BMD, IBM: Outcome Measures Study Seeks Participants

A study to determine the natural history and outcome measures for clinical trials in Becker MD and sporadic (nongenetic) inclusion-body myositis is open in Ohio

Determining the right outcome measures to use in a clinical trial is a crucial step.
Article Highlights:
  • Reliable outcome measures and natural histories for Becker muscular dystrophy and sporadic inclusion-body myositis are lacking.
  • Researchers at Nationwide Children's Hospital in Columbus, Ohio, are recruiting people with BMD or sIBM for a study to find reliable outcome measures and determine natural history for these disorders.
  • Participants who wish to be contacted about future trials can join a list.
by Margaret Wahl on November 18, 2013 - 5:00am

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.

To participate

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.

Your rating: None Average: 4 (6 votes)
MDA cannot respond to questions asked in the comments field. For help with questions, contact your local MDA office or clinic or email publications@mdausa.org. See comment policy