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Neurodegenerative disease

Establishing a clinical trial battery for Huntington disease

Nature Reviews Neurology volume 8, pages 250–251 (2012)Cite this article

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The success of clinical trials in Huntington disease (HD) will depend to a large degree on the quality of the outcome measures. Using data from the TRACK-HD study, a recent publication proposes a battery of assessments that could be used as outcomes in future clinical trials in patients with early HD.

A limitation to the care of Huntington disease (HD) is a dearth of proven treatment options. At least 16 distinct compounds have been tested through human clinical trials,1,2 and a few animal models of stem cell replacement3 have also been generated. Several refined and new compounds, as well as efforts to reduce the expression of the mutant huntingtin gene, are now being incorporated into the design of clinical trials. The success of all efforts to improve the care of people with HD depends on the development and validation of objective, quantifiable and feasible outcome measures.4

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Figure 1: HD progression groups located on a hypothetical spectrum.

References

  1. Paulsen, J. Cognitive impairment in Huntington disease: diagnosis and treatment. Curr. Neurol. Neurosci. Rep. 11, 474–483 (2011).

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  2. Ross, C. A. & Tabrizi, S. J. Huntington's disease: from molecular pathogenesis to clinical treatment. Lancet Neurol. 10, 83–98 (2011).

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Acknowledgements

We would like to thank Jennifer Rapp, editorial associate, for her assistance with this article.

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Affiliations

  1. The University of Iowa Roy J. and Lucille A. Carver College of Medicine, Research, 1-305, Medical Education Building, 500 Newton Road, Iowa City, 52242-1000, IA, USA

    Jane S. Paulsen

  2. The University of Iowa Roy J. and Lucille A. Carver College of Medicine, Research, 1-328, Medical Education Building, 500 Newton Road, Iowa City, 52242-1000, IA, USA

    Jeffrey D. Long

Authors
  1. Jane S. Paulsen
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  2. Jeffrey D. Long
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Correspondence to Jeffrey D. Long.

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The authors declare no competing financial interests.

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Cite this article

Paulsen, J., Long, J. Establishing a clinical trial battery for Huntington disease. Nat Rev Neurol 8, 250–251 (2012). https://doi.org/10.1038/nrneurol.2012.67

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  • DOI: https://doi.org/10.1038/nrneurol.2012.67

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