A randomized, placebo-controlled trial of latrepirdine in Huntington disease

Topic: Brain proteins, microglia and lab models

Objectives To evaluate the safety and tolerability of latrepirdine in Huntington disease (HD) and explore its effects on cognition, behavior, and motor symptoms. Design Double-blind, randomized, placebo-controlled trial. Setting Multicenter outpatient trial. Participants Ninety-one participants with mild to moderate HD enrolled at 17 US and UK centers from July 18, 2007, through July 16, 2008. Intervention Latrepirdine, 20 mg 3 times daily (n = 46), or matching placebo (n = 45) for a 90-day treatment period. Main outcome measures The primary outcome variable was tolerability, defined as the ability to complete the study at the assigned drug dosage. Secondary outcome variables included score changes from baseline to day 90 on the Unified Huntington's Disease Rating Scale (UHDRS), the Mini-Mental State Examination (MMSE), and the Alzheimer Disease Assessment Scale-cognitive subscale (ADAS-cog). Results Latrepirdine was well tolerated (87% of the patients given latrepirdine completed the study vs 82% in the placebo group), and adverse event rates were comparable in the 2 groups (70% in the latrepirdine group and 80% in the placebo group). Treatment with latrepirdine resulted in improved mean MMSE scores compared with stable performance in the placebo group (treatment effect, 0.97 points; 95% confidence interval, 0.10-1.85; P = .03). No significant treatment effects were seen on the UHDRS or the ADAS-cog. Conclusions Short-term administration of latrepirdine is well tolerated in patients with HD and may have a beneficial effect on cognition. Further investigation of latrepirdine is warranted in this population with HD.

Key findings

• Results Latrepirdine was well tolerated (87% of the patients given latrepirdine completed the study vs 82% in the placebo group), and adverse event rates were comparable in the 2 groups (70% in the latrepirdine group and 80% in the placebo group).
• Treatment with latrepirdine resulted in improved mean MMSE scores compared with stable performance in the placebo group (treatment effect, 0.97 points; 95% confidence interval, 0.10-1.85; P = .03).
• No significant treatment effects were seen on the UHDRS or the ADAS-cog.

Why it matters

In this 90-day trial in people with Huntington disease, latrepirdine was well tolerated and improved mean MMSE scores compared with placebo, but no significant treatment effects were seen on the other measures. It did not test whether the drug treats, cures, or prevents Alzheimer's disease.

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Study details

• Participants46
• Evidence strengthHigh

Read the original

• DOI: 10.1001/archneurol.2009.334
• PubMed: 20142523
• Original source →
• Free full text: PubMed Central →

Full text: free to read — PubMed Central lists this copy as free to read: the link above (checked 10 Oct 2026).

@article{kieburtz2010-20142523,
  author  = {Kieburtz, K. and McDermott, M. P. and Voss, T. S. and Corey-Bloom, J. and Deuel, L. M. and Dorsey, E. R. and Factor, S. and Geschwind, M. D. and others},
  title   = {{A randomized, placebo-controlled trial of latrepirdine in Huntington disease}},
  journal = {Archives of neurology},
  year    = {2010},
  doi     = {10.1001/archneurol.2009.334},
  pmid    = {20142523},
  url     = {https://doi.org/10.1001/archneurol.2009.334}
}

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• A new rating scale for Alzheimer's disease American Journal of Psychiatry · 1984-11-01 · usefulness score 54/100 · Cognitive tests and assessment

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