Challenges assessing clinical endpoints in early Huntington disease

The PREDICT-HD Investigators of the Huntington Study Group

Research output: Contribution to journalArticle

44 Citations (Scopus)

Abstract

The basic aim of this study was to evaluate the current accepted standard clinical endpoint for the earliest-studied HD participants likely to be recruited into clinical trials. As the advent of genetic testing for HD, it is possible to identify gene carriers before the diagnosis of disease, which opens up the possibility of clinical trials of disease-modifying treatments in clinically asymptomatic persons. Current accepted standard clinical endpoints were examined as part of a multinational, 32-site, longitudinal, observational study of 786 research participants currently in the HD prodrome (gene-positive but not clinically diagnosed). Clinical signs and symptoms were used to prospectively predict functional loss as assessed by current accepted standard endpoints over 8 years of follow-up. Functional capacity measures were not sensitive for HD in the prodrome; over 88% scored at ceiling. Prospective evaluation revealed that the first functional loss was in their accustomed work. In a survival analysis, motor, cognitive, and psychiatric measures were all predictors of job change. To our knowledge, this is the first prospective study ever conducted on the emergence of functional loss secondary to brain disease. We conclude that future clinical trials designed for very early disease will require the development of new and more sensitive measures of real-life function.

Original languageEnglish (US)
Pages (from-to)2595-2603
Number of pages9
JournalMovement Disorders
Volume25
Issue number15
DOIs
StatePublished - Nov 1 2010

Fingerprint

Huntington Disease
Clinical Trials
Genetic Testing
Brain Diseases
Survival Analysis
Genes
Signs and Symptoms
Observational Studies
Psychiatry
Longitudinal Studies
Prospective Studies
Research

Keywords

  • Clinical endpoints
  • Functional capacity
  • Huntington disease
  • Prodromal HD
  • UHDRS

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

Cite this

The PREDICT-HD Investigators of the Huntington Study Group (2010). Challenges assessing clinical endpoints in early Huntington disease. Movement Disorders, 25(15), 2595-2603. https://doi.org/10.1002/mds.23337

Challenges assessing clinical endpoints in early Huntington disease. / The PREDICT-HD Investigators of the Huntington Study Group.

In: Movement Disorders, Vol. 25, No. 15, 01.11.2010, p. 2595-2603.

Research output: Contribution to journalArticle

The PREDICT-HD Investigators of the Huntington Study Group 2010, 'Challenges assessing clinical endpoints in early Huntington disease', Movement Disorders, vol. 25, no. 15, pp. 2595-2603. https://doi.org/10.1002/mds.23337
The PREDICT-HD Investigators of the Huntington Study Group. Challenges assessing clinical endpoints in early Huntington disease. Movement Disorders. 2010 Nov 1;25(15):2595-2603. https://doi.org/10.1002/mds.23337
The PREDICT-HD Investigators of the Huntington Study Group. / Challenges assessing clinical endpoints in early Huntington disease. In: Movement Disorders. 2010 ; Vol. 25, No. 15. pp. 2595-2603.
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