Alzheimer's Disease in Down SyndromeLIFE-DSRNCT04149197
Down Syndrome Clinical Trials - Study of Alzheimer's Disease in Down Syndrome
An observational study of Alzheimer's Disease in Down Syndrome, run by LuMind IDSC Foundation at 14 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 25 years and older
- Conditions the study is for
- Alzheimer's Disease in Down Syndrome
- How many people
- 252 participants
- Where
- 14 sitesUnited States
- Study dates
- 30 Jun 2019to 17 Apr 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 25 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Males and females, aged 25 and older, with a diagnosis of DS, typically supported by karyotype analysis documenting full trisomy for chromosome 21 or complete unbalanced translocation of chromosome 21.
Who may be able to join
- 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 21. 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.
Who cannot join
None listed.
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
United States14 sites
Emory University
Atlanta, Georgia, 30322
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Massachusetts General Hospital
Boston, Massachusetts, 02114
Massachusetts General Hospital
Charlestown, Massachusetts, 02129
Rush University Medical Center
Chicago, Illinois, 60612
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Case Western Reserve University
Cleveland, Ohio, 044106
Duke University Medical Center
Durham, North Carolina, 27710
Kansas University Medical Center
Fairway, Kansas, 66205
University California Irvine
Irvine, California, 92697
University of California, San Diego
La Jolla, California, 92093
University Of Kentucky
Lexington, Kentucky, 40536
Advocate Health
Park Ridge, Illinois, 60068
Barrow Neurological Institute
Phoenix, Arizona, 85013
About this study
Official title: The Down Syndrome Clinical Trials Network (DS-CTN) Study of Alzheimer's Disease in Down Syndrome
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.
Read the detailed 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.
- Conditions
- Alzheimer's Disease in Down Syndrome
- Keywords
- Down Syndrome, Alzheimer's Disease
- Medical subject headings
- Down Syndrome, Alzheimer Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: through study completion, an average of 2 years
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
Measured: through study completion, an average of 2 years
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).
Measured: through study completion, an average of 2 years
Health Measures
New-onset seizures or significantly increased frequency of seizures
Measured: through study completion, an average of 2 years
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.
Measured: through study completion, an average of 2 years
Secondary outcomes (3)
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.
Measured: through study completion, an average of 2 years
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.
Measured: through study completion, an average of 2 years
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.
Measured: through study completion, an average of 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2019
- Study start30 Jun 2019
- Primary completion17 Apr 2024
- Study completion17 Apr 2024
Why it stopped: COVID delayed study timelines. Competing studies began enrolling at our sites.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alzheimer's Disease in Down SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 252 participants (actual)
Sponsor and investigators
- Lead sponsor
- LuMind IDSC Foundation
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lois Kelly, Study director, LuMind IDSC Foundation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04149197
- Sponsor's protocol ID
- ADC-059-LIFE-DSR
- Acronym
- LIFE-DSR
- Status last verified
- Jun 2024
- First submitted
- 26 Sep 2019
- Last updated
- 27 Jun 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Down Syndrome Clinical Trials Network (DS-CTN) Study of Alzheimer's Disease in Down Syndrome
- ClinicalTrials.gov ID
- NCT04149197
- Acronym / sponsor ID
- LIFE-DSR / ADC-059-LIFE-DSR
- Conditions
- Alzheimer's Disease in Down Syndrome
- Keywords
- Down Syndrome, Alzheimer's Disease
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: COVID delayed study timelines. Competing studies began enrolling at our sites.
- Enrolment
- 252 participants (actual)
- Lead sponsor
- LuMind IDSC FoundationUniversity, hospital or other
- Study start
- 30 Jun 2019 (actual)
- Primary completion
- 17 Apr 2024 (actual)
- Study completion
- 17 Apr 2024 (actual)
- First posted
- 4 Nov 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 25 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Males and females, aged 25 and older, with a diagnosis of DS, typically supported by karyotype analysis documenting full trisomy for chromosome 21 or complete unbalanced translocation of chromosome 21.
Key inclusion criteria (6 listed)
- 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 21. 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.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Cognitive Measure
Time frame: 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.
Functional Measure
Time frame: 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
Behavioral Measure
Time frame: through study completion, an average of 2 years
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).
Health Measures
Time frame: through study completion, an average of 2 years
New-onset seizures or significantly increased frequency of seizures
Behavioral Measure
Time frame: 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.
3 secondary outcomes
Exploratory Outcome Measure Preliminary composite measure from the scales being used in LIFE-DSR
Time frame: through study completion, an average of 2 years
Exploratory Outcome Measure Novel multi-domain instrument for AD-DS development
Time frame: through study completion, an average of 2 years
Exploratory Outcome Measure Potential Screening Measures
Time frame: through study completion, an average of 2 years
Results and safety
The study was stopped early (reason given: COVID delayed study timelines. Competing studies began enrolling at our sites.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jun 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lois Kelly study director
LuMind IDSC Foundation
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Alzheimer's Disease in Down Syndrome, run by LuMind IDSC Foundation at 14 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 27 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: COVID delayed study timelines. Competing studies began enrolling at our sites.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cognitive Measure (time frame: through study completion, an average of 2 years). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jun 2024.