Related Experiment Video
Updated: Aug 7, 2025
04:53
A Clinical Trial Assessing the Safety, Efficacy, and Delivery of Olive-Oil-Based Three-Chamber Bags for Parenteral Nutrition
Published on: September 20, 2019
10.7K
Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints.
Elias Laurin Meyer1, Peter Mesenbrink2, Nicholas A Di Prospero3
1Center for Medical Data Science, Medical University of Vienna, Vienna, Austria.
Plos One
|March 9, 2023
Summary
Platform trials accelerate drug development for non-alcoholic steatohepatitis (NASH), a progressive liver disease. EU-PEARL explored platform trial designs, decision rules, and simulations, including correlated binary endpoints, to address this unmet medical need.
Area of Science:
- Hepatology
- Clinical Trial Design
- Pharmaceutical Development
Background:
- Non-alcoholic steatohepatitis (NASH) is a progressive liver disease with significant unmet medical needs.
- Nonalcoholic fatty liver disease (NAFLD) encompasses a spectrum of liver conditions.
- Platform trials offer advantages for drug development and participant engagement.
Purpose of the Study:
- To describe the EU-PEARL consortium's activities on platform trials for NASH.
- To present a proposed platform trial design, decision rules, and simulation results for NASH.
- To discuss considerations for simulating correlated binary endpoints in clinical trials.
Main Methods:
- Simulation study based on a set of assumptions for NASH platform trials.
- Discussion of trial design learnings with health authorities.
- Exploration of options for simulating correlated binary endpoints.
Main Results:
- Simulation results for a proposed NASH platform trial design were generated.
- Learnings from health authority interactions regarding trial design were identified.
- Practical considerations for simulating co-primary binary endpoints were discussed.
Conclusions:
- Platform trials represent a promising approach for accelerating NASH drug development.
- The proposed EU-PEARL platform trial design and simulation provide valuable insights.
- Addressing challenges in endpoint simulation is crucial for efficient clinical trials in NASH.
Related Concept Videos
01:11Clinical Trials: Overview
3.1K
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...
3.1K
01:16Clinical Trials
6.9K
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...
There are four phases in a clinical trial. A phase one...
6.9K
01:23Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
153
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,...
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
153
01:29Chronic Pancreatitis II: Collaborative Care
122
The management of chronic pancreatitis is multifaceted, involving a comprehensive approach that includes thorough assessment, diagnostic testing, and a variety of management strategies.
Assessment:
Assessment:
122
01:20Study Designs in Epidemiology
315
Epidemiological study designs are fundamental tools for investigating the distribution, determinants, and control of health conditions in populations. They help researchers understand the relationships between exposures and outcomes, and they broadly fall into two categories: "observational" and "experimental" studies.
Observational studies are those where the researcher does not intervene but rather observes natural variations. They include cross-sectional, cohort, and...
Observational studies are those where the researcher does not intervene but rather observes natural variations. They include cross-sectional, cohort, and...
315
01:28Preclinical Development: Overview
4.5K
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...
4.5K