ArticleBritish journal of clinical pharmacology2014
Bayesian adaptive designs in single ascending dose trials in healthy volunteers.
Article in British journal of clinical pharmacology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
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Who cites it
5 citing papers in PubMed.
- A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier.British journal of clinical pharmacology · 2019Trial
- Results and evaluation of a first-in-human study of RG7342, an mGlu5 positive allosteric modulator, utilizing Bayesian adaptive methods.British journal of clinical pharmacology · 2018Trial
- SPIRIT-DEFINE explanation and elaboration: recommendations for enhancing quality and impact of early phase dose-finding clinical trials protocols.EClinicalMedicine · 2025Article
- Bayesian estimation in NONMEM.CPT: pharmacometrics & systems pharmacology · 2024Article
- Model-Based Assessment of Alternative Study Designs in Pediatric Trials. Part II: Bayesian Approaches.CPT: pharmacometrics & systems pharmacology · 2016Article
Corrections and comments
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Authors and funding
10 authors.
Funding
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Abstract
aimRecent 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.
methodsA trial simulation approach was used and seven different scenarios of dose-response were tested.
resultsThe 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.
conclusionsThe 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.
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