A randomized, double-blind, placebo-controlled trial of pridopidine in Huntington's disease

Kieburtz Karl, Andrew McGarry, Michael P. McDermott, Elise Kayson, Francis Walker, Jody Goldstein, Christopher Hyson, Pinky Agarwal, Patricia Deppen, Jess Fiedorowicz, Sandra Kostyk, Allison Wright, Blair Leavitt, Martha Nance, Mark Ledoux, Kathleen M. Shannon, Andrew Siderowf, Merit Cudkowicz, Karen Rabinowitz, Victoria RossArthur Watts, Joakim Tedroff

Research output: Contribution to journalArticle

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Abstract

We examined the effects of 3 dosages of pridopidine, a dopamine-stabilizing compound, on motor function and other features of Huntington's disease, with additional evaluation of its safety and tolerability. This was a randomized, double-blind, placebo-controlled trial in outpatient neurology clinics at 27 sites in the United States and Canada. Two hundred twenty-seven subjects enrolled from October 24, 2009, to May 10, 2010. The intervention was pridopidine, either 20 (n=56), 45 (n=55), or 90 (n=58) mg daily for 12 weeks or matching placebo (n=58). The primary outcome measure was the change from baseline to week 12 in the Modified Motor Score, a subset of the Unified Huntington's Disease Rating Scale Total Motor Score. Measures of safety and tolerability included adverse events and trial completion on the assigned dosage. After 12 weeks, the treatment effect (relative to placebo, where negative values indicate improvement) of pridopidine 90 mg/day on the Modified Motor Score was -1.2 points (95% confidence interval [CI], -2.5 to 0.1 points; P = .08). The effect on the Total Motor Score was -2.8 points (95% CI, -5.4 to -0.1 points; nominal P = .04). No significant effects were seen in secondary outcome measures with any of the active dosages. Pridopidine was generally well tolerated. Although the primary analysis did not demonstrate a statistically significant treatment effect, the overall results suggest that pridopidine may improve motor function in Huntington's disease. The 90 mg/day dosage appears worthy of further study. Pridopidine was well tolerated.

Original languageEnglish (US)
Pages (from-to)1407-1415
Number of pages9
JournalMovement Disorders
Volume28
Issue number10
DOIs
StatePublished - Jan 1 2013

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Huntington Disease
Placebos
Outcome Assessment (Health Care)
Confidence Intervals
Safety
Neurology
Ambulatory Care Facilities
Canada
pridopidine
Dopamine
Therapeutics

All Science Journal Classification (ASJC) codes

  • Neurology
  • Clinical Neurology

Cite this

Karl, K., McGarry, A., McDermott, M. P., Kayson, E., Walker, F., Goldstein, J., ... Tedroff, J. (2013). A randomized, double-blind, placebo-controlled trial of pridopidine in Huntington's disease. Movement Disorders, 28(10), 1407-1415. https://doi.org/10.1002/mds.25362

A randomized, double-blind, placebo-controlled trial of pridopidine in Huntington's disease. / Karl, Kieburtz; McGarry, Andrew; McDermott, Michael P.; Kayson, Elise; Walker, Francis; Goldstein, Jody; Hyson, Christopher; Agarwal, Pinky; Deppen, Patricia; Fiedorowicz, Jess; Kostyk, Sandra; Wright, Allison; Leavitt, Blair; Nance, Martha; Ledoux, Mark; Shannon, Kathleen M.; Siderowf, Andrew; Cudkowicz, Merit; Rabinowitz, Karen; Ross, Victoria; Watts, Arthur; Tedroff, Joakim.

In: Movement Disorders, Vol. 28, No. 10, 01.01.2013, p. 1407-1415.

Research output: Contribution to journalArticle

Karl, K, McGarry, A, McDermott, MP, Kayson, E, Walker, F, Goldstein, J, Hyson, C, Agarwal, P, Deppen, P, Fiedorowicz, J, Kostyk, S, Wright, A, Leavitt, B, Nance, M, Ledoux, M, Shannon, KM, Siderowf, A, Cudkowicz, M, Rabinowitz, K, Ross, V, Watts, A & Tedroff, J 2013, 'A randomized, double-blind, placebo-controlled trial of pridopidine in Huntington's disease', Movement Disorders, vol. 28, no. 10, pp. 1407-1415. https://doi.org/10.1002/mds.25362
Karl K, McGarry A, McDermott MP, Kayson E, Walker F, Goldstein J et al. A randomized, double-blind, placebo-controlled trial of pridopidine in Huntington's disease. Movement Disorders. 2013 Jan 1;28(10):1407-1415. https://doi.org/10.1002/mds.25362
Karl, Kieburtz ; McGarry, Andrew ; McDermott, Michael P. ; Kayson, Elise ; Walker, Francis ; Goldstein, Jody ; Hyson, Christopher ; Agarwal, Pinky ; Deppen, Patricia ; Fiedorowicz, Jess ; Kostyk, Sandra ; Wright, Allison ; Leavitt, Blair ; Nance, Martha ; Ledoux, Mark ; Shannon, Kathleen M. ; Siderowf, Andrew ; Cudkowicz, Merit ; Rabinowitz, Karen ; Ross, Victoria ; Watts, Arthur ; Tedroff, Joakim. / A randomized, double-blind, placebo-controlled trial of pridopidine in Huntington's disease. In: Movement Disorders. 2013 ; Vol. 28, No. 10. pp. 1407-1415.
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