Safety and Efficacy Study of A0001 in Patients With the A3243G Mitochondrial DNA Point Mutation

A Phase 2a, Double Blind, Randomized, Placebo-controlled, 28 Day, Two-arm, Parallel Group Study of A0001 in Patients With the A3243G Mitochondrial DNA Point Mutation and Evidence of Impaired Mitochondrial Function

This is a phase 2a, double-blind, placebo-controlled, single-center study. Twenty-one patients who qualify for the study will be randomly assigned to either active drug or placebo. The study will take place at Newcastle University. Patients will have a 66% chance of getting active drug. Patients will be required to take study treatment orally twice a day for 28 days. A baseline visit will occur within 21 days of screening visit. All patients will be followed for 1 week after completion of study or early withdrawal from the study.

Inclusion Criteria:

• Diagnosis of neuromuscular symptoms due to the A3243G mitochondrial DNA point mutation
• PCR/ATP ratio of <1.9 following the Cardiac MRS at screening

Exclusion Criteria:

• Any major illness not due to the A3243G mitochondrial DNA point mutation in the past three months or any significant ongoing chronic medical illness, especially significant central nervous neurological disease limiting capacity to carry out the study
• Use of any investigational product within the past 30 days