Alzheimer's Clinical Trial Consortium for Down Syndrome (ACTC-DS) Trial-Ready Cohort - Down Syndrome (TRC-DS)

Plain English Summary

Trial-Ready Cohort-Down Syndrome (TRC-DS) is a Not Applicable clinical trial sponsored by University of Southern California studying Down Syndrome, Alzheimer Disease, Dementia. This study is gathering information from adults with Down syndrome to help plan future clinical trials for Alzheimer's disease. It is for adults with Down syndrome, aged 25-55, who are generally healthy and do not have dementia. Participants will have regular check-ups, cognitive tests, genetic and biomarker tests, and imaging scans. The main alternative is to wait for specific Alzheimer's treatment trials to become available. The trial aims to enroll 550 participants.

Official Summary

The purpose of the Trial-Ready Cohort - Down Syndrome (TRC-DS) is to enroll 120 healthy adults with Down syndrome (DS), between the ages of 25-55, into a trial ready cohort (TRC), and up to 550 participants in total including co-enrolled in the Alzheimer Biomarkers Consortium - Down Syndrome (ABC-DS) study. Participants enrolled in the TRC-DS will undergo longitudinal cognitive and clinical assessment, genetic and biomarker testing, as well as imaging and biospecimen collection. Using these outcome measures, researchers will analyze the relationships between cognitive measures and biomarkers of Alzheimer's disease (AD) to identify endpoints for AD clinical trials in DS that best reflect disease progression. To learn more about the study and participating sites, visit our study website at: https://www.trcds.org/. TRC-DS is collaborating with the Alzheimer's Disease Biomarker Consortium-Down Syndrome (ABC-DS) to allow study participants to be concurrently enrolled in both ABC-DS and TRC-DS, referred to as "co-enrollment". ABC-DS is a longitudinal, observational research study that is overseen at University of Pittsburgh Coordinating Center. ABC-DS participants who express interest in potentially joining a clinical trial in the future and who meet TRC-DS eligibility criteria, may choose to co-enroll in TRC-DS at an ABC-DS Site. Co-enrolled participants will adhere to the ABC-DS protocol and schedule of activities, but agree to share their data with the TRC-DS team and to receive invitations for future participation in clinical trials. Fore more information on ABC-DS please visit https://www.nia.nih.gov/research/abc-ds or http://abcds.pitt.edu/.

Who Can Participate

Here is what you need to know about eligibility for this trial. You can join if you have a diagnosis of Down syndrome and are between 25 and 55 years old. You must be in good general health, without a dementia diagnosis, and able to undergo tests. You cannot join if you have other serious medical conditions that could interfere with the study, or if you cannot have an MRI scan. Individuals with a history of certain cancers (excluding non-melanoma skin cancer) or significant blood/thyroid abnormalities may not be eligible. This trial is studying Down Syndrome, Alzheimer Disease, Dementia, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

The primary outcome measures how well the study can enroll participants, which is crucial for ensuring that future treatment studies have enough people to provide reliable results. The specific primary outcome measures are: Enrollment of participants into the Trial-Ready Cohort in DS (TRC-DS). (5 years). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This study does not have a traditional clinical phase designation. It may be an observational study that follows patients without intervening in their care, an expanded access or compassionate use program, or other non-interventional research. These studies contribute valuable data about disease progression, treatment patterns, and patient outcomes.

Why This Trial Matters

This trial is important because it aims to identify the best ways to measure Alzheimer's disease progression in people with Down syndrome, paving the way for effective treatment trials. This research targets Down Syndrome, Alzheimer Disease, Dementia, where improved treatment options are needed.

Investor Insight

This observational study is a critical step in building the infrastructure for future therapeutic trials in a currently underserved population, potentially opening a significant market for Alzheimer's The large enrollment target of 550 participants suggests significant investment in this program.

Is This Trial Right for Me?

Ask your doctor if this study is a good fit for you and what the tests involve. Participation includes regular visits for cognitive assessments, medical check-ups, and sample collection over several years. You will need a reliable study partner (like a family member or caregiver) to help provide information about your health. This trial is currently recruiting participants. The trial is being conducted at 20 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: OBSERVATIONAL
• Enrollment: 550 participants

Primary Outcomes

• Enrollment of participants into the Trial-Ready Cohort in DS (TRC-DS). (5 years)

Secondary Outcomes

• Change in cognition as measured by the Modified Cued Recall Test (Baseline and Month 48 or until enrollment into a clinical trial)
• Change in behavior as measured by the Neuropsychiatric Inventory (NPI) (Screening and Month 48, or until enrollment into a clinical trial)
• Change in cognition as measured by the Down Syndrome Mental Status Exam (DSMSE) (Screening and Month 48, or until enrollment into a clinical trial)
• Change in behavior as measured by the Vineland 3 (Informant Version) (Baseline and Month 48, or until enrollment into a clinical trial)
• Change in cognition as measured by the National Task Group Early Detection Screen for Dementia (NTG-EDSD) (Baseline and Month 48, or until enrollment into a clinical trial)

Full Eligibility Criteria

Inclusion Criteria:

1. Diagnosis of DS (including trisomy 21, mosaic trisomy 21, Robertsonian translocation trisomy 21 or partial trisomy 21) (as confirmed by genetic testing or medical record review)
2. Provision of signed and dated informed consent form; this includes adults with DS who can provide consent, or for whom an LAR provides consent on behalf of the individual to participate. Adults with DS who cannot consent must sign and date an assent accompanied with a signed and dated consent by legally authorized representative (LAR).
3. Stated availability and willingness to comply with all study procedures and availability for the duration of the study or until referred to a clinical trial
4. Male or female, aged 25-55 inclusive
5. In good general health as evidenced by medical history with no diagnosis of dementia
6. Permitted CNS-active medications, stable in dose for at least 4 weeks or longer. If new medications have been started, medical monitoring team will review on case by case basis to recommend timing of baseline cognitive testing
7. Adequate visual and auditory acuity to allow neuropsychological testing
8. Mental Age of 4 years or greater (based upon the Kaufman Brief Intelligence Test, Second Edition, KBIT-2, verbal age equivalent, or based upon medical records)
9. Ability to complete KBIT-2 with IQ equal to or greater than 40
10. Must speak English or Spanish fluently
11. Must have a reliable Study Partner (may be caregiver, sibling, parent) who is capable of providing correct information about the participant's clinical symptoms and history

Exclusion Criteria:

1. Any significant disease or unstable medical condition that could affect participation (i.e., unstable psychiatric disease, unstable cardiac problems, chronic renal failure, chronic hepatic disease, severe pulmonary disease)
2. Participants in whom magnetic resonance imaging (MRI) is contraindicated including, but not limited to, those with a non-compatible pacemaker, presence of MRI-incompatible metallic fragments near the eyes or spinal cord, or cochlear implant (Dental fillings do not present a risk for MRI)
3. Participants unable to complete MRI procedure
4. History, within the last 5 years of a primary or recurrent malignant disease with the exception of non-melanoma skin cancers, resected cutaneous squamous cell carcinoma in situ, basal cell carcinoma, cervical carcinoma in situ, or in situ prostate cancer with normal prostate-specific antigen post-treatment
5. Clinically significant abnormalities in B12 or TFTs that might interfere with the study. A low B12 is exclusionary, unless follow-up labs (homocysteine (HC) and methylmalonic acid (MMA)) indicate that it is not physiologically significant. A high TSH is exclusionary unless follow up T3/T4 levels indicate that it is not physiologically significant.
6. Clinically significant abnormalities in screening laboratories
7. For participants undergoing CSF collection: a current blood clotting or bleeding disorder, or significantly abnormal PT or PTT at screening or if on anti-coagulation therapy (e.g. warfarin)
8. Concurrent participation in a clinical trial for an investigational product or concurrent participation in longitudinal study with overlapping outcome measures/procedures is prohibited with the exception of ABC-DS co-enrollment or as approved by project director
9. Participants whom the investigator deems to be otherwise ineligible. The Investigators should consult with the Coordinating Center on any issues that may disqualify the participant from participation in future clinical trials to determine whether enrollment into TRC-DS would be appropriate

Trial Locations

• Barrow Neurological Institute, Phoenix, Arizona, United States
• University of California, Irvine School of Medicine, Co-Enrolling through ABC-DS Only, Orange, California, United States
• Linda Crnic Institute for Down Syndrome, University of Colorado, Aurora, Colorado, United States
• Advocate Medical Group Adult Down Syndrome Center, Park Ridge, Illinois, United States
• Indiana University, Indianapolis, Indiana, United States
• University of Kansas Medical Center, Kansas City, Kansas, United States
• University of Kentucky, Co-Enrolling through ABC-DS Only, Lexington, Kentucky, United States
• Massachusetts General Hospital, Co-Enrolling through ABC-DS Only, Boston, Massachusetts, United States
• Washington University, St. Louis, St Louis, Missouri, United States
• Cleveland Clinic, Lou Ruvo Center for Brain Health, Las Vegas, Las Vegas, Nevada, United States
• ...and 10 more locations

Frequently Asked Questions

Related Conditions

• Down Syndrome
• Alzheimer Disease
• Dementia
• Cognitive Impairment
• Neurodegenerative Diseases
• Geriatrics
• Developmental Disabilities
• Clinical Trial Preparedness
• Biomarker Research
• Aging

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