Open Actively Recruiting

A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD)


Brief Summary

The primary purpose of this study is to evaluate the effect of SAGE-718 on cognitive performance and functioning in participants with HD.

Primary Purpose
Study Type
Phase 2


Healthy Volunteers
Minimum Age
25 Years
Maximum Age
65 Years

Inclusion Criteria:

  • Meet all the following criteria for HD:
    • Genetically confirmed disease with huntingtin gene CAG expansion ≥36.
    • UHDRS-Total Functional Capacity (TFC) score >6 and <13.
    • No features of juvenile HD.
  • Score <26 on the Montreal Cognitive Assessment (MoCA) at screening.
  • Be willing to invite a study partner, if available, who is reliable, competent, and at least 18 years of age to participate in the study.
  • Be ambulatory (use of assistance devices such as a walker or cane is acceptable, as is occasional use of wheelchair, as judged by the investigator. Individuals requiring a wheelchair on a regular basis are excluded), able to travel to the study center, and, as judged by the investigator, is likely to be able to continue to travel to the study center to complete study visits for the duration of the study.

Exclusion Criteria:

  • Have participated in a previous clinical study of SAGE-718, have participated in a previous gene therapy study, or have participated in any other drug, biologic, or device trial within 30 days or 5 half-lives (whichever is longer), unless the patient participated solely in the placebo arm of the study. Additionally, participants who have received treatment with antisense oligonucleotides (ASOs) or an mRNA splicing modifier will be excluded.
  • Have a diagnosis of an ongoing neurodegenerative condition other than HD, including but not limited to, Alzheimer's Disease, vascular dementia, dementia with Lewy bodies, or Parkinson's Disease.

Join this Trial

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Study Stats
Protocol No.
Brain/Neurological Diseases
Genetic and Rare Diseases
Principal Investigator
Aaron Fisher
  • UCLA Westwood
For Providers
For detailed technical eligibility, visit