Phase 2
N=189
A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD)
Huntington's Disease
Bottom Line
View on ClinicalTrials.gov: NCT05107128 ↗Enrolled (actual)
189
Serious AEs
4.8%
Results posted
Sep 2025
Primary outcome: Primary: Change From Baseline in the SDMT — 2.0; 0.8 score on a scale — p=0.1675
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- SAGE-718 (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 25+ yrs
- Sex
- All
- Sponsor
- Supernus Pharmaceuticals, Inc.
- Primary completion
- Sep 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in the SDMT |
2.0; 0.8 | 0.1675 |
| SECONDARY Change From Baseline in the Unified Huntington's Disease Rating Scale (UHDRS) - Independence Scale |
-0.8; -1.5 | 0.4872 |
| SECONDARY Change From Baseline in the Trail Making Test Part B |
-5.7; -10.7 | 0.4089 |
| SECONDARY Change From Baseline in the One Touch Stockings of Cambridge (OTS-Mean Latency Until Correct Response) |
-4.56; -3.85 | 0.5766 |
| SECONDARY Change From Baseline in the Paced Tapping Test (PTAP) |
0.710; -0.157 | 0.1777 |
| SECONDARY Change From Baseline in the Huntington's Disease Everyday Functioning (Hi-DEF) Home Subdomain Score |
-4.6; -0.7 | 0.0291 sig |
| SECONDARY Change From Baseline in the Clinical Global Impression - Severity (CGI-S) Cognitive Status Subdomain Score |
-0.0; -0.1 | 0.2923 |
| SECONDARY Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) |
75.5; 62.1 | — |
Summary
The primary purpose of this study is to evaluate the effect of SAGE-718 on cognitive performance and functioning in participants with HD.
Eligibility Criteria
Inclusion Criteria
- Meet all the following criteria for HD at Screening (Days -28 to -2):
- Genetically confirmed disease with huntingtin gene CAG expansion greater than or equal to (≥) 36.
- At screening, Unified Huntington's Disease Rating Scale (UHDRS)-Total Functional Capacity (TFC) score greater than (>) 6 and less than (<)13, suggesting no more than a moderate level of functional impairment.
- No features of juvenile HD.
- Score of 15 to 25 (inclusive) on the Montreal Cognitive Assessment (MoCA) at screening indicating the presence of cognitive impairment.
- 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.
- Completion of Huntington's Disease Cognitive Assessment Battery (HD-CAB) Trail Making-B Test in less than 240 seconds at Screening (Days -28 to -2).
Exclusion Criteria
- Have participated in a previous clinical study of SAGE-718, have previous exposure to gene therapy, have participated in any HD investigational drug, biologic, or device trial within 180 days, or a non-HD drug, biologic, or device trial within 30 days or 5 half-lives (whichever is longer).
(Note: Participants with confirmation of enrollment in the placebo arm of these trials would not 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.
Data sourced from ClinicalTrials.gov (NCT05107128). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.