Evaluation of MC1 density and longitudinal change in FA Patient
Research type
Research Study
Full title
Evaluation of density and longitudinal change in Mitochondrial Complex 1 in patients with Friedreich's Ataxia and Healthy Volunteers
IRAS ID
294625
Contact name
Eugenii Rabiner
Contact email
Sponsor organisation
Imanova Limited trading as Invicro
Duration of Study in the UK
1 years, 3 months, 29 days
Research summary
Friedreich's Ataxia is a rare disease that can damage the nervous system and can cause mobility problems. One of the characteristics of this disease is damage to the mitochondria. Mitochondria are parts of the normal cells that help the cell make its energy. One part of the mitochondria is the mitochondrial complex 1(MC1). We hope to measure the amount of MC1 in the heart and brain in patients with Friedreich's Ataxia using PET and MRI scans. We are interested to evaluate the regional differences and longitudinal changes in density of MC1 in the brain and the heart over 12 months period. We will also enroll Healthy Volunteers to undergo PET and MRI scans of the heart only, for comparison to the cardiac data obtained in the patients.
Previously we have measured the amount of MC1 in the brain using PET radiotracer called [18F]BCPP-EF. PET (positron emission tomography) is a medical imaging method, which uses marker molecules labelled with a small radioactive tag (called PET tracers) to measure the quantities of various molecules in the human body. We would now like to see if it is possible to use [18F]BCPP-EF to measure MC1 in the heart. We hope to use MC1 as a measure of the effectiveness of therapies to treat Friedreich’s Ataxia in the future. All subjects will have 1 screening visit.
Eligible patients, will receive 2 PET(Computed Tomography) scans and 2 MRI (magnetic resonance imaging) scans at 12 months apart. Participants will be exposed to radiation levels equivalent to just over 3 years of naturally occurring background radiation.
Healthy volunteers will receive 1 PET and 1 MRI scan of the heart, for comparison with the cardiac data from patients.
The brain data obtained in patients will be compared to that of healthy volunteers of comparable age and sex from the Invicro database.REC name
Wales REC 7
REC reference
21/WA/0136
Date of REC Opinion
3 Jun 2021
REC opinion
Further Information Favourable Opinion