A 28-Day Randomized, Placebo-Controlled, Double-Blind, Parallel Groups and Normative Comparison Study to Evaluate the Effect of SAGE-718 on Functioning Capacity in Participants With Huntington's Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 69
- 试验地点
- 1
- 主要终点
- Difference in Huntington's Disease Cognitive Assessment Battery (HD-CAB) Composite Score Between Participants With HD vs HP at Baseline
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 25 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •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.
- •Genetically confirmed disease with cytosine, adenine, and guanine (CAG) expansion ≥
- •At screening, Unified Huntington's Disease Rating Scale (UHDRS) Total Functional Capacity (TFC) >6 and <13, suggesting no more than a moderate level of functional impairment.
- •No features of juvenile HD.
- •CAG-Age-Product (CAP) score >70, as calculated using the CAP formula: Age × (CAG - 30) / 6.
- •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).
排除标准
- •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.
研究组 & 干预措施
SAGE-718
Participants with HD will receive SAGE-718 1.2 milligrams (mg), orally, once daily for up to 28 days.
干预措施: SAGE-718 (Drug)
Placebo
Participants with HD will receive SAGE-718-matching placebo, orally, once daily for up to 28 days.
干预措施: Placebo (Drug)
结局指标
主要结局
Difference in Huntington's Disease Cognitive Assessment Battery (HD-CAB) Composite Score Between Participants With HD vs HP at Baseline
时间窗: Baseline
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).
次要结局
- Number of Participants With HD With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs(Up to Day 42)
- Number of Participants With HD With Clinically Significant Change From Baseline in Vital Sign Measurements(Up to Day 42)
- Number of Participants With HD With Clinically Significant Change From Baseline in Clinical Laboratory Assessments(Up to Day 42)