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Clinical Trials

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...

Clinical Trials: Overview

Clinical Trials: Overview

Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...

Bioavailability Study Design: Healthy Subjects Versus Patients

Bioavailability Study Design: Healthy Subjects Versus Patients

Bioavailability studies are essential for evaluating a drug's therapeutic efficacy and understanding its absorption patterns under various physiological conditions. Conducting such studies on target patient populations provides more relevant data by simulating real-world disease states. However, practical challenges often necessitate the use of young, healthy adult volunteers as study subjects.Patients may exhibit altered drug absorption patterns due to the effects of the disease itself,...

Bioavailability Study Design: Single Versus Multiple Dose Studies

Bioavailability Study Design: Single Versus Multiple Dose Studies

Bioavailability studies are essential for understanding how a drug is absorbed, distributed, metabolized, and excreted in the body. These studies assess the extent and rate at which the active pharmaceutical agent becomes available at the site of action. The design of bioavailability studies can involve single-dose or multiple-dose regimens, each with distinct advantages and limitations.Single-dose studies are the preferred approach due to their simplicity and reduced drug exposure for...

Preclinical Development: Overview

Preclinical Development: Overview

Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...

Bioequivalence Experimental Study Designs: Repeated Measures, Cross-Over, Carry-Over, and Latin Square Designs

Bioequivalence Experimental Study Designs: Repeated Measures, Cross-Over, Carry-Over, and Latin Square Designs

Bioequivalence experimental study designs play a pivotal role in testing the effectiveness of various treatments. Key among these are the repeated measures, cross-over, carry-over, and Latin square designs. In the repeated measures design, each subject receives all treatments, allowing for temporal comparisons. This type of design is useful in reducing variability but requires careful planning to avoid bias.The cross-over design, an economical method, involves sequential administration of...

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Related Experiment Video

Updated: May 9, 2026

A Clinical Trial Assessing the Safety, Efficacy, and Delivery of Olive-Oil-Based Three-Chamber Bags for Parenteral Nutrition

A Clinical Trial Assessing the Safety, Efficacy, and Delivery of Olive-Oil-Based Three-Chamber Bags for Parenteral Nutrition

Published on: September 20, 2019

Clinical Trial Simulation to Inform Phase 2: Comparison of Concentrated vs. Distributed First-in-Patient Study

M G Dodds¹, D H Salinger, J Mandema

¹Department of Pharmacokinetics & Drug Metabolism, Amgen, Seattle, Washington, USA.

CPT: Pharmacometrics & Systems Pharmacology

|July 26, 2013

Summary

This summary is machine-generated.

Clinical trial simulation and model-based meta-analysis informed early drug development decisions. Comparing trial designs for psoriasis, placebo-maximum dose studies were slightly better for advancement decisions than dose-ranging studies.

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Area of Science:

Pharmacometrics and Systems Pharmacology
Drug Development
Clinical Trial Design

Background:

Clinical trial simulation (CTS) and model-based meta-analysis (MBMA) are valuable tools for understanding early-phase clinical trial performance.
Informing Phase 2 decision-making requires robust evaluation of first-in-patient (FIP) trial designs.

Purpose of the Study:

To compare the performance of dose-ranging designs versus placebo-maximum dose designs for early decision-making in psoriasis drug development.
To evaluate the utility of CTS and MBMA in optimizing FIP trial strategies.

Main Methods:

Model-based meta-analysis (MBMA) of monoclonal antibodies in psoriasis was used to establish an advancement threshold.
Clinical trial simulations (CTS) compared dose-ranging designs against placebo-maximum dose designs for psoriasis FIP trials.

Main Results:

A >50 percentage point improvement over placebo at the maximum dose was identified as the advancement threshold for psoriasis treatments.
Placebo-maximum dose designs made the correct advancement decision more often than dose-ranging designs in most simulated scenarios.
Dose-ranging studies provided crucial dose-response (D-R) information essential for Phase 2 dose selection.

Conclusions:

While placebo-maximum dose designs offered marginal advantages in early advancement decisions for psoriasis, dose-ranging studies are critical for informing Phase 2 dose selection.
Clinical trial simulation enhances drug development efficiency and decision quality by prospectively assessing study design benefits and limitations.