A Feasibility Study of Bezafibrate in Mitochondrial Myopathy
Research type
Research Study
Full title
A Feasibility Study of Bezafibrate in Mitochondrial Myopathy
IRAS ID
167358
Contact name
Hannah Steele
Contact email
Sponsor organisation
Newcastle upon Tyne Hospitals NHS Foundation Trust
Eudract number
2015-000508-24
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 0 months, 1 days
Research summary
Mitochondrial diseases are rare, inherited disorders arising due to deficient energy production within the cells of the body. Consequently, the typical clinical features occur in organs with high energy requirements. Mitochondrial disorders exhibit highly variable clinical effects, both between individuals and within families. Characteristic symptoms include muscle weakness (myopathy), hearing loss, migraine, epilepsy and stroke like episodes in addition to diabetes and heart problems. Mitochondrial disorders can therefore impact considerably on both quality of life and life expectancy. Despite this, no proven disease modifying treatments are available.
Pre-clinical studies have identified that several existing medications improve mitochondrial function. Of these, bezafibrate has the best supportive data and, because it is already licensed as a treatment for high blood fats, has a well characterised side effect profile. We therefore want to assess whether bezafibrate can improve mitochondrial function and energy production, and thereby the clinical symptoms of mitochondrial disease.
To do this we will ask ten people with mitochondrial muscle disease to take bezafibrate three times daily for twelve weeks.
We will assess:
• whether bezafibrate improves mitochondrial function and energy production in humans;
• at what dose of bezafibrate this effect is demonstrated;
• the safety and tolerability of bezafibrate in mitochondrial myopathy.
Participants will be asked to have a muscle biopsy before and after bezafibrate treatment, as well as blood tests every week.
We will also assess several other measures including:
• Novel markers of mitochondrial disease in blood and magnetic resonance imaging (MRI) of skeletal and cardiac muscle;
• The effect of bezafibrate on measures of exercise capacity;
• Physical activity level;
• Disease burden using several clinically validated measures;
• Quality of life using three clinically validated scales.If indicated, the results of this trial would inform a larger placebo-controlled trial of bezafibrate in mitochondrial disease.
We have received funding from the MRC's Confidence in Concept fund awarded to Newcastle University.
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
15/NE/0166
Date of REC Opinion
12 Jun 2015
REC opinion
Further Information Favourable Opinion