Related Experiment Video

Updated: Feb 12, 2026

In Silico Clinical Trials for Cardiovascular Disease
09:09

In Silico Clinical Trials for Cardiovascular Disease

Published on: May 27, 2022

2.3K

Design and Endpoints for Clinical Trials in Primary Sclerosing Cholangitis

Cyriel Y Ponsioen1, Keith D Lindor2, Ruby Mehta3

  • 1Department of Gastroenterology & Hepatology, Academic Medical Center, Amsterdam, The Netherlands.

Hepatology (Baltimore, Md.)
|March 26, 2018
PubMed

Insights

Primary sclerosing cholangitis (PSC) is a rare liver disease lacking effective treatments. This review examines endpoints for clinical trials to better measure treatment success in PSC patients.

Area of Science:

  • Hepatology
  • Gastroenterology
  • Clinical Trial Design

Background:

  • Primary sclerosing cholangitis (PSC) is a rare, chronic cholestatic liver disease.
  • Currently, no approved effective therapies exist for PSC.
  • There is a critical need for standardized outcome measures in PSC clinical trials.

Purpose of the Study:

  • To review regulatory perspectives on clinical trial endpoints for PSC.
  • To evaluate the natural history data relevant to endpoint selection.
  • To identify and discuss potential clinical and surrogate endpoints for evaluating therapeutic interventions in PSC.

Main Methods:

  • Comprehensive literature search of PubMed and regulatory agency websites.
  • Review of published natural history studies of PSC.
  • Analysis of proposed endpoints in existing and ongoing PSC clinical trials.

Main Results:

  • Regulatory guidance emphasizes meaningful clinical benefit but lacks specific PSC endpoints.
  • Natural history data show significant variability, complicating endpoint validation.
  • Several candidate endpoints, including liver biochemistry, imaging, and patient-reported outcomes, are discussed.

Conclusions:

  • Standardized and validated endpoints are crucial for advancing PSC therapeutic development.
  • A combination of clinical and surrogate endpoints may be necessary to capture treatment effects.
  • Further research and consensus are needed to establish optimal endpoints for PSC trials.

More Related Videos

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides05:16

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides

Published on: May 7, 2020

7.3K
Enumeration of Major Peripheral Blood Leukocyte Populations for Multicenter Clinical Trials Using a Whole Blood Phenotyping Assay14:45

Enumeration of Major Peripheral Blood Leukocyte Populations for Multicenter Clinical Trials Using a Whole Blood Phenotyping Assay

Published on: September 16, 2012

15.5K

Related Concept Videos

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...
10.9K
Clinical Trials: Overview01:11

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...
5.0K
Statistical Software for Data Analysis and Clinical Trials01:12

Statistical Software for Data Analysis and Clinical Trials

Statistical software is pivotal in data analysis and clinical trials by providing tools to analyze data, draw conclusions, and make predictions. These software packages range from simple data management applications to complex analytical platforms, supporting various statistical tests, models, and simulation techniques. Their significance lies in their ability to handle vast amounts of data with precision and efficiency, enabling researchers to validate hypotheses, identify trends, and make...
1.6K
Precipitation Titration: Endpoint Detection Methods01:19

Precipitation Titration: Endpoint Detection Methods

In argentometric precipitation titrations, endpoints can be detected visually by the Mohr, Volhard, and Fajans methods. In the Mohr method, adding a soluble chromate indicator gives an initial yellow color to the analyte solution. As the titrant is added, the first excess of silver ions forms a red silver chromate precipitate, marking the endpoint. The solution pH should be maintained at about 8 by adding solid CaCO3.
In the Volhard method, a standard excess of AgNO3 is first added to the...
6.0K
Trial and Error and Algorithm01:12

Trial and Error and Algorithm

A problem-solving strategy is a plan of action used to find a solution. Different strategies have distinct action plans. Trial and error involves trying different solutions until one works. For instance, to fix a broken printer, you might check ink levels, ensure the paper tray isn't jammed, and verify the printer's connection to your laptop. This method can be time-consuming but is commonly used. Thomas Edison, for example, used trial and error to find a suitable filament for the light...
429
Group Design02:01

Group Design

The most basic experimental design involves two groups: the experimental group and the control group. The two groups are designed to be the same except for one difference— experimental manipulation. The experimental group gets the experimental manipulation—that is, the treatment or variable being tested—and the control group does not. Since experimental manipulation is the only difference between the experimental and control groups, we can be sure that any differences between...
10.7K