Deutetrabenazine for Chorea Associated with Huntington Disease



Title: Effect of Deutetrabenazine on Chorea Among Patients With Huntington Disease

Authors: Frank, S. MD et al


 

What You Need to Know:

Compared to placebo, the use of deutetrabenazine, a vesicular monoamine transporter type 2 inhibitor structurally related to tetrabenazine, resulted in improved motor signs in patients with chorea associated with Huntington disease (HD) at 12 weeks.

Trial Design:

  • Double-blind study conducted at 34 Huntington Study Group sites
  • 90 ambulatory adults with HD; baseline total maximal chorea score: 8 or higher
  • Enrolled from August 2013 to August 2014
  • Patients received deutetrabenazine (n=45) or placebo (n=45)
  • Over 8 weeks, deutetrabenazine or placebo titrated to optimal dose; maintained for 4 weeks, then 1-week washout
  • Primary endpoint: total maximal chorea score change from baseline to maintenance therapy
  • Secondary endpoints: proportion of patients achieving treatment success based on Patient Global Impression of Change (PGIC), Clinical Global Impression of Change (CGIC), change in 36-Item Short Form-physical functioning subscale score (SF-36), change in Berg Balance Test

Key Outcomes:

  • Mean total maximal chorea score improvement: deutetrabenazine group: from 12.1 (95% CI, 11.2-12.9) to 7.7 (95% CI, 6.5-8.9); placebo group: from 13.2 (95% CI, 12.2-14.3) to 11.3 (95% CI, 10.0-12.5)
  • PGIC: deutetrabenazine group: treatment success in 23 patients (51%); placebo group: 9 patients (20%)
  • CGIC: deutetrabenazine group: treatment success in 19 patients (42%); placebo group: 6 patients (13%)
  • SF-36 scores: deutetrabenazine group: decreased from 47.5 (95% CI, 44.3-50.8) to 47.4 (44.3-50.5); placebo group: decreased from 43.2 (95% CI, 40.2-46.3) to 39.9 (95% CI, 36.2-43.6); treatment benefit of 4.3 (95% CI, 0.4 to 8.3) (P = 0.03).
  • Berg Balance Test: deutetrabenazine group: improved by 2.2 units; placebo group: improved by 1.3 units
  • Adverse events: depression, anxiety, akathisia (similar between groups)