AIMM: Acipimox in Mitochondrial Myopathy
Research type
Research Study
Full title
Randomised, double-blinded, placebo-controlled, adaptive design trial of the efficacy of acipimox in patients with Mitochondrial Myopathy
IRAS ID
244484
Contact name
Gràinne Gorman
Contact email
Sponsor organisation
Newcastle Upon Tyne Hospitals NHS Foundation Trust
Eudract number
2018-002721-29
Duration of Study in the UK
2 years, 8 months, 24 days
Research summary
Mitochondria are tiny cellular structures that play a critical role in cellular energy (ATP) production. When these 'powerhouses' malfunction, it may result in chronic illnesses known as mitochondrial diseases. It has been estimated that mitochondrial diseases affect up to 15,000 adults (and a similar numbers of children) in the UK.
Mitochondrial diseases are rare, inherited conditions in which muscle symptoms including muscle weakness, fatigue, and pain are extremely prevalent and often debilitating. Patients and carers have helped to design this study, and they identified muscle symptoms to be the most important target for any new drug treatments.
We plan to test a drug (acipimox) that has originally been used to treat high cholesterol and improve diabetic control. The drug has also been shown to boost ATP levels within muscle cells and it is this function that we wish to test in patients with mitochondrial disease to relieve the debilitating muscle symptoms.
The benefits of using a drug that has already been used in other conditions means that we already understand the behaviour and side effects of the drug in humans, and we already know how to administer the drug in addition to understanding about effective doses.
We will invite patients with a genetically proven diagnosis of m.3243A>G-related mitochondrial disease, or single large-scale mtDNA deletion, with muscle involvement.
Participants will be asked to take a tablet 3 times a day, for 12 weeks. At the start and end of treatment, the participants will be asked to come to Newcastle Upon Tyne to undergo a small muscle biopsy. They will also be asked to undergo a number of tests of everyday functioning (muscle strength, walking, mental agility), as well as complete questionnaires asking about their mental health and well-being.
REC name
HSC REC B
REC reference
18/NI/0199
Date of REC Opinion
4 Dec 2018
REC opinion
Further Information Favourable Opinion