The Down Syndrome Clinical Trials Network (DS-CTN) Study of Alzheimer's Disease in Down Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- 入组人数
- 252
- 试验地点
- 13
- 主要终点
- Cognitive Measure
研究概览
简要总结
The primary objective is to characterize trajectories of change on the primary outcome measures in this study population through longitudinal collection of measures of cognition, function, behavior, and health status.
详细描述
The DS-CTN will engage in rigorous, high quality research to better understand AD-DS and the medical needs of this population. The LIFE-DSR study will utilize the DS-CTN network to achieve these goals through evidence-based research and a combination of proven and novel methodologies. The network will initially recruit and employ experts and clinical trial sites who have access to adults with DS and whose focus is on AD-DS.
This trial also aims to develop sensitive and well-validated assessment instruments of cognition, behavior and function appropriate for future clinical trials directed at AD-DS in adults with DS ages 25 and older.
In addition, this LIFE-DSR study will serve as a parent protocol that includes one or more sub-studies, each of which has its own protocol and consent form. These sub-studies will recruit from eligible participants enrolled in this LIFE-DSR protocol.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 25 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 25 years or older
- •Diagnosis of DS, typically supported by karyotype analysis documenting full trisomy for chromosome 21 or complete unbalanced translocation of chromosome
- •Karyotype analysis is not required for study entry
- •Participants, or Legal Authorized Representative, and their study partner if applicable, in the opinion of the investigator, are able to understand and willing to sign written informed consent.
- •Participants must have a study partner who has frequent interaction with the participant on a regular basis, will agree to participate in annual clinic visits, can provide accurate responses to questions about the participant, and facilitate participation in the study visits, in the opinion of site PI or study coordinator.
- •Participant and study partner must be capable of reliably completing study assessments.
- •Exclusion Criteria Participants and study partners who, in the opinion of the site PI, are not able to complete trial procedures or adhere to the schedule of study assessments will be excluded from study participation.
排除标准
- 未提供
结局指标
主要结局
Cognitive Measure
时间窗: through study completion, an average of 2 years
Severe Impairment Battery (SIB) with Shoebox Test. If the participant achieves a score 60 or above on the SIB, the Down Syndrome Mental Status Examination (DS-MSE) will also be administered. Subscale Ranges: Social Interaction - 0 - 6 Orientation - 0 - 8 Visuospatial Ability - 0 - 8 Construction - 0 - 12 Language - 0 - 56 Memory - 0 - 16 Praxis - 0 - 8 Attention - 0 - 15 Orienting to name - 0 - 4 Higher scores reflect better performance for each subscale. Scores above 60 mean the participant completes the Shoebox Memory Test.
Behavioral Measure
时间窗: through study completion, an average of 2 years
Neuropsychiatric Inventory (NPI) - Neuropsychiatric Inventory (NPI) evaluates both the frequency and severity of 10 neuropsychiatric features, including delusions, hallucinations, agitation/aggression, dysphoria, anxiety, euphoria, apathy, disinhibition, irritability and lability, and aberrant motor behavior, as well as evaluates sleep and appetite/eating disorders. Frequency assessments range from 1 (occasionally, less than once per week) to 4 (very frequently, once or more per day or continuously). Severity assessments range from 1 (mild) to 3 (severe). The score for each subscale is the product of severity and frequency and the total score is the sum of all subscales.
Functional Measure
时间窗: through study completion, an average of 2 years
The Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Vineland-3 is an adaptive behavior measure used to assess intellectual/developmental/other disabilities. Total score range: 0 - 140 Expressive Communication: 0 - 98 49 questions - range: 0 - 2 per Q Written Communication: 0 - 76 38 questions - range: 0 - 2 per Q Personal Daily Living Skills - 0 - 110 55 questions - range: 0 - 2 per Q
Health Measures
时间窗: through study completion, an average of 2 years
New-onset seizures or significantly increased frequency of seizures
次要结局
- Exploratory Outcome Measure Preliminary composite measure from the scales being used in LIFE-DSR(through study completion, an average of 2 years)
- Exploratory Outcome Measure Novel multi-domain instrument for AD-DS development(through study completion, an average of 2 years)
- Exploratory Outcome Measure Potential Screening Measures(through study completion, an average of 2 years)