Down Syndrome Clinical Trials - Study of Alzheimer's Disease in Down Syndrome

Alzheimer's Disease in Down Syndrome Clinical Trial

— LIFE-DSR  

Official title:

The Down Syndrome Clinical Trials Network (DS-CTN) Study of Alzheimer's Disease in Down Syndrome

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04149197
• Other study ID #: ADC-059-LIFE-DSR
• Status: Active, not recruiting
• Start date: June 30, 2019
• Est. completion date: December 30, 2026

Study information

• Verified date: March 2024
• Source: LuMind IDSC Foundation
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 252
• Est. completion date: December 30, 2026
• Est. primary completion date: December 30, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 25 Years and older

Eligibility

Inclusion Criteria:

1. Age 25 years or older

2. Diagnosis of DS, typically supported by karyotype analysis documenting full trisomy for chromosome 21 or complete unbalanced translocation of chromosome 21. Karyotype analysis is not required for study entry

3. 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.

4. 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.

5. 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.

Related Conditions & MeSH terms

• Alzheimer Disease
• Alzheimer's Disease in Down Syndrome
• Down Syndrome
• Syndrome

Locations

Country	Name	City	State
United States	Emory University	Atlanta	Georgia
United States	Kennedy Krieger Institute	Baltimore	Maryland
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Massachusetts General Hospital	Charlestown	Massachusetts
United States	Rush University Medical Center	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Case Western Reserve University	Cleveland	Ohio
United States	Duke University Medical Center	Durham	North Carolina
United States	Kansas University Medical Center	Fairway	Kansas
United States	University California Irvine	Irvine	California
United States	University of California, San Diego	La Jolla	California
United States	University Of Kentucky	Lexington	Kentucky
United States	Advocate Health	Park Ridge	Illinois
United States	Barrow Neurological Institute	Phoenix	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
LuMind IDSC Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Cognitive Measure	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.	through study completion, an average of 2 years
Primary	Functional Measure	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	through study completion, an average of 2 years
Primary	Behavioral Measure	Dementia Questionnaire for People with Learning Disabilities (DLD) - Measures specific cognitive and functional deterioration as a result of dementia, and functional deterioration as a result of severe sensory or psychiatric problems. Range of total possible scores: 0 - 100 50 Questions with a possible range of 0 - 2 per question. Higher scores reflect worse performance.; Score Calculation: Categories are mixed throughout the questionnaire. To calculate the score at the end, scores on each page are added up and categorizes into Cognitive Scores, or SCS (categories 1 - 3: Short-term memory, Long-term memory, and Spatial & Temporal Orientation) and Social Scores, or SOS (Categories 4 - 8: Speech, Practical Skills, Mood, Activity & Interest, Behavioral Disturbance).	through study completion, an average of 2 years
Primary	Health Measures	New-onset seizures or significantly increased frequency of seizures	through study completion, an average of 2 years
Primary	Behavioral Measure	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.	through study completion, an average of 2 years
Secondary	Exploratory Outcome Measure Preliminary composite measure from the scales being used in LIFE-DSR	To derive a preliminary composite measure from the scales being used in LIFE-DSR that is most sensitive to change in this population and which can be validated in a future prospective study. If the participant achieves a score 60 or above on the SIB, the Down Syndrome Mental Status Examination (DS-MSE) will also be administered. The maximum score is 100.	through study completion, an average of 2 years
Secondary	Exploratory Outcome Measure Novel multi-domain instrument for AD-DS development	To integrate and further validate a novel and highly sensitive multidomain instrument for AD-DS upon its completion (Professor A. Strydom, Kings College, London). This instrument is being developed under a separate protocol and upon its validation will be integrated into LIFE-DSR to evaluate its responsiveness to change in clinically important domains.	through study completion, an average of 2 years
Secondary	Exploratory Outcome Measure Potential Screening Measures	Individual subscales from the larger test batteries included in the LIFE-DSR Primary Outcome Measures, Severe Impairment Battery (SIB) with Shoebox Test, the Down Syndrome Mental Status Examination (DS-MSE), NPI, and Vineland3. These could then be further validated in a future prospective study.; SIB - 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; Scores above 60 mean the participant completes the Shoebox Memory Test.; Vineland-3 -Total score range: 0 - 140; DLD - Questions with a possible range of 0 - 2 per question. Higher scores reflect worse performance.; NPI - The score for each subscale is the product of severity and frequency and the total score is the sum of all subscales.	through study completion, an average of 2 years