I graduated from Bristol University in 2004, and subsequently worked as a general paediatrician in South Wales and the South East. This broad basis is helpful for a condition as varied and complex as mitochondrial disease. I joined the Newcastle mitochondrial clinical team in 2018. My main focus is delivering clinical trials and translational research.
AIMM study (Acipimox in Mitochondrial Myopathy): Randomised, double-blinded, placebo-controlled, adaptive design trial of the efficacy of acipimox in patients with Mitochondrial Myopathy
Principal Investigators: Professor Grainne Gorman
Other Team Members Involved: Prof Bobby McFarland, Professor Robert Taylor, Dr Jane Newman, Dr Oksana Porogyelova, Huizhong Su, Laura Bone, Laura Brown and Clare Masarella
I am leading the AIMM (Acipimox in Mitochondrial Myopathy) clinical trial. This trial aims to investigate whether acipimox, currently used to manage lipids in patients with diabetes, can treat some of the debilitating muscle problems experienced by patients with mitochondrial disease.
Other clinical trials I have been or am currently involved with include:
Abliva: A phase Ia/Ib, multiple-site study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of KL1333 after a single and multiple ascending oral doses in healthy subjects and patients with primary mitochondrial disease
Reneo: An open-label study to evaluate the safety and tolerability of 12 weeks treatment with oral REN001 in patients with primary mitochondrial myopathy, with an optional extension of treatment
Khenergyze: A Phase IIb double-blind, randomised, placebo controlled, multi-centre, confirmative three-way crossover study on cognitive function with two doses of KH176 in subjects with a genetically confirmed mitochondrial tRNALeu(UUR) m.3243A>G mutation
LION: Leigh syndrome: Investigating Outcome measures & Natural history (LION) – a prospective, longitudinal cohort study
Sponsor/Funder: The Newcastle Upon Tyne Hospitals Foundation Trust/Medical Research Council – Developmental Pathway Funding Stream (DPFS)