Trial-Ready Cohort - Down syndrome (TRC-DS)
Research type
Research Study
Full title
Alzheimer’s Clinical Trial Consortium for Down Syndrome (ACTC- DS): Trial-Ready Cohort -Down Syndrome (TRC-DS)
IRAS ID
287116
Contact name
John O'Brien
Contact email
Sponsor organisation
Cambridgeshire and Peterborough NHS Foundation Trust & the University of Cambridge
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 7 months, 1 days
Research summary
Down syndrome (DS) is caused by three copies of chromosome 21 instead of the usual two. This has serious consequences for the health of people with DS, including an extreme risk for Alzheimer’s disease (AD). AD neuropathology (brain tissue disease changes) includes deposits of tau and “beta-amyloid” (A-beta) proteins and occurs in the almost all people with DS who are 40 years or older. The most likely cause is due to an extra copy of the APP gene, which is located on chromosome 21, causing an over-production of A-beta protein and thus resulting in AD. It can take over a decade to establish the AD pathology and over ten years before the disease manifests clinically. AD is the primary cause of death the over the age of 35-highlighting the pressing need for the inclusion of individuals with DS in clinical trials that can target the brain pathology before it manifests clinically. Biomarkers include blood tests or brain scans can be used as proxy measures of aspects of disease. Adults with DS exhibit significant changes in AD-related biomarker measures of AD between the ages of 35-55, which is the age range that AD-related cognitive decline and dementia typically manifests. We are one of 17 international sites that aim to enrol 120 non-demented adults with DS (ages 25-55); locally we aim to enrol 8 adults. We will characterise clinically (including cognitive tests), and measure biomarkers (blood-based, spinal fluid and brain scans) and select participants most likely to benefit from a preventive clinical trial. The selected cohort will be offered the opportunity to be part of an international clinical trial to prevent AD using treatment targeting A-beta. The trial will start after sufficient participants have enrolled, expected to be in 2023. An application for the clinical trial will be undertaken separately.
REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
23/NW/0002
Date of REC Opinion
27 Jan 2023
REC opinion
Further Information Favourable Opinion