Investigators will determine how best to measure and track symptoms of dysferlin deficiency, with the goal of defining outcome measures for use in clinical trials
Update (July 17, 2014): This story has been updated to reflect that recruitment for this study has been extended through Aug. 31, 2014; that Sarah Shira is now the person to contact at the Jain Foundation; and that the foundation is now located in Seattle.
A new, multinational study of type 2B limb-girdle muscular dystrophy (LGMD2B) and Miyoshi myopathy — both of which result from mutations in the gene for the muscle protein dysferlin and are known as dysferlinopathies or dysferlin deficiency — is inviting people with either disorder to participate.
This International Clinical Outcome Study for Dysferlinopathy seeks to:
The study, funded by the Jain Foundation, is being conducted at 14 centers in the United States, Europe, Japan and Australia. U.S. study sites are in Washington, D.C., St. Louis, Mo., Columbus, Ohio, and Charlotte, N.C.
You do not need to live near a study center, as funds are available for participants and a helper to travel to the nearest site.
If you are not sure whether you have a dysferlinopathy (LGMD2B or Miyoshi myopathy), funds are available to conduct the necessary tests to find out. Contact Sarah Shira, director of patient relations and diagnostics at the Jain Foundation in Seattle, at email@example.com or (425) 882-1440.
Prospective participants must:
Recruitment begins in September 2012 and will continue until Aug. 31, 2014.
For more information, see International Clinical Outcome Study for Dysferlinopathy (LGMD2B/Miyoshi), or send an email to firstname.lastname@example.org.
About Clinical Trials
A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.