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Trial of Idebenone in People with Primary Progressive Multiple Sclerosis

See All Multiple Sclerosis Trials

The National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health, is looking for individuals to participate in clinical studies. Participating in clinical trials allows you to play an active role in research on the nature and causes of many disorders of the brain and nervous system, and to possibly help physician-scientists develop future treatments. The information below is designed to help you quickly learn about actively recruiting research studies for which you or someone you know may be eligible.


This study evaluates the safety and effectiveness of using idebenone to treat primary progressive multiple sclerosis (PP-MS). MS is an inflammatory disorder of the central nervous system that progressively weakens and destroys the pathways of the nervous system. About 10 to 15 percent of individuals develop PP-MS—characterized by progressive accumulation of disability from the disease onset—without any marked improvements or relapses. There are currently no effective treatments for PP-MS.

Idebenone is a man-made drug that is similar to a naturally occurring compound known as coenzyme Q10, a common dietary supplement. Research data suggest that idebenone may be able to limit demyelination and death of brain cells and thereby slow or halt the progression of neurological dysfunction such as that occurring in MS.

The study will last 3 years and will be divided into two parts: a 1-year pre-treatment baseline and 2 years of treatment with either idebenone or placebo. Half of patients will receive idebenone and half will receive only placebo, an inactive drug, during this study. The pre-treatment part will last for approximately 5 clinic visits over 1 year. After these 5 visits, participants will receive a 6-month supply of study medication (either idebenone or placebo) to take three times a day. Participants will continue to have regular followup clinic visits with brain magnetic resonance imaging (MRI) scans, blood tests, and other evaluations of brain and nervous system function.


Phase 1/2

Eligibility Criteria:

Please follow this link for trial eligibility information to share with your doctor.

Study Design: 

Interventional, Purpose: Treatment

Study Locations: 

Bethesda, Maryland

For more information:

Contact: Joan Ohayon, CRNP; Tel: 301-496-0064; Email:

Last Reviewed July 17, 2012