The AIMM (Acipimox in Mitochondrial Myopathy) trial is a clinical trial to determine whether a drug, acipimox, can be used to improve muscle symptoms in patients with mitochondrial myopathy (muscle weakness).
The trial is funded by the Medical Research Council and is being run by The Newcastle upon Tyne Hospitals NHS Foundation Trust along with Newcastle University. It is a single centre trial and all participants will be seen in Newcastle upon Tyne.
Acipimox is a drug currently used to treat some patients with high cholesterol and diabetes. However, some laboratory studies have indicated that it might also help improve energy production by mitochondria within muscle cells. Therefore, we are keen to see whether treatment with acipimox improves muscle symptoms in patients with muscle weakness or fatigue due to mitochondrial myopathy.
Participants on the trial will receive 12 weeks (3 months) treatment with either acipimox or placebo (a pill that does not contain active medicine but looks exactly like the study drug). The design of the trial means that neither the participant nor their doctors will know whether they are receiving acipimox or placebo (called a double-blinded trial). A number of tests (trial assessments) will be done before the start of treatment and at the end of 12 weeks to determine whether treatment with the trial medication has had an effect. These tests includes a skeletal muscle biopsy, blood tests, functional/exercise tests and a number of questionnaires.
AIMM participants will be required to attend a screening visit followed by two trial visits (one before the start of treatment and one at the end of trial) at the trial site in Newcastle upon Tyne (each visit may take place across several days). Weekly telephone follow-up calls will also be made to each participant.
To be considered for the trial, patients must be aged 16 years or over and have a m.3243A>G mutation or a single large-scale mtDNA deletion (where a piece of DNA is missing from lots of copies of mitochondrial DNA in each cell) and have evidence of muscle myopathy.