Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05358821 | 28-Day Study of SAGE-718 on Functioning Capacity in Participants With Huntington's Disease | PHASE2 | COMPLETED | 69 | — | — | May 26, 2022 | Apr 10, 2024 | Sep 15, 2025 | 14 | United States, Canada |
| NCT03787758 | A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Patients With Huntington's Disease - Part B | PHASE1 | COMPLETED | 6 | — | — | Feb 28, 2019 | Oct 7, 2019 | Sep 17, 2025 | 2 | United States |
HD-CAB assesses cognitive function using 6 subtests:Symbol Digit Modalities Test-correctly coded items(0-110); One Touch Stockings of Cambridge(OTS)-mean time to reach correct response(range not defined); Trail Making Test Trail B (TMT-B)-time to complete task(0-240 sec); Hopkins Verbal Learning Test Revised-total correct recall trials(0-48); Paced Tapping Test-reciprocal of standard deviation(SD) of intertap intervals (range not defined); Emotion Recognition Test-negative emotions correctly identified(0-24). Values of OTS \& TMT B are multiplied by -1 to represent higher is better direction as other tests. Each of 6 subtests scores was transformed to z-score(range not defined;low negative value represents cognitive impairment), which is calculated by subtracting mean and dividing by SD of HP at baseline. HD-CAB composite=average of 6 z-scores. Negative HD-CAB of HD participants=decline in cognitive function relative to HP. Assessment is relative to reference group(healthy population).
| Arm | Type | Description |
|---|---|---|
| SAGE-718 | EXPERIMENTAL | Participants with HD will receive SAGE-718 1.2 milligrams (mg), orally, once daily for up to 28 days. |
| Placebo | PLACEBO_COMPARATOR | Participants with HD will receive SAGE-718-matching placebo, orally, once daily for up to 28 days. |
| Healthy Participants | NO_INTERVENTION | HP enrolled in this study will not receive any investigational product (IP) (SAGE-718 or placebo). |
| Name | Type | Description |
|---|---|---|
| SAGE-718 | DRUG | SAGE-718 oral softgel lipid capsules |
| Placebo | DRUG | SAGE-718-matching oral capsules |
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. Be ambulatory, able to travel to the study center, and, judged by the investi...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novartis AG Sponsored ADR | NVS | 2 | PHASE3 | Votoplam |
| Neurocrine Biosciences, Inc. | NBIX | 2 | PHASE3 | Valbenazine |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | intra-striatal rAAV5-miHTT |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |