mitoPK
Research type
Research Study
Full title
[11C]PK11195 PET as a biomarker to diagnose and monitor natural history in mitochondrial disease.
IRAS ID
208982
Contact name
Patrick F Chinnery
Contact email
Sponsor organisation
Cambridge University Hospitals NHS Foundation Trust and University of Cambridge
Clinicaltrials.gov Identifier
609/M/C/1691, Cambridge University Insurance reference
Duration of Study in the UK
6 years, 6 months, 12 days
Research summary
Mitochondrial disorders have emerged as one of the most common inherited neurological disorders which affect multiple organ systems. They are caused by mutations in either mitochondrial DNA or nuclear genes that code for mitochondrial proteins, and affect approximately 1:5000 of the UK population. The clinical picture is heterogeneous, overlapping with many common brain and metabolic disorders. This makes it challenging to diagnose, and patients often undergo invasive testing e.g., a muscle biopsy to measure their mitochondrial function. Although the advent of high throughput genetic testing improved the situation, there is still a need to develop new diagnostic approaches using non-invasive techniques.
There are currently no treatments for multi-system mitochondrial disorders, in part due to the lack of objective clinical biomarkers which show a change over a period of time.
The aim of this pilot study is to determine the suitability of the radioactive compound, [11C]PK11195, as a diagnostic biomarker in the study of mitochondrial disease. [11C]PK11195 binds selectively to a small translocator protein (TSPO), found predominantly on the mitochondrial outer membrane. Introducing this compound into the body and performing non-invasive imaging techniques allows us to visualise [11C]PK11195 binding - a potential measure of mitochondrial abundance. This method could be used in future to confirm the on-target activity of potential therapeutic agents, and give a preliminary indicator of their efficacy. Despite being available for over a decade, this compound has not yet been studied in patients with mitochondrial disease.
Study participants will be patients of both genders (18-70 years) with diagnosed mitochondrial disease. The study will be completed at the Wolfson Brain Imaging Centre. Participants will first undergo a neurological examination and will then be injected with a small amount of [11C]PK11195 and undergo a combined PET/MRI scan. Participation will conclude as soon as the scan is complete.REC name
London - Riverside Research Ethics Committee
REC reference
16/LO/2093
Date of REC Opinion
22 Dec 2016
REC opinion
Further Information Favourable Opinion