Related Experiment Video

Updated: Aug 14, 2026

Quantitative Immunofluorescence to Measure Global Localized Translation
09:13

Quantitative Immunofluorescence to Measure Global Localized Translation

Published on: August 22, 2017

Translational clinical trials: an entropy-based approach to sample size

Steven Piantadosi1

  • 1Oncology Biostatistics, Johns Hopkins School of Medicine, Baltimore, MD 21205, USA.

Clinical Trials (London, England)
|November 11, 2005
PubMed

Insights

Translational clinical trials are small studies essential for early therapeutic evidence. This paper introduces an information-based approach to understand their statistical properties and determine appropriate sample sizes.

Area of Science:

  • Biomedical research
  • Clinical pharmacology
  • Experimental therapeutics

Background:

  • Translational clinical trials are small, early-stage studies of novel therapies.
  • These trials are critical for assessing treatment effects on disease targets and informing future research.
  • The unique statistical properties of translational trials are often overlooked within traditional clinical trial frameworks.

Purpose of the Study:

  • To discuss the translational clinical trial setting.
  • To present an information (entropy)-based approach for understanding translational trial properties and applications.
  • To propose a method for motivating sample size in translational trials.

Main Methods:

  • Review of the translational clinical trial setting.
  • Application of an information (entropy)-based framework.
  • Development of a sample size motivation approach.

Main Results:

  • Identification of translational trials as crucial for early therapeutic evaluation.
  • Presentation of an entropy-based method to analyze translational trial characteristics.
  • Proposal of a novel approach for determining sample size in early-phase trials.

Conclusions:

  • Translational trials, combining biological insights with experimental design, effectively reduce uncertainty for emerging therapies.
  • An information-based approach offers valuable insights into the statistical properties and utility of translational trials.
  • A structured method for sample size determination is presented to enhance the design and interpretation of these vital studies.

Related Concept Videos

Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches01:23

Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches

Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast, controlled...
Improving Translational Accuracy02:07

Improving Translational Accuracy

Base complementarity between the three base pairs of mRNA codon and the tRNA anticodon is not a failsafe mechanism. Inaccuracies can range from a single mismatch to no correct base pairing at all. The free energy difference between the correct and nearly correct base pairs can be as small as 3 kcal/ mol. With complementarity being the only proofreading step, the estimated error frequency would be one wrong amino acid in every 100 amino acids incorporated. However, error frequencies observed in...
Improving Translational Accuracy02:07

Improving Translational Accuracy

Base complementarity between the three base pairs of mRNA codon and the tRNA anticodon is not a failsafe mechanism. Inaccuracies can range from a single mismatch to no correct base pairing at all. The free energy difference between the correct and nearly correct base pairs can be as small as 3 kcal/ mol. With complementarity being the only proofreading step, the estimated error frequency would be one wrong amino acid in every 100 amino acids incorporated. However, error frequencies observed in...
Clinical Trials01:16

Clinical Trials

Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
Bioequivalence Data: Statistical Interpretation01:16

Bioequivalence Data: Statistical Interpretation

The statistical interpretation of bioequivalence data is a significant aspect of pharmaceutical research. Bioequivalence refers to the absence of any significant difference in the rate and extent to which the active ingredient in pharmaceutical products becomes available at the site of drug action when administered at the same molar dose under similar conditions. This helps determine if different drug products have similar absorption rates, ensuring their interchangeability.Statistical...
Contaminants and Errors01:16

Contaminants and Errors

Effective sample preparation is crucial for accurate and reliable laboratory analysis. During this process, two significant sources of error can arise: concentration bias from improper sample splitting and contamination caused by methods used to reduce particle size, such as grinding or homogenization. Identifying and minimizing these potential errors is crucial to ensuring the validity of the analysis.
Another key consideration is determining the appropriate number of samples required to...