Idebonone in MELAS

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Dr. Michio Hirano and The Mitochondrial Group at Columbia Presbyterian Hospital are conducting a clinical trial for patients with MELAS and the A3243G mitochondrial DNA point mutation and to seek everyone's help in recruiting patients.

They are conducting a Phase IIa study of idebenone with MRS lactate as primary outcome. It is hoped that this trial will be the first step towards a subsequent clinical efficacy trial. To participate, patients must meet the following inclusion criteria:

(1) 8-64 years old,

(2) >37 kg(=82 lbs) weight,

(3) documented 3243 mutation,

(4) history of seizures or stroke.

Additional inclusion criteria will be determined at the first visit. In some cases we may be able to verify the presence of the mutation if documentation missing.

We are excited that a controlled clinical trial is available for people burdened by MELAS and hope that you will let your patients know about this opportunity.

The study is IRB-approved.


Interested patients can contact Kris Engelstad at 212 305 6834 or

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Last revised 03-Sep-2010 by Steven Leber