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Results Announced from Clinical Trial of Idebenone in Primary Progressive MS

March 9, 2018

  • Results from a 3-year clinical trial conducted by researchers at the U.S. National Institutes of Health suggest that an oral compound called idebenone, a synthetic version of a dietary supplement called coenzyme Q10, was safe in people with primary progressive MS.
  • After two years on treatment, there were no differences in disability progression or other indicators of benefit between those taking idebenone and those on inactive placebo.
  • Individuals who participated in the trial are being invited to join a one-year followup observational study in which everyone will take idebenone for a year, to further evaluate its effectiveness and safety.
  • This study’s trial design and the outcome measures used will help inform knowledge about progressive MS, and aid in the design of future studies aimed at treating progressive forms of multiple sclerosis.
 
DETAILS
Background: Idebenone is a synthetic form of a naturally occurring compound known as coenzyme Q10, a common dietary supplement. Preliminary research had suggested that idebenone might be able to limit nervous system damage to slow progressive disability in MS. Idebenone is being tested in other neurological and muscular conditions.
 
The Study: This 3-year, phase I/II clinical trial was conducted by Bibiana Bielekova, MD, and others at the National Institutes of Neurological Disorders and Stroke. The trial recruited 77 people with primary progressive MS; ultimately 66 completed the trial. For the first year, participants were monitored, and then for the following two years, half received oral idebenone and half received inactive placebo. A variety of testing occurred over the course of the trial, including MRI imaging, blood and spinal fluid testing, and measures of disability to monitor safety and signs of potential benefit. The team developed a disability rating scale called CombiWISE to estimate potential benefit.
 
The preliminary results were announced by a company, Santhera Pharmaceuticals, which markets idebenone for another condition. According to the company’s press release, the treatment was safe and well tolerated. The assessments of disability progression and other tests showed no apparent benefit of idebenone over placebo after two years of treatment.
 
Individuals who participated in the trial are being invited to join a one-year followup observational study in which everyone will take idebenone for a year, to further evaluate its effectiveness and safety.
 
Conclusions: Although idebenone was not shown to slow primary progressive MS over two years, the trial design and the outcome measures used will help inform knowledge about progressive MS, and aid in the design of future studies aimed at treating progressive forms of multiple sclerosis.

About Multiple Sclerosis

Multiple sclerosis is an unpredictable, often disabling disease of the central nervous system that disrupts the flow of information within the brain, and between the brain and body. Symptoms range from numbness and tingling to blindness and paralysis. The progress, severity and specific symptoms of MS in any one person cannot yet be predicted, but advances in research and treatment are leading to better understanding and moving us closer to a world free of MS. Most people with MS are diagnosed between the ages of 20 and 50, with at least two to three times more women than men being diagnosed with the disease. MS affects more than 2.3 million people worldwide.

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