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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...
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...
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...
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...
Drug Administration and Therapy Phases: Overview01:26

Drug Administration and Therapy Phases: Overview

Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
Measurement of Bioavailability: Pharmacodynamic Methods01:20

Measurement of Bioavailability: Pharmacodynamic Methods

Pharmacodynamic methods provide insights into a drug's effects on physiological processes over time and play a crucial role in understanding bioavailability and therapeutic efficacy. These methods can be broadly classified into acute pharmacological and therapeutic response approaches, each with distinct mechanisms and applications.The acute pharmacological response method directly correlates a drug's physiological effects, such as ECG or pupil diameter changes, to its time course in the body.

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Related Experiment Video

Updated: Jul 11, 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

Outcome measures for early phase clinical trials.

Paul H Gordon1, Bin Cheng, Jacqueline Montes

  • 1Department of Neurology, The Eleanor and Lou Gehrig MDA/ALS Research Center, College of Physicians and Surgeons, Mailman School of Public Health, Columbia University, New York, NY 10032, USA. phg8@columbia.edu

Amyotrophic Lateral Sclerosis : Official Publication of the World Federation of Neurology Research Group on Motor Neuron Diseases
|September 14, 2007
PubMed
Summary

For amyotrophic lateral sclerosis (ALS) clinical trials, the ALS Functional Rating Scale-Revised (ALSFRS-R) is a reliable outcome measure. It provides complete data, predicts survival, and can be used in short trials, though patient-perceived changes may require significant ALSFRS-R shifts.

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A Computerized Test Battery to Study Pharmacodynamic Effects on the Central Nervous System of Cholinergic Drugs in Early Phase Drug Development
07:02

A Computerized Test Battery to Study Pharmacodynamic Effects on the Central Nervous System of Cholinergic Drugs in Early Phase Drug Development

Published on: February 11, 2019

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Last Updated: Jul 11, 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

A Computerized Test Battery to Study Pharmacodynamic Effects on the Central Nervous System of Cholinergic Drugs in Early Phase Drug Development
07:02

A Computerized Test Battery to Study Pharmacodynamic Effects on the Central Nervous System of Cholinergic Drugs in Early Phase Drug Development

Published on: February 11, 2019

Area of Science:

  • Neurology
  • Clinical Trials
  • Drug Development

Background:

  • Early phase clinical trials are crucial for screening neuroprotective agents in amyotrophic lateral sclerosis (ALS).
  • Identifying optimal outcome measures for brief, early-phase ALS trials is essential for efficient drug development.
  • Patient perception of treatment efficacy is a key consideration in outcome measure selection.

Purpose of the Study:

  • To assess the performance of various outcome measures in early-phase ALS clinical trials.
  • To determine the degree of change in the ALSFRS-R that patients can perceive.
  • To evaluate the utility of different measures in short-duration ALS studies.

Main Methods:

  • Thirty ALS patients completed six monthly assessments: ALS Functional Rating Scale-Revised (ALSFRS-R), forced vital capacity, manual muscle testing (MMT), and quality of life.
  • Patients reported perceived changes using algorithm scales.
  • Statistical analyses included linear mixed effects models and Cox proportional-hazards models.

Main Results:

  • The ALSFRS-R demonstrated the most complete data (99.5%) and highest within-subject correlation (0.91).
  • ALSFRS-R changes significantly predicted survival (p = 0.002).
  • A one-unit change in patient-perceived function corresponded to a 9-point decrease in ALSFRS-R (p = 0.025).

Conclusions:

  • Standard outcome measures, including ALSFRS-R, are dependable for short-duration, early-phase ALS trials.
  • The ALSFRS-R is the most robust measure for predicting survival and data completeness in ALS studies.
  • Significant changes in ALSFRS-R may be needed before patients perceive a treatment effect.