A Study to Evaluate the Effect of SAGE-718 on Functioning Capacity in Participants With Huntington's Disease

• Status: Not yet recruiting
• Trial end date: 2024-09-01
• Target enrollment: 0
• Participant gender: All

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 and the effect of SAGE-718 on cognitive performance and functioning in participants with HD.

• Phase: Phase 2
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Sage Therapeutics

Criteria

Inclusion Criteria:

For all

1. 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

2. Have:

1. Genetically confirmed disease with cytosine, adenine, and guanine (CAG) expansion ≥36

2. Unified Huntington's Disease Rating Scale (UHDRS) Total Functional Capacity (TFC) >6 and <13, suggesting no more than a moderate level of functional impairment.

3. No features of juvenile HD [ie, Unified Huntington's Disease Rating Scale(UHDRS) Diagnostic Confidence Level (DCL) = 4 before age 25].

3. CAG-Age-Product (CAP) score >70, as calculated using the CAP formula: AGE × (CAG - 30) / 6.49.

4. Score <26 on the Montreal Cognitive Assessment (MoCA) at Screening, indicating the presence of cognitive impairment.

Exclusion Criteria:

For All

1. 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.

2. Receive any prohibited medications within 30 days of Screening and during participation in the study

Additional criteria for participants with HD only

3. Had gastric bypass surgery, has a gastric sleeve or lap band, or has had any related procedures that interfere with gastrointestinal transit.

4. Receive any prohibited medications within 30 days of Screening and during participation in the Study.

5. 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 90 days or 5 half-lives (whichever is longer), unless the participant was enrolled solely in the placebo arm of the study.