Related Experiment Video

Updated: Apr 27, 2026

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

10.1K

IPF clinical trial design and endpoints

Steven D Nathan1, Keith C Meyer

  • 1aAdvanced Lung Disease and Transplant Program, Department of Medicine, Inova Fairfax Hospital, Falls Church, Virginia bDepartment of Internal Medicine, Section of Allergy, Pulmonary and Critical Care Medicine, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin, USA.

Current Opinion in Pulmonary Medicine
|July 16, 2014
PubMed
Abstract

Insights

Developing new therapies for idiopathic pulmonary fibrosis (IPF) requires careful clinical trial design. This review examines endpoints for IPF clinical trials to improve drug development success.

Area of Science:

  • Pulmonary Medicine
  • Clinical Trial Design
  • Drug Development

Background:

  • Idiopathic pulmonary fibrosis (IPF) lacks effective therapies, necessitating novel treatment strategies.
  • Numerous IPF therapeutics are under investigation, highlighting the urgency for robust clinical trials.
  • Past clinical trial outcomes, though often disappointing, have informed current research and endpoint selection.

Purpose of the Study:

  • To review and discuss clinical trial endpoints for idiopathic pulmonary fibrosis (IPF).
  • To evaluate the utility and potential pitfalls of various proposed and utilized endpoints in IPF studies.
  • To guide investigators in selecting optimal endpoints for prospective IPF clinical trials.

Main Methods:

  • Literature review of published and proposed clinical trial endpoints for IPF.
  • Analysis of endpoint utility based on their application in past and ongoing IPF trials.
  • Discussion of methodological considerations and challenges in endpoint selection for IPF research.

Main Results:

  • Various endpoints have been employed in IPF trials, including forced vital capacity (FVC) and composite endpoints.
  • The selection of endpoints significantly impacts the interpretation of drug efficacy and trial outcomes.
  • Insights from previous trials underscore the need for validated and sensitive endpoints in IPF research.

Conclusions:

  • Careful selection of appropriate clinical trial endpoints is crucial for demonstrating the efficacy of novel IPF therapies.
  • Understanding the strengths and limitations of different endpoints can optimize trial design and accelerate drug development.
  • This review provides a framework for considering endpoint utility and potential pitfalls in IPF clinical trial design.

More Related Videos

Author Spotlight: Methodologies and Advancements of Chronic Pain Management Research08:33

Author Spotlight: Methodologies and Advancements of Chronic Pain Management Research

Published on: January 5, 2024

2.0K
Author Spotlight: Exercise Test for Evaluation of the Functional Efficacy of the Pig Cardiovascular System02:47

Author Spotlight: Exercise Test for Evaluation of the Functional Efficacy of the Pig Cardiovascular System

Published on: May 12, 2023

2.2K

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...
8.5K
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...
4.7K
Bioavailability Study Design: Healthy Subjects Versus Patients01:15

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,...
241
Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions01:15

Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions

PK–PD modeling has significantly influenced FDA regulatory decisions, particularly drug approval, dosage optimization, and labeling. These models integrate pharmacokinetics (PK) and pharmacodynamics (PD) to predict drug behavior and effects, aiding in optimizing dosing regimens and enhancing the probability of clinical trial success.One notable example is Nesiritide (Natrecor®), a recombinant human brain natriuretic peptide for treating acute decompensated congestive heart failure...
97
Preclinical Development: Overview01:28

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...
4.7K
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,...
627