Melas Study

Melas Treatment Trial 2011

A Phase 2A, double blind, randomised, placebo-controlled, 28 day, two-arm, parallel group study of A0001 in patients with the 3243AG mitochondrial DNA point mutation and evidence of impaired mitochondrial function

The Newcastle team has just launched a new phase 2A treatment trial for mitochondrial disease (m.3243A>G MELAS). The study is a randomised control trial using A0001, or alpha tocopherol quinone, a coenzyme Q10 analogue, and the main end point is cardiac function assessed by MRI. There are no invasive procedures and the treatment trial lasts for 1 month. The team are keen to recruit as many patients as possible who are known to have the m.3243A>G mitochondrial DNA mutation.

If you have any patients that are interested, please contact:

Dr. Chinnery Email: or
Dr. Grainne Gorman Email:
and the study team will pass on more details either to yourself or the patient.

Melas Study 2007

Clinical Trials in Mitochondrial Disease

There are two clinical trials ongoing in Newcastle studying the effects of and novel agents in MELAS (mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes, and LHON (Leber hereditary optic neuropathy). The research team are actively recruiting at present.

Please contact:
Dr Grainne Gorman ( for the MELAS trial
Dr Patrick Yu Wai Man ( for the LHON trial
Professor Patrick Chinnery ( for both studies.

For further details regarding the study please click on the link to follow Melas Study
Please note that this is a members' only section and you will be required to log in using member details. If you do not have your user name and password please email the IICN at and your details will be forwarded to you.

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