Caudate volume as an outcome measure in clinical trials for Huntington's disease: a pilot study

E H Aylward1, A Rosenblatt, K Field

  • 1Department of Radiology, University of Washington, Box 357115, Seattle, WA 98195, USA. eaylward@u.washington.edu

Brain Research Bulletin
|November 26, 2003
PubMed

Insights

Caudate volume decrease is a significant indicator in Huntington's disease (HD) progression. Measuring this change with MRI is a feasible outcome for clinical trials, potentially requiring fewer participants.

Area of Science:

  • Neuroimaging
  • Clinical Trials
  • Neurology

Background:

  • Longitudinal changes in caudate volume are observable in Huntington's disease (HD).
  • Assessing caudate volume change via MRI is crucial for evaluating treatment efficacy in HD clinical trials.

Purpose of the Study:

  • To determine the feasibility and validity of caudate volume measurement using MRI as an outcome measure in a clinical trial setting for Huntington's disease.
  • To evaluate the significance of caudate volume change over time in HD patients undergoing treatment.

Main Methods:

  • Pilot study involving 19 symptomatic Huntington's disease patients across two clinical trial sites.
  • Measurement of caudate volumes using magnetic resonance imaging (MRI) on pre- and post-treatment scans.
  • Analysis of data from the CARE-HD (Co-enzyme Q10 and Remacemide: Evaluation in Huntington's Disease) 30-month clinical trial.

Main Results:

  • A statistically significant decrease in caudate volume was observed over the study period.
  • Power analysis suggests that a relatively small number of subjects are needed for trials utilizing caudate volume as an outcome measure.
  • Identified advantages and disadvantages of employing MRI-based caudate volume measurement in clinical trials.

Conclusions:

  • Quantitative neuroimaging of caudate volume is a feasible and valid outcome measure for Huntington's disease clinical trials.
  • Recommends the adoption of MRI caudate volume assessment in future HD treatment studies.
  • Highlights the potential for efficient clinical trial design with this neuroimaging biomarker.

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...
Huntington Disease l: Introduction01:21

Huntington Disease l: Introduction

Huntington disease or HD is a progressive, fatal neurodegenerative disorder inherited in an autosomal dominant pattern.PathophysiologyIt is caused by expansion of the CAG trinucleotide repeat in the HTT gene on chromosome 4 (4p16.3), producing an abnormal huntingtin protein with an expanded polyglutamine tract. This misfolded protein disrupts cellular function, leading to neuronal death. Normal alleles have ≤26 repeats, 27–35 are intermediate (risk of expansion), 36–39 show reduced penetrance,...