AIMM - Acipimox in Mitochondrial Myopathy Study

What is this study about?

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. 

Who can take part? 

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 (muscle weakness).

What's involved in taking part?

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 testing 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.

Are there any risks?

Acipimox is a commonly used, licensed medication however as with all medications there is a risk that some people who take them may experience side effects. Before agreeing to take part in the trial all potential participants will be screened to make sure that they are suitable to take the trial medication and will have potential side effects discussed with them.

Participation in the trial will involve providing blood samples, having two skeletal muscle biopsies (one at the beginning of the trial and one at the end) and completing a number of functional/exercise tests. Again, before participants agree to be entered onto the trial they will have details of all the study assessments and activities fully explained to them and all efforts will be made to minimise any potential discomfort from these tests.

Who will benefit?

We hope that the information that we collect from this study will help us understand whether acipimox could be a useful future treatment for muscle myopathy in mitochondrial disease.

We also hope that the results will help us better understand the movement and exercise tests we are going to use in this clinical trial, so that we can improve the application of these testing techniques in the mitochondrial clinic.

However, we cannot guarantee that there will be a direct benefit to participants from taking part in this trial. 

How do I find out more?

If you are a patient who would like further information about the AIMM trial, please contact Jan Gebbie or Angela Green, telephone: 0191 208 3105, email: [email protected] or [email protected].