A study to determine the best outcome measures — ways to evaluate the effects of a treatment — for use in clinical studies in Becker muscular dystrophy (BMD) and sporadic (nongenetic) inclusion-body myositis (sIBM) is underway at Nationwide Children's Hospital in Columbus, Ohio, under the supervision of neurologist Jerry Mendell working with physical therapists Linda Lowes and Lindsay Alfano.
Mendell directs the Center for Gene Therapy, the Senator Paul D. Wellstone Cooperative Muscular Dystrophy Research Center, and the MDA neuromuscular disease clinic at Nationwide Children's. Lowes and Alfano are doing the direct patient testing for this study.
Conducting clinical trials in BMD and sIBM, Mendell says, has been hampered by a lack of knowledge of their "natural history" (usual disease course) and the related lack of reliable ways to measure any change from the expected course induced by an experimental treatment.
Participants in the outcome measures study will be asked to perform timed functional tests, such as standing up from a chair and walking for six minutes, as well as tests of muscle strength.
If participants wish to be contacted about upcoming clinical trials for which they may be eligible, their information will be added to a list for that purpose.
Contact study coordinators Linda Lowes at Linda.Lowes@nationwidechildrens.org or Lindsay Alfano at Lindsay.Alfano@NationwideChildrens.org; or call (614) 722-6881 in Columbus, Ohio.
For more information
For more information about this and other studies at the Center for Gene Therapy at Nationwide Children's, see Center for Gene Therapy Clinical Studies.