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

Summary

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.

Timeline

Start

2010-02

Primary completion

2010-11

Completion

2010-11

Outcome

Outcome not reported

Stopped: “Terminated for Commercial Reasons. There were no safety issues involved in the decision to terminate the study.”

Drugs

Indications