Acipimox in Mitochondrial Myopathy

• Study stage: Closedown/Analysis
• Sponsor: The Newcastle upon Tyne Hospitals NHS Foundation Trust
• Funder: MRC- DPFS
• Therapeutic area: Neurological
• Type of study: CTIMP

Aim: To determine whether Acipimox can be repurposed as a potent stimulator of mitochondrial biogenesis. Participants will be treated with Acipimox or matched placebo to determine what effect treatment has on the Adenosine Triphosphate (ATP) content of the muscle.

Primary outcome:

To establish if ATP content in skeletal muscle is increased, as measured by luminescence assay, in patients with mitochondrial disease and muscle involvement following treatment with Acipimox for 12 weeks.

• Population: Adult
• Design: RCT Adaptive
• Setting: Secondary care
• Planned Sample Size: 120

Website: https://www.thelilyfoundation.org.uk/lily-research/uk-trials-research-studies/aimm-acipimox-mitochondrial-myopathy-study/