MC1 in Friedreich’s Ataxia and Healthy Volunteers
Research type
Research Study
Full title
Mitochondrial Complex 1 (MC1) density in patients with Friedreich’s Ataxia and healthy volunteers
IRAS ID
257578
Contact name
Eugenii A. Rabiner
Contact email
Sponsor organisation
Imanova Limited trading as Invicro
Duration of Study in the UK
1 years, 2 months, 30 days
Research summary
Friedreich’s Ataxia is a rare disease that causes damage to the nervous system and movement problems. One of the characteristics of this disease is damage to the mitochondria. Mitochondria are parts of normal cells that help the cell make its energy. One part of the mitochondria is called mitochondrial complex 1 (MC1). We hope to measure the amount of MC1 in the heart in healthy volunteers and in patients with Friedreich’s Ataxia in this study. We’re interested in the amount of MC1 in the heart as most patients with Friedreich’s Ataxia have cardiac problems. Previously we have measured the amount of MC1 in the brain using a 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.
Healthy volunteers and Friedreich’s Ataxia patients will have 1 screening visit. Eligible participants (up to 26 healthy men and women and up to 12 patients, aged 18-65) will receive either 1 or 2 PET scans depending on which part of the study they are taking part in. The patients will also complete 1 MRI (magnetic resonance imaging) scan.
Participants will be exposed to radiation levels equivalent to just over 3 years of naturally occurring background radiation.REC name
Wales REC 5
REC reference
19/WA/0070
Date of REC Opinion
28 Feb 2019
REC opinion
Favourable Opinion