Phase 2
N=69
28-Day Study of SAGE-718 on Functioning Capacity in Participants With Huntington's Disease
Huntington Disease
Bottom Line
View on ClinicalTrials.gov: NCT05358821 ↗Enrolled (actual)
69
Serious AEs
0.0%
Results posted
May 2025
Primary outcome: Primary: Difference in Huntington's Disease Cognitive Assessment Battery (HD-CAB) Composite Score Between Participants With HD vs HP at Baseline — -3.3657; 0.0000 Z score — p=<0.0001
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
- Feb 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Difference in Huntington's Disease Cognitive Assessment Battery (HD-CAB) Composite Score Between Participants With HD vs HP at Baseline |
-3.3657; 0.0000 | <0.0001 sig |
| SECONDARY Number of Participants With HD With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs |
6; 11; 0; 0 | — |
| SECONDARY Number of Participants With HD With Clinically Significant Change From Baseline in Vital Sign Measurements |
0; 0 | — |
| SECONDARY Number of Participants With HD With Clinically Significant Change From Baseline in Clinical Laboratory Assessments |
0; 0 | — |
Summary
The primary purpose of this study is to assess the magnitude of the baseline difference between participants with early Huntington's Disease (HD) and healthy participants (HP) with respect to measures of cognitive performance.
Eligibility Criteria
Inclusion Criteria
For all
- Agree to refrain from drugs of abuse for the duration of the study and from alcohol during the 48 hours preceding each study visit.
Additional criteria for participants with HD only:
- Be ambulatory, able to travel to the study center, and, 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.
- Have:
- Genetically confirmed disease with cytosine, adenine, and guanine (CAG) expansion ≥36.
- At screening, Unified Huntington's Disease Rating Scale (UHDRS) Total Functional Capacity (TFC) >6 and 70, as calculated using the CAP formula: Age × (CAG - 30) / 6.49.
- Score of 15 to 25 (inclusive) on the Montreal Cognitive Assessment (MoCA) at screening, indicating the presence of cognitive impairment.
Additional criteria for HP only:
- Score ≥26 on the MoCA at screening.
- Have no known family history of HD; or, have known family history of HD but have genetic test results available that show a normal CAG repeat length for both Huntingtin (HTT) alleles (<36).
Exclusion Criteria
For All
- Receive any prohibited medications within 30 days of Screening and during participation in the study
- Have participated in a previous clinical study of SAGE-718, have previous exposure to gene therapy; or 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 enrolment in the placebo arm of these trials would not be excluded.)
- Is known to be allergic to any of SAGE-718 excipients, including soy lecithin.
Additional criteria for participants with HD only:
- Had gastric bypass surgery, has a gastric sleeve or lap band, or has had any related procedures that interfere with gastrointestinal transit.
- Receive any prohibited medications within 30 days of Screening and during participation in the study.
Data sourced from ClinicalTrials.gov (NCT05358821). 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.