

3.  Model-Based Assessment of Alternative Study Designs in Pediatric Trials. Part II: Bayesian Approaches.

Authors:  G Smania; P Baiardi; A Ceci; M Cella; P Magni
Journal:  CPT Pharmacometrics Syst Pharmacol       Date:  2016-08-17
  3 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.