Deep and Frequent Phenotyping Study (2018)
Principal Investigator:
Dr Vanessa Raymont
Approved Research ID:
40876
Approval date:
February 12th 2020
Lay summary
Alzheimer's disease remains one of the most common unmet medical needs today. Without a single treatment for the disease process itself, clinical trials are failing almost certainly because they are performed too late in the disease process. Trials need to be performed in the preclinical or prodromal phase but such designs necessitate the use of biomarkers. The purpose of our study is to provide such biomarkers. Success in doing so will speed trials, reduce costs, increase productivity and ultimately contribute to more effective pipelines for early clinical drug development. This aligns closely with UK Biobank's goal of improving prevention and treatment of dementia. We plan to enlist 250 people into the study, 200 with AD before symptoms have started. We seek to work with UK Biobank to identify these people in a screening process making use of data already collected in UKBiobank, with the permission of participants. Those people meeting the study inclusion criteria will have a series of tests including PET and MRI imaging, other brain scans (EEG and MEG), measures in the eye, of gait and movement, and of cognition using standard approaches and twith connected devices including smart phones. In addition participants will have blood tests and spinal fluid tests and all of these will be repeated many times over the period of one year. These data will be used to find a biomarker of progression to be used in clinical trials of potential therapies. Given the intensive nature of the proposal, we conducted a six month pilot study in people with mild memory problems. All assessments were completed with the exception of repeat lumbar puncture where headache was encountered at baseline (an expected occurrence in less than 10% of people), in one participant who took an unanticipated holiday, and in a small number of cases of equipment failure. We worked closely with the Alzheimer's Society who formed a stakeholder consultation group and conducted participant feedback studies using questionnaires, focus groups and individual interviews. The feedback received was universally positive, no participants found any unacceptable and the stakeholder consultation group helped to inform protocol development.